CLINSQUARE: CLINICAL RESEARCH COURSES
Monday, December 23, 2024
CLINSQUARE: Clinical Rsearch Courses by LIFEPRONOW
Home Blog Page 12

Avacta group announces Phase I clinical study of AVA6000

admin
-
0
Avacta group announces Phase I clinical study of AVA6000

Sep 01, 2022: “Avacta Group plc, a clinical stage oncology drug company and developer of powerful diagnostics based on its innovative Affimer® and pre|CISION™ platforms, announces that the first-in-human Phase I trial (ALS-6000-101) of AVA6000 Pro-doxorubicin will advance to the fourth dose cohort of patients following a positive review of the safety and tolerability data from the dosing of the third cohort.

Avacta’s Safety Data Monitoring Committee (SDMC), comprised of clinicians currently recruiting patients, has completed its review of the safety data from the third cohort dosed with AVA6000 at 160mg/m2 in the ongoing Phase I trial.

Following this review, the SDMC has recommended that the clinical trial continues as planned and escalates to the next dose of AVA6000 at 200mg/m2.

AVA6000 is a novel form of doxorubicin that has been modified with Avacta’s pre|CISION™ FAP-activated delivery platform to improve its safety and therapeutic index.

AVA6000 has been designed to limit cell penetration of the drug, and therefore its cell killing effect, until it is specifically activated by fibroblast activation protein α (FAP) which is in high concentration in many solid tumours compared with healthy tissues.

The resulting reduced exposure of healthy tissues to active doxorubicin has the potential to significantly increase its therapeutic index by reducing the incidence of adverse effects, including cardiotoxicity and myelosuppression. Anthracyclines such as doxorubicin, a generic chemotherapeutic agent, with a market size that is expected to grow to $1.38bn by 2024, are widely used as part of standard of care in several tumour types, but its use is limited by cumulative dose toxicity associated with cardiomyopathy.

Dr Alastair Smith, Chief Executive Officer of Avacta Group, commented:

“We are very much encouraged by this recommendation from the SDMC to move onto the fourth dose cohort in our ongoing ALS-6000-101 Phase 1 dose escalation study.

This very positive progress reflects the safety profile and tolerability demonstrated in patients enrolled in the study to date.”

https://avacta.com/phase-i-clinical-study-of-ava6000-to-advance-to-the-fourth-cohort/

FDA Authorizes Moderna, Pfizer-BioNTech Bivalent COVID-19 Vaccines for Use as a Booster Dose

admin
-
0
FDA Authorizes Moderna, Pfizer-BioNTech Bivalent COVID-19 Vaccines for Use as a Booster Dose

August 31, 2022: “The U.S. Food and Drug Administration amended the emergency use authorizations (EUAs) of the Moderna COVID-19 Vaccine and the Pfizer-BioNTech COVID-19 Vaccine to authorize bivalent formulations of the vaccines for use as a single booster dose at least two months following primary or booster vaccination.

The bivalent vaccines, which we will also refer to as “updated boosters,” contain two messenger RNA (mRNA) components of SARS-CoV-2 virus, one of the original strain of SARS-CoV-2 and the other one in common between the BA.4 and BA.5 lineages of the omicron variant of SARS-CoV-2.

The Moderna COVID-19 Vaccine, Bivalent, is authorized for use as a single booster dose in individuals 18 years of age and older.

The Pfizer-BioNTech COVID-19 Vaccine, Bivalent, is authorized for use as a single booster dose in individuals 12 years of age and older. 

The monovalent COVID-19 vaccines that are authorized or approved by the FDA and have been administered to millions of people in the United States since December 2020 contain a component from the original strain of SARS-CoV-2. 

What you need to know:

  • The authorized bivalent COVID-19 vaccines, or updated boosters, include an mRNA component of the original strain to provide an immune response that is broadly protective against COVID-19 and an mRNA component in common between the omicron variant BA.4 and BA.5 lineages to provide better protection against COVID-19 caused by the omicron variant. 
  • The BA.4 and BA.5 lineages of the omicron variant are currently causing most cases of COVID-19 in the U.S. and are predicted to circulate this fall and winter.
    In June, the agency’s Vaccines and Related Biological Products Advisory Committee voted overwhelmingly to include an omicron component in COVID-19 booster vaccines.
  • For each bivalent COVID-19 vaccine, the FDA based its decision on the totality of available evidence, including extensive safety and effectiveness data for each of the monovalent mRNA COVID-19 vaccines, safety and immunogenicity data obtained from a clinical study of a bivalent COVID-19 vaccine that contained mRNA from omicron variant BA.1 lineage that is similar to each of the vaccines being authorized, and nonclinical data obtained using a bivalent COVID-19 vaccine that contained mRNA of the original strain and mRNA in common between the BA.4 and BA.5 lineages of the omicron variant.
  • Based on the data supporting each of these authorizations, the bivalent COVID-19 vaccines are expected to provide increased protection against the currently circulating omicron variant. Individuals who receive a bivalent COVID-19 vaccine may experience side effects commonly reported by individuals who receive authorized or approved monovalent mRNA COVID-19 vaccines.
  • With today’s authorization, the monovalent mRNA COVID-19 vaccines are not authorized as booster doses for individuals 12 years of age and older.
  • The agency will work quickly to evaluate future data and submissions to support authorization of bivalent COVID-19 boosters for additional age groups as we receive them.

Who is eligible to receive a single booster dose and when:

  • Individuals 18 years of age and older are eligible for a single booster dose of the Moderna COVID-19 Vaccine, Bivalent if it has been at least two months since they have completed primary vaccination or have received the most recent booster dose with any authorized or approved monovalent COVID-19 vaccine. 
  • Individuals 12 years of age and older are eligible for a single booster dose of the Pfizer-BioNTech COVID-19 Vaccine, Bivalent if it has been at least two months since they have completed primary vaccination or have received the most recent booster dose with any authorized or approved monovalent COVID-19 vaccine.

“The COVID-19 vaccines, including boosters, continue to save countless lives and prevent the most serious outcomes (hospitalization and death) of COVID-19,” said FDA Commissioner Robert M. Califf, M.D. “

As we head into fall and begin to spend more time indoors, we strongly encourage anyone who is eligible to consider receiving a booster dose with a bivalent COVID-19 vaccine to provide better protection against currently circulating variants.”

The Moderna COVID-19 Vaccine, Bivalent and the Pfizer-BioNTech COVID-19 Vaccine, Bivalent contain mRNA from the SARS-CoV-2 virus.

The mRNA in these vaccines is a specific piece of genetic material that instructs cells in the body to make the distinctive “spike” protein of the original virus strain and the omicron variant lineages BA.4 and BA.5. The spike proteins of BA.4 and BA.5 are identical. 
    
“The FDA has been planning for the possibility that the composition of the COVID-19 vaccines would need to be modified to address circulating variants. We sought input from our outside experts on the inclusion of an omicron component in COVID-19 boosters to provide better protection against COVID-19.

We have worked closely with the vaccine manufacturers to ensure the development of these updated boosters was done safely and efficiently.

The FDA has extensive experience with strain changes for annual influenza vaccines. We are confident in the evidence supporting these authorizations,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research.

“The public can be assured that a great deal of care has been taken by the FDA to ensure that these bivalent COVID-19 vaccines meet our rigorous safety, effectiveness and manufacturing quality standards for emergency use authorization.”

For each of the bivalent COVID-19 vaccines authorized today, the FDA evaluated immunogenicity and safety data from a clinical study of a booster dose of a bivalent COVID-19 vaccine that contained a component of the original strain of SARS-CoV-2 and a component of omicron lineage BA.1.

The FDA considers such data as relevant and supportive of vaccines containing a component of the omicron variant BA.4 and BA.5 lineages. Furthermore, data pertaining to the safety and effectiveness of the current mRNA COVID-19 vaccines, which have been administered to millions of people, including during the omicron waves of COVID-19, contributed to the agency’s evaluation.

Data Supporting the Moderna COVID-19 Vaccine, Bivalent Authorization

To evaluate the effectiveness of a single booster dose of the Moderna COVID-19 Vaccine, Bivalent for individuals 18 years of age and older, the FDA analyzed immune response data among approximately 600 individuals 18 years of age and older who had previously received a two-dose primary series and one booster dose of monovalent Moderna COVID-19 Vaccine.

These participants received a second booster dose of either the monovalent Moderna COVID-19 Vaccine or Moderna’s investigational bivalent COVID-19 vaccine (original and omicron BA.1) at least 3 months after the first booster dose.

After 28 days, the immune response against BA.1 of the participants who received the bivalent vaccine was better than the immune response of those who had received the monovalent Moderna COVID-19 Vaccine.

The safety of a single booster dose of the Moderna COVID-19 Vaccine, Bivalent for individuals 18 years of age and older is supported by safety data from a clinical study which evaluated a booster dose of Moderna’s investigational bivalent COVID-19 vaccine (original and omicron BA.1), safety data from clinical trials which evaluated primary and booster vaccination with the monovalent Moderna COVID-19 Vaccine, and postmarketing safety data with the monovalent Moderna COVID-19 Vaccine. 

The safety data accrued with the bivalent vaccine (original and omicron BA.1) and with the monovalent Moderna COVID-19 Vaccine are relevant to the Moderna COVID-19 Vaccine, Bivalent because these vaccines are manufactured using the same process.

The clinical study that evaluated the safety of a booster dose of the bivalent vaccine (original and omicron BA.1) included approximately 800 participants 18 years of age and older who had previously received a two dose primary series and one booster dose of the monovalent Moderna COVID-19 Vaccine, and then at least 3 months later, received a second booster dose with either the monovalent Moderna COVID-19 Vaccine or Moderna’s investigational bivalent COVID-19 vaccine (original and omicron BA.1). 

Among the study participants who received the bivalent vaccine, the most commonly reported side effects included pain, redness and swelling at the injection site, fatigue, headache, muscle pain, joint pain, chills, swelling of the lymph nodes in the same arm of the injection, nausea/vomiting and fever. 

Data Supporting the Pfizer-BioNTech COVID-19 Vaccine, Bivalent Authorization

To evaluate the effectiveness of a single booster dose of the Pfizer-BioNTech COVID-19 Vaccine, Bivalent for individuals 12 years of age and older, the FDA analyzed immune response data among approximately 600 adults greater than 55 years of age who had previously received a 2-dose primary series and one booster dose with the monovalent Pfizer-BioNTech COVID-19 Vaccine.

These participants received a second booster dose of either the monovalent Pfizer-BioNTech COVID-19 Vaccine or Pfizer-BioNTech’s investigational bivalent COVID-19 vaccine (original and omicron BA.1) 4.7 to 13.1 months after the first booster dose.

After one month, the immune response against BA.1 of the participants who received the bivalent vaccine was better than the immune response of those who had received the monovalent Pfizer-BioNTech COVID-19 Vaccine.

The safety of a single booster dose of the Pfizer-BioNTech COVID-19 Vaccine, Bivalent for individuals 12 years of age and older is based on safety data from a clinical study which evaluated a booster dose of Pfizer-BioNTech’s investigational bivalent COVID-19 vaccine (original and omicron BA.1), safety data from clinical trials which evaluated primary and booster vaccination with the monovalent Pfizer-BioNTech COVID-19 Vaccine, and postmarketing safety data with the monovalent Pfizer-BioNTech COVID-19 Vaccine. 

The safety data accrued with the bivalent vaccine (original and omicron BA.1) and with the monovalent Pfizer-BioNTech COVID-19 Vaccine are relevant to Pfizer-BioNTech COVID 19 Vaccine, Bivalent because these vaccines are manufactured using the same process. 

The clinical study that evaluated the safety of a booster dose of the bivalent vaccine (original and omicron BA.1) included approximately 600 participants greater than 55 years of age who had previously received a 2-dose primary series, one booster dose of the monovalent Pfizer-BioNTech COVID-19 Vaccine, and then 4.7 to 13.1 months later, received a second booster dose of either the monovalent Pfizer-BioNTech COVID-19 Vaccine or Pfizer-BioNTech’s investigational bivalent COVID-19 vaccine (original and omicron BA.1).

Among the study participants who received the bivalent vaccine, the most commonly reported side effects included pain, redness and swelling at the injection site, fatigue, headache, muscle pain, chills, joint pain, and fever. 

The fact sheets for both bivalent COVID-19 vaccines for recipients and caregivers and for healthcare providers include information about the potential side effects, as well as the risks of myocarditis and pericarditis. 

With today’s authorization, the FDA has also revised the EUA of the Moderna COVID-19 Vaccine and the Pfizer-BioNTech COVID-19 Vaccine to remove the use of the monovalent Moderna and Pfizer-BioNTech COVID-19 vaccines for booster administration for individuals 18 years of age and older and 12 years of age and older, respectively.

These monovalent vaccines continue to be authorized for use for administration of a primary series for individuals 6 months of age and older as described in the letters of authorization.

At this time, the Pfizer-BioNTech COVID-19 Vaccine remains authorized for administration of a single booster dose for individuals 5 through 11 years of age at least five months after completing a primary series of the Pfizer-BioNTech COVID-19 Vaccine.

The amendments to the EUAs were issued to Moderna TX Inc. and Pfizer Inc.”

https://www.fda.gov/news-events/press-announcements/coronavirus-covid-19-update-fda-authorizes-moderna-pfizer-biontech-bivalent-covid-19-vaccines-use

XenpozymeTM approved by FDA as first disease-specific treatment for ASMD

admin
-
0
XenpozymeTM approved by FDA as first disease-specific treatment for ASMD

August 31, 2022: “The U.S. FDA has approved XenpozymeTM (olipudase alfa-rpcp) for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients.

Xenpozyme is the first therapy indicated specifically for the treatment of ASMD, and is currently the only approved treatment for this disease.

Bill Sibold
Executive Vice President, Head, Specialty Care at Sanofi
“Sanofi teams have been dedicated to bringing hope to patients living with ASMD and their families.

This is a devastating and extremely rare disease that affects both children and adults. The approval of Xenpozyme represents the culmination of bold work done in research and development, and our unwavering commitment to this historically overlooked community.”

ASMD, historically known as Niemann-Pick disease types A, A/B, and B, is an extremely rare, progressive genetic disease with significant morbidity and mortality. It has been estimated that there are fewer than 120 patients diagnosed with ASMD in the U.S. Approximately two-thirds of patients with ASMD in the U.S. are pediatric.

Signs and symptoms of ASMD can present in infancy, childhood, or adulthood, and may include enlarged spleen or liver, difficulty breathing, lung infections, and unusual bruising or bleeding, among other disease manifestations.

Until now, management of ASMD included supportive care to address the impact of individual symptoms and careful monitoring to detect potential disease complications.

David Guy
Parent to Kaila, age 16, living with ASMD
“As young parents, it was initially devastating to me and my wife when our daughter, Kaila, received her diagnosis of ASMD.

We faced so many unknowns when we first heard the diagnosis: what does this mean, how will this affect her, and most importantly what hope is there for a treatment option? We were grateful to find hope when we enrolled Kaila in the clinical trials for olipudase alfa.”

In the U.S., Xenpozyme received Breakthrough Therapy designation, which expedites the development and review of drugs intended to treat serious or life-threatening diseases and conditions.

The FDA evaluated Xenpozyme under Priority Review, which is reserved for medicines that represent potentially significant improvements in efficacy or safety in treating serious conditions..

In March 2022, Xenpozyme was approved in Japan under the SAKIGAKE (or “pioneer”) designation, marking the first approval for olipudase alfa anywhere in the world. In June 2022, the European Commission (EC) approved Xenpozyme for use in Europe.

ASMD represents a spectrum of disease, with two types that may represent opposite ends of a continuum referred to as ASMD type A and ASMD type B. ASMD type A/B is an intermediate form that includes varying degrees of central nervous system (CNS) involvement.

ASCEND and ASCEND-Peds clinical trials showed that Xenpozyme improved lung function and reduced spleen and liver volumes in adults and children

The approval is based on positive data from the ASCEND and ASCEND-Peds clinical trials, in which Xenpozyme showed clinically relevant improvement in lung function (as measured by diffusing capacity of the lung for carbon monoxide, or DLco) and platelet count, and reduction of spleen and liver volumes, with a demonstrated safety profile.

Melissa Wasserstein
MD, Pediatric Genetic Medicine, Albert Einstein College of Medicine and the Children’s Hospital at Montefiore
“ASMD is an extremely rare, progressive, and potentially fatal genetic disease that impacts children and adults around the world. Until now, those living with ASMD have had no FDA-approved treatment to combat this devastating condition.

I’m proud of the work that has been done and look forward to witnessing the impact that this treatment may have on those living with ASMD.”

The ASCEND trial evaluated the efficacy and safety of Xenpozyme; 31 adult patients with ASMD type A/B or type B were randomized to receive Xenpozyme or placebo for 52 weeks (primary analysis).

In the trial, Xenpozyme improved lung function, assessed as the percent change from baseline to Week 52 in predicted diffusing capacity of the lung for carbon monoxide (DLco), and reduced spleen volume, evaluated as percent change from baseline in multiples of normal (MN).

  • Twelve (12) patients treated with Xenpozyme had a mean change in percent predicted DLco from baseline (49.1%) to Week 52 (59.4%).
    This change represents a 23.9% relative improvement compared to a 3% improvement in DLco from baseline in the 17 patients from the placebo group (48.5%) to Week 52 (49.9%).
    The difference between the two arms (20.9%) was nominally statistically significant (p=0.0003).
  • Thirteen (13) patients treated with Xenpozyme had a mean reduction in spleen volume by 38.9% from baseline (11.5 MN) to Week 52 (7.2 MN) compared to a mean increase by 0.5% for the 17 patients in the placebo group from baseline (11.2 MN) to Week 52 (11.2 MN). The difference between the two arms (39.4%) was nominally statistically significant (p<0.0001).
  • Twelve (12) patients treated with Xenpozyme had a mean reduction in liver volume by 26.5% from baseline (1.4 MN) to Week 52 (1.0 MN) compared to a mean decrease of 1.8% for the 17 patients in the placebo group from baseline (1.6 MN) to Week 52 (1.6 MN).
    The difference between the two arms (24.7%) was nominally statistically significant (p<0.0001).
  • Thirteen (13) patients treated with Xenpozyme had a mean improvement in platelet count by 18.3% from baseline (109.3×109/L) to Week 52 (126.4×109/L) compared to increase by 2.7% for the 16 patients in the placebo group from baseline (115.6×109/L) to Week 52 (120.2×109/L). The difference between the two arms (15.6%) was nominally statistically significant (p=0.0280).
  • All ASCEND patients treated with Xenpozyme showed improvement in key endpoints (DLco and spleen and liver volume).
  • Most frequently reported adverse drug reactions in adults (incidence ≥10%) were headache, cough, diarrhea, hypotension, and ocular hyperemia.

The single-arm ASCEND-Peds trial studied 8 pediatric patients younger than 12 years of age with ASMD type A/B or type B who all received Xenpozyme, with a primary objective of evaluating the safety and tolerability of Xenpozyme for 64 weeks. All patients completed the study and continued in an extension trial.

The ASCEND-Peds trial also explored efficacy endpoints of progressive lung disease, spleen and liver enlargement, and platelet count. After one year of treatment (52 weeks):

  • Three (3) patients who were able to perform the test at baseline treated with Xenpozyme had a mean relative improvement of 45.9% in percent predicted DLco from baseline (48.5%) to Week 52 (70.9%) (children over the age of five were assessed if they were able to perform the test).
  • Eight (8) patients treated with Xenpozyme had mean reduction in spleen volume by 46.7% from baseline (18.3 MN) to Week 52 (9.5 MN).
  • Eight (8) patients treated with Xenpozyme had a mean reduction in liver volume by 38.1% from baseline (2.5 MN) to Week 52 (1.6 MN).
  • Seven (7) patients treated with Xenpozyme had a mean improvement in platelet count by 37.6% from baseline (136.7×109/L; n=8) to Week 52 (184.5×109/L).
  • Serious adverse reactions of anaphylactic reaction were reported in 2 (25%) Xenpozyme-treated pediatric patients.
  • Treatment-related serious adverse reactions, hypersensitivity reactions including anaphylaxis, and infusion associated reactions occurred within 24 hours of infusion and were observed in a higher percentage of pediatric patients than in adult patients.
  • Most frequently reported adverse drug reactions in pediatric patients (incidence ≥20%) were pyrexia, cough, diarrhea, rhinitis, abdominal pain, vomiting, headache, urticaria, nausea, rash, arthralgia, pruritus, fatigue, and pharyngitis.

A scientific innovation for patients living with ASMD

Xenpozyme, a hydrolytic lysosomal sphingomyelin-specific enzyme replacement therapy, is designed to replace deficient or defective acid sphingomyelinase (ASM), an enzyme that allows for the breakdown of the lipid sphingomyelin.

individuals with ASMD, the deficiency in the ASM enzyme leads to sphingomyelin accumulation in various tissues. Xenpozyme is not expected to cross the blood-brain barrier or modulate CNS manifestations of ASMD.

Xenpozyme has not been studied in patients with ASMD type A.

Xenpozyme is adminstered intravenously every two weeks, and its administration requires a dose escalation phase followed by a maintenance phase.

Xenpozyme is expected to be available in the U.S. in the coming weeks. The U.S. list price, or wholesale acquisition cost, of Xenpozyme is $7,142.00 per vial.

Actual patient out-of-pocket costs may be lower, as the list price does not reflect insurance coverage, co-pay support for eligible patients, or financial assistance from patient support programs.

Sanofi is committed to helping eligible U.S. patients access the support they need and to help reduce barriers throughout their treatment journey.”

https://www.sanofi.com/en/media-room/press-releases/2022/2022-08-31-18-30-00-2507978

FDA grants priority review to efanesoctocog alfa for people with hemophilia A

admin
-
0
FDA grants priority review to efanesoctocog alfa for people with hemophilia A

August 30, 2022: “The U.S.FDA has accepted for priority review the Biologics License Application (BLA) for efanesoctocog alfa (BIVV001) for the treatment of hemophilia A, a rare and life-threatening bleeding disorder.

The target action date for the FDA decision is February 28, 2023. Sanofi and Sobi® collaborate on the development and commercialization of efanesoctocog alfa.

Steve Pipe, MD
Professor and Director of Pediatric Hemophilia and Coagulation Disorders Program, University of Michigan
“Factor therapy remains a cornerstone of hemophilia treatment, but innovation has been needed in this area to address challenges related to bleed protection and cumbersome treatment regimens. If approved, efanesoctocog alfa can deliver close to normal factor activity levels for the majority of the week, potentially offering a new tier of protection.

Such therapeutic benefits would represent important advances in unmet medical needs for people with hemophilia A and may transform the prophylactic treatment landscape.”

The BLA is supported by data from the pivotal XTEND-1 Phase 3 study. Results were recently presented at the 30th International Society of Thrombosis and Haemostasis Congress.

The data demonstrate a clinically meaningful prevention of bleeds and superiority to prior factor prophylaxis based on an intra-patient comparison.

Efanesoctocog alfa was well-tolerated, and inhibitor development to factor VIII was not detected.

The most common treatment-emergent adverse events (>5% of participants overall) were headache, arthralgia, fall, and back pain.

Dietmar Berger, MD, PhD
Global Head of Development and Chief Medical Officer at Sanofi
“The results from the pivotal XTEND-1 Phase 3 study demonstrate efanesoctocog alfa’s ability to reduce annualized bleeding rates, which supports its potential as a therapy with best-in-disease efficacy. 

We look forward to working closely with the FDA during the review process as we aim to bring this novel therapy to the hemophilia A community.”

The FDA grants priority review to therapies that have the potential to provide significant improvements in the treatment, diagnosis, or prevention of serious conditions.

Efanesoctocog alfa received Breakthrough Therapy designation from the FDA in May 2022 and it is the first factor VIII therapy to receive this recognition.

The FDA also granted efanesoctocog alfa Orphan Drug designation in August 2017 and Fast Track designation in February 2021.

Regulatory submission in the EU will follow availability of data from the ongoing XTEND-Kids pediatric study, with both events expected in 2023.

The European Commission granted efanesoctocog alfa Orphan Drug designation in June 2019.

About Phase 3 XTEND-1 Study (NCT04161495)
The Phase 3 XTEND-1 study (NCT04161495) was an open-label, non-randomized interventional study assessing the safety, efficacy and pharmacokinetics of once-weekly efanesoctocog alfa in people 12 years of age or older (n=159) with severe hemophilia A who were previously treated with factor VIII replacement therapy. 

The study consisted of two parallel treatment arms — the prophylaxis Arm A (n=133), in which patients who had received prior factor VIII prophylaxis were treated with once-weekly intravenous efanesoctocog alfa prophylaxis (50 IU/kg) for 52 weeks, and the on-demand Arm B (n=26), in which patients who had received prior on-demand factor VIII therapy began 26 weeks of on-demand efanesoctocog alfa (50 IU/kg), then switched to once-weekly prophylaxis (50 IU/kg) for an additional 26 weeks.

The primary efficacy endpoint was the annualized bleeding rate (ABR) in Arm A, and the key secondary endpoint was an intra-patient comparison of ABR during the efanesoctocog alfa weekly prophylaxis treatment period versus the prior factor VIII prophylaxis ABR for participants in Arm A who had participated in a previous observational study (Study 242HA201/OBS16221).

About hemophilia A
Hemophilia A is a rare, genetic disorder in which the ability of a person’s blood to clot is impaired due to a lack of factor VIII. Hemophilia A occurs in about one in 5,000 male births annually, and more rarely in females.

People with hemophilia can experience bleeding episodes that can cause pain, irreversible joint damage and life-threatening hemorrhages. Factor replacement therapy remains a cornerstone of care and can be used across multiple treatment scenarios.

About efanesoctocog alfa (BIVV001)
Efanesoctocog alfa is a novel and investigational recombinant factor VIII therapy that is designed to extend protection from bleeds with once-weekly prophylactic dosing for people with hemophilia A.

It builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN® polypeptides to extend its time in circulation. It is the first investigational factor VIII therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies.

Efanesoctocog alfa is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.

About the Sanofi and Sobi collaboration
Sobi and Sanofi collaborate on the development and commercialization of Alprolix® and Elocta®/Eloctate®. 

The companies also collaborate on the development and commercialization of efanesoctocog alfa.

Sobi has final development and commercialization rights in the Sobi territory (essentially Europe, North Africa, Russia and most Middle Eastern markets).

Sanofi has final development and commercialization rights in North America and all other regions in the world excluding the Sobi territory.

About Sobi®
Sobi is a specialized international biopharmaceutical company transforming the lives of people with rare diseases. Providing sustainable access to innovative medicines in the areas of haematology, immunology and specialty care, Sobi has approximately 1,600 employees across Europe, North America, the Middle East and Asia.”

https://www.sanofi.com/en/media-room/press-releases/2022/2022-08-30-05-30-00-2506359

FDA Removes N95 Respirators from Medical Device Shortage List

admin
-
0
FDA Removes N95 Respirators from Medical Device Shortage List

August 26, 2022: “The U.S. FDA announced the removal of N95 respirators from the agency’s medical device shortage list, signaling that demand or projected demand for this type of face protection device commonly used in health care settings no longer exceeds the supply.

This action is the result of increased domestic manufacturing of N95 respirators, as well as updates to the FDA’s supply chain assessment based on engagement with industry and federal stakeholders, and the Centers for Disease Control and Prevention’s National Institute for Occupational Safety and Health’s (NIOSH) approval of new disposable N95s and reusable respirators. 

“Throughout the COVID-19 pandemic, one of the FDA’s top priorities has been to ensure frontline health care workers have access to the critical protections they need,” said Suzanne Schwartz, M.D., M.B.A., director of the FDA’s Center for Devices and Radiological Health’s Office of Strategic Partnerships and Technology Innovation.

“We have worked very closely with our partners at NIOSH, the Occupational Safety and Health Administration and with U.S. manufacturers to stabilize, rebuild and secure health care access to high-quality, single-use respirators, including those that are American-made.

Our national capacity for production of these devices is stronger and our supply chain is more resilient because of these collective efforts on behalf of the dedicated people working to save lives.” 

Since the start of the pandemic, the FDA has been closely monitoring the respirator supply chain and working with federal partners and industry to address critical shortages of personal protective equipment (PPE), including respirators, which were one of the first medical devices identified as being in critical shortage during the public health emergency.

The FDA has taken many actions to provide flexible policies and help ensure that patients and health care providers have timely and continued access to high-quality medical devices.

These actions included issuing Emergency Use Authorizations (EUAs) and guidance documents to provide recommendations and help expand the availability and capability for various protective medical devices in high demand during the COVID-19 public health emergency. 

The FDA is required by law to maintain a device shortage list to provide transparency to the American public, particularly those who use or purchase medical devices.

The FDA determines it is appropriate to remove a product code from the device shortage list when the agency finds the demand or projected demand for the device no longer exceeds the supply for a period of time. 

The FDA continues to take a phased approach to ensuring the availability of PPE and supply chain impacts.

The removal of respirators from the device shortages list does not impact the existing Enforcement Policy for Face Masks, Barrier Face Coverings, Face Shields, Surgical Masks, and Respirators During the Coronavirus Disease (COVID-19) Public Health Emergency guidance or the NIOSH-Approved Air Purifying Respirators for Use in Health Care Settings During Response to the COVID-19 Public Health Emergency EUA at this time.

These policies remain in effect. 

The FDA will continue to monitor the supply chain and update the device shortage list and device discontinuance list as the COVID-19 public health emergency evolves.”

https://www.fda.gov/news-events/press-announcements/fda-removes-n95-respirators-medical-device-shortage-list-signaling-sufficient-supply

Novartis presents new data in breast and prostate cancer at ESMO

admin
-
0
Novartis presents new data in breast and prostate cancer at ESMO

August 30, 2022: “Novartis will showcase new data from across its oncology portfolio at the European Society for Medical Oncology (ESMO) Congress 2022 with over 35 accepted abstracts from Novartis-sponsored and investigator-initiated trials including new data in advanced breast cancer and metastatic castration-resistant prostate cancer.

“We are excited to share the data being presented across our portfolio of cancer therapies, which reinforce our commitment to pursuing every possible approach to address the urgent and significant unmet medical needs of people living with cancer,” said Jeff Legos, Executive Vice President, Global Head of Oncology & Hematology Development, Novartis.

“Our presentations at ESMO will highlight our continued dedication to advancing innovative treatment options for these critical diseases.”

Key highlights of data accepted by ESMO:

MedicineAbstract TitleAbstract Number/ Presentation Details
Kisqali® (ribociclib)*

 		Pooled exploratory analysis of survival in patients (pts) with HR+/HER2− advanced breast cancer (ABC) and visceral metastases (mets) treated with ribociclib (RIB) + endocrine therapy (ET) in the MONALEESA (ML) trials
​		Abstract #205P
Poster Session
Saturday, September 10

 
Kisqali® (ribociclib)*

 		HARMONIA SOLTI-2101 / AFT-58: A head-to-head phase III study comparing ribociclib (RIB) and palbociclib (PAL) in patients with hormone receptor-positive/HER2-negative/HER2-Enriched (HR+/HER2-/HER2-E) advanced breast cancer (ABC)†
Abstract # 272TiP
Poster Session
Saturday, September 10
Piqray® (alpelisib)BYLieve trial (alpelisib [ALP] + endocrine therapy [ET]) versus real-world (RW) standard of care (SOC) in patients (pts) with PIK3CA-mutated (mut), hormone receptor-2 positive (HR+), human epidermal growth factor receptor 2-negative (HER2–) advanced 3 breast cancer (ABC) who progressed on cyclin-dependent kinase 4/6 inhibitor (CDKi) 4 therapy (tx)

 		Abstract #222P
Poster Session
Saturday, September 10

 
Tislelizumab​

 		Final Analysis of RATIONALE-301: Randomized, Phase 3 study of tislelizumab versus sorafenib as first-line treatment for unresectable hepatocellular carcinoma

 		Abstract #LBA36
Proffered Paper Session
Saturday, September 10
09:15 – 09:25 AM CEST

 
Tislelizumab​Tislelizumab (TIS) versus docetaxel (TAX) as second- or third-line therapy in previously treated patients (pts) with locally advanced non-small cell lung cancer (NSCLC): Asian versus non-Asian subgroup analysis of the RATIONALE-303 study

 		Abstract #1031P
Poster Session
Monday, September 12

 
Pluvicto™ (lutetium 177Lu vipivotide tetraxetan) (formerly referred to as 177Lu-PSMA-617)Association between prostate-specific antigen decline and clinical outcomes in patients with metastatic castration-resistant prostate cancer in the VISION trialAbstract #1372P
Poster Session
Sunday, September 11

 
Pluvicto™ (lutetium 177Lu vipivotide tetraxetan)Radiographic progression-free survival correlation with patient-relevant outcomes: a post hoc analysis of time-to-event endpoints of the VISION trial

 		Abstract #1374P
Poster Session
Sunday, September 11,

 
Prostate CancerQuality of life across three countries using a large-scale, fully digital survey of patients with prostate cancer 

 		Abstract #1401P
Poster Session
Sunday, September 11

 
Canakinumab
(ACZ885)		CANOPY-A: phase III study of canakinumab (CAN) as adjuvant therapy in patients (pts) with completely resected non-small cell lung cancer (NSCLC)

 		Abstract #LBA49
Proffered Paper Session
Sunday, September 11
09:20 – 09:30 AM CEST

 
Vijoice® (alpelisib)Clinical benefit of alpelisib in pediatric patients with PIK3CA-related overgrowth spectrum (PROS): an EPIK-P1 analysis

 		Abstract #468P
Poster Session
Monday, September 12

 

Product Information
For full prescribing information, including approved indications and important safety information about marketed products, please visit
https://www.novartisoncology.com/news/product-portfolio.

https://www.novartis.com/news/media-releases/novartis-presents-new-data-breast-and-prostate-cancer-esmo

Evusheld combination approved for prevention & treatment of COVID-19

admin
-
0
Evusheld combination approved for prevention & treatment of COVID-19

August 30, 2022: “AstraZeneca’s Evusheld (tixagevimab and cilgavimab, formerly AZD7442), a long-acting antibody combination, has been approved in Japan for both prevention (pre-exposure prophylaxis) and treatment of symptomatic disease caused by SARS-CoV-2 infection.

The decision marks the first global marketing approval for Evusheld as a treatment for COVID-19.

In prevention, Japan’s Ministry of Health, Labour and Welfare (MHLW) granted Evusheld Special Approval for Emergency for adults and adolescents (12 years of age and older weighing at least 40kg). 

Evusheld is approved for use in those whom SARS-CoV-2 vaccination is not recommended and who may have an inadequate response to a COVID-19 vaccine due to immunodeficiencies.

Recipients of Evusheld for prevention should not be currently infected with or have had recent known exposure to a person infected with SARS-CoV-2.

In treatment, Evusheld is approved for adults and adolescents (12 years of age and older weighing at least 40kg) with risk factors for severe SARS-CoV-2 infection who do not require supplemental oxygen.

Kazuhiro Tateda, M.D. Ph.D., Professor, Department of Microbiology and Infectious Disease, Toho University, Tokyo, Japan, said: “COVID-19 continues to have a significant impact on our daily lives in Japan.

Many people, including older adults, patients with comorbidities, and immunocompromised patients, remain at risk for poor outcomes from severe COVID-19. 

Evusheld will be a much-needed new option, offering long-term protection for those who do not achieve an adequate immune response after vaccination and helping prevent severe disease and death in those who do become infected.”

Itaru Matsumura, M.D., Ph.D. Professor & Chairman, Department of Hematology & Rheumatology, Kindai University Faculty of Medicine, Otsuka, Japan, said: “Despite the progress of vaccinations and stringent safety precautions, there are very large number of new infections in Japan.

The approval of Evusheld is expected to provide a non-vaccine prophylactic option for those who cannot expect a full immune response from COVID-19 vaccination, such as patients with blood cancers.”

Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “The approvals of Evusheld in Japan represent an important milestone in our ongoing efforts to help combat COVID-19 on all fronts. 

Evusheld is now the only long-acting antibody combination authorised for both COVID-19 prevention and treatment, allowing us to help protect even more vulnerable patients such as the immunocompromised from this devastating disease.”

The Japanese government has agreed to purchase 300,000 units of Evusheld (150mg each of tixagevimab and cilgavimab), and AstraZeneca is working with the government and partners to make first doses available as soon as possible.

The approvals were based on efficacy and safety data from the Evusheld clinical development programme, including the PROVENT Phase III pre-exposure prophylaxis trial, the TACKLE Phase III outpatient treatment trial, and Phase I trials, including in Japan.

In PROVENT, a 300mg intramuscular (IM) dose of Evusheld significantly reduced the risk of developing symptomatic COVID-19 by 77% (95% confidence interval (CI): 46, 90; p<0.001) COVID-19 compared to placebo at the primary analysis.

An 83% (95% CI: 66, 91) relative risk reduction was shown at a six-month median follow-up analysis, with protection from the virus lasting six months.

In TACKLE, a 600mg IM dose of Evusheld significantly reduced the relative risk of progressing to severe COVID-19 or death (from any cause) by 50% (95% confidence interval [CI] 15, 71; p=0.010) through day 29 compared to placebo in non-hospitalised patients with mild-to-moderate COVID-19 who were symptomatic for seven days or less, the trial’s primary endpoint.

In pre-specified analyses of participants who received treatment within three days of symptom onset, Evusheld reduced the risk of developing severe COVID-19 or death (from any cause) by 88% compared to placebo (95% CI 9, 98), and the risk reduction was 67% (95% CI 31, 84) when participants received Evusheld within five days of symptom onset.2  

Evusheld was generally well-tolerated in the trials.

The recommended dose for prevention of symptomatic disease caused by SARS-CoV-2 infection in Japan is 150mg of tixagevimab and 150mg of cilgavimab, administered as separate sequential IM injections.

Depending on the prevalence of SARS-CoV-2 variants, 300mg of tixagevimab and 300mg of cilgavimab may be administered for prevention.

The recommended dose for treatment of COVID-19 is 300mg of tixagevimab and 300mg of cilgavimab, administered as separate sequential IM injections.

Evusheld has been shown to retain in vitro neutralisation activity against the main Omicron variants currently circulating globally, including BA.5 and BA.2.

Evusheld is also authorised for use for pre-exposure prophylaxis (prevention) of COVID-19 in the US (emergency use), EU and many other countries.

Regulatory submissions are progressing for both prevention and treatment indications around the world.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2022/evusheld-approved-for-covid-19-in-japan.html

Pfizer and BioNTech Announce Updated COVID-19 Vaccine Data Supporting Efficacy in Children

admin
-
0
Pfizer and BioNTech Announce Updated COVID-19 Vaccine Data Supporting Efficacy in Children

August 23, 2022: “Pfizer Inc. and BioNTech SE announced updated efficacy results from a Phase 2/3 trial evaluating a three 3-µg dose series of the Pfizer-BioNTech COVID-19 Vaccine in children 6 months through 4 years of age, reinforcing previously reported interim vaccine efficacy data collected in March and April 2022.

Emergency Use Authorization (EUA) of this vaccine was granted by the U.S. Food and Drug Administration (FDA) for this age group on June 17 and an application for conditional Marketing Authorization in this age group is under review by the European Medicines Agency (EMA).This press release features multimedia.

View the full release here: https://www.businesswire.com/news/home/20220823005341/en/

Participants in the study received either the Pfizer-BioNTech COVID-19 Vaccine (3-μg) as a three-dose series or placebo (2:1 randomization). Vaccine efficacy, a secondary endpoint in the trial, was 73.2% (2-sided 95% CI: 43.8%, 87.6%) among children 6 months through 4 years of age without evidence of prior COVID-19 infection.

This analysis was based on 13 cases in the Pfizer-BioNTech COVID-19 Vaccine group (n=794) and 21 cases in the placebo group (n=351), diagnosed from March to June 2022. The study protocol specified that this formal efficacy analysis should be performed when at least 21 total symptomatic COVID-19 cases were identified, each at least seven days after the third dose.

Consistent with the time period when the cases occurred, sequencing of viral RNA from illness visit nasal swabs indicated that observed cases were primarily caused by Omicron BA.2.

Omicron BA.4 and BA.5 strains were emerging during the period of the study, with only a few cases accrued and efficacy results against these strains were inconclusive.

Consistent with our approach in adults, we are working with the FDA to prepare an EUA application for an Omicron BA.4/BA.5-adapted bivalent vaccine in children 6 months through 11 years of age.

“Building on the strong safety and immunogenicity data that led to FDA authorization of our COVID-19 vaccine for children 6 months through 4 years, we are pleased to share confirmatory evidence that a full course of vaccination helps protect against symptomatic disease, particularly during a time when the Omicron BA.2 strain was predominant,” said Albert Bourla, Chairman and Chief Executive Officer, Pfizer.

“While these results confirm that three 3-µg doses of our COVID-19 vaccine provide young children with a high level of protection at a time when the Omicron BA.2 strain was highly prevalent with a favorable safety profile, we are also developing an Omicron BA.4/BA.5-adapted bivalent vaccine in this age group to address these sublineages,” said Prof. Ugur Sahin, M.D., CEO and Co-Founder of BioNTech.

Among children ages 6 through 23 months, the vaccine was 75.8% (2-sided 95% CI: 9.7%, 94.7%) effective at preventing COVID-19, based on 4 cases in the vaccine group (n=296) and 8 cases in the placebo group (n=147), after a median of 1.9 months (range: 0.0, 4.9) follow-up after the third dose.

For children ages 2 through 4 years of age, the vaccine was 71.8% (2-sided 95% CI: 28.6%, 89.4%) effective at preventing COVID-19, based on 9 cases in the vaccine group (n=498) and 13 cases in the placebo group (n=204), after a median of 2.4 months (range: 0.0, 4.9) follow-up after the third dose.

Three 3-µg doses of the Pfizer-BioNTech COVID-19 Vaccine continue to be well-tolerated in this age group.

The majority of adverse events observed in this age group have been mild or moderate, with a safety profile similar to placebo.

On July 8, Pfizer and BioNTech submitted safety and immunogenicity data to the European Medicines Agency (EMA) requesting an update to the Conditional Marketing Authorization (CMA) in the European Union (EU) to include children ages 6 months through 4 years.

The companies plan to submit the updated efficacy data to the FDA, EMA and other regulatory agencies around the world in the coming weeks.

The Pfizer-BioNTech COVID-19 Vaccine, which is based on BioNTech’s proprietary mRNA technology, was developed by both BioNTech and Pfizer. BioNTech is the Marketing Authorization Holder for BNT162b2 (COMIRNATY®) in the United States, the European Union, the United Kingdom, Canada and other countries, and the holder of emergency use authorizations or equivalents in the United States (jointly with Pfizer) and other countries. Submissions to pursue regulatory approvals in those countries where emergency use authorizations or equivalent were initially granted are planned.

About the Phase 1/2/3 Trial in Children

The Phase 1/2/3 trial has enrolled more than 10,000 children ages 6 months to under 12 years of age in the United States, Finland, Poland, Brazil and Spain from more than 90 clinical trial sites.

The trial evaluated the safety, tolerability, and immunogenicity of three doses of the Pfizer-BioNTech COVID-19 Vaccine in three age groups: ages 5 to under 12 years; ages 2 to under 5 years; and ages 6 months to under 2 years.

Based on the Phase 1 dose-escalation portion of the trial, children ages 5 to under 12 years received a two-dose schedule of 10 µg each while children under age 5 received a lower 3 µg dose for each injection in the Phase 2/3 study.

The trial enrolled children with or without prior evidence of SARS-CoV-2 infection.

U.S. Indication & Authorized Use

Pfizer-BioNTech COVID-19 Vaccine is FDA authorized under Emergency Use Authorization (EUA) for active immunization to prevent coronavirus disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) in individuals 6 months of age and older. Pfizer-BioNTech COVID-19 Vaccine is FDA authorized to provide:

Primary Series

  • A 3-dose primary series to individuals 6 months through 4 years of age
  • a 2-dose primary series to individuals 5 years through 11 years of age
  • a 2-dose primary series to individuals 12 years of age and older
  • a third primary series dose to individuals 5 years of age and older with certain kinds of immunocompromise

Booster Series

  • a single booster dose to individuals 5 through 11 years of age who have completed a primary series with Pfizer-BioNTech COVID-19 Vaccine
  • a first booster dose to individuals 12 years of age and older who have completed a primary series with Pfizer-BioNTech COVID-19 Vaccine or COMIRNATY® (COVID-19 Vaccine, mRNA)
  • a first booster dose to individuals 18 years of age and older who have completed primary vaccination with a different authorized or approved COVID-19 vaccine. The booster schedule is based on the labeling information of the vaccine used for the primary series
  • a second booster dose to individuals 50 years of age and older who have received a first booster dose of any authorized or approved COVID-19 vaccine
  • a second booster dose to individuals 12 years of age and older with certain kinds of immunocompromise and who have received a first booster dose of any authorized or approved COVID-19 vaccine

COMIRNATY®INDICATION

COMIRNATY® (COVID-19 Vaccine, mRNA) is a vaccine approved for active immunization to prevent coronavirus disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) in individuals 12 years of age and older.

  • COMIRNATY® is administered as a 2-dose primary series

HOW IS COMIRNATY ® GIVEN?

COMIRNATY® is administered as an injection into the muscle as a 2-dose primary series, 3 weeks apart.

COMIRNATY® AUTHORIZED USES

COMIRNATY® (COVID-19 Vaccine, mRNA) is FDA authorized under Emergency Use Authorization (EUA) to provide:

Primary Series

  • a third primary series dose to individuals 12 years of age and older with certain kinds of immunocompromise

Booster Dose

  • a first booster dose to individuals 12 years of age and older who have completed a primary series with Pfizer-BioNTech COVID-19 Vaccine or COMIRNATY®
  • a first booster dose to individuals 18 years of age and older who have completed primary vaccination with another authorized or approved COVID-19 vaccine. The booster schedule is based on the labeling information of the vaccine used for the primary series
  • a second booster dose to individuals 50 years of age and older who have received a first booster dose of any authorized or approved COVID-19 vaccine
  • a second booster dose to individuals 12 years of age and older with certain kinds of immunocompromise and who have received a first booster dose of any authorized or approved COVID-19 vaccine

Emergency Use Authorization

Emergency uses of the vaccine have not been approved or licensed by FDA, but have been authorized by FDA, under an Emergency Use Authorization (EUA) to prevent Coronavirus Disease 2019 (COVID 19) in individuals 6 months of age and older.

The emergency uses are only authorized for the duration of the declaration that circumstances exist justifying the authorization of emergency use of the medical product under Section 564(b)(1) of the FD&C Act unless the declaration is terminated or authorization revoked sooner.

INTERCHANGEABILITY

FDA-approved COMIRNATY® (COVID-19 Vaccine, mRNA) and the Pfizer-BioNTech COVID-19 Vaccine FDA authorized for Emergency Use Authorization (EUA) for individuals 12 years of age and older can be used interchangeably by a vaccination provider when prepared according to their respective instructions for use.

The formulation of the Pfizer-BioNTech COVID-19 Vaccine authorized for use in individuals 6 months through 4 years of age, 5 through 11 years of age, and 12 years of age and older are different and should therefore not be used interchangeably.

IMPORTANT SAFETY INFORMATION

Tell your vaccination provider about all of the vaccine recipient’s medical conditions, including if the vaccine recipient:

  • has any allergies
  • has had myocarditis (inflammation of the heart muscle) or pericarditis (inflammation of the lining outside the heart)
  • has a fever
  • has a bleeding disorder or is on a blood thinner
  • is immunocompromised or are on a medicine that affects the immune system
  • is pregnant, plan to become pregnant, or are breastfeeding
  • has received another COVID-19 vaccine
  • has ever fainted in association with an injection
  • Pfizer-BioNTech COVID-19 Vaccine or COMIRNATY® (COVID-19 Vaccine, mRNA) may not protect all vaccine recipients
  • The vaccine recipient should not receive Pfizer-BioNTech COVID-19 Vaccine or COMIRNATY® (COVID-19 Vaccine, mRNA) if the vaccine recipient has had a severe allergic reaction to any of its ingredients or had a severe allergic reaction to a previous dose of Pfizer-BioNTech COVID-19 Vaccine or COMIRNATY®
  • There is a remote chance that Pfizer-BioNTech COVID-19 Vaccine or COMIRNATY® (COVID-19 Vaccine, mRNA) could cause a severe allergic reaction.
    A severe allergic reaction would usually occur within a few minutes to 1 hour after getting a dose of the vaccine.
    For this reason, your vaccination provider may ask you to stay at the place where the vaccine was administered for monitoring after vaccination.
    If the vaccine recipient experiences a severe allergic reaction, call 9-1-1 or go to the nearest hospital
  • Seek medical attention right away if the vaccine recipient has any of the following symptoms: difficulty breathing, swelling of the face and throat, a fast heartbeat, a bad rash all over the body, dizziness, and weakness
  • Myocarditis (inflammation of the heart muscle) and pericarditis (inflammation of the lining outside the heart) have occurred in some people who have received the vaccine, more commonly in males under 40 years of age than among females and older males.
    In most of these people, symptoms began within a few days following receipt of the second dose of the vaccine. The chance of having this occur is very low

Seek medical attention right away if the vaccine recipient has any of the following symptoms after receiving the vaccine, particularly during the 2 weeks after receiving a vaccine dose:

  • Chest pain
  • Shortness of breath
  • Feelings of having a fast-beating, fluttering, or pounding heart
  • Fainting
  • Unusual and persistent irritability
  • Unusual and persistent poor feeding
  • Unusual and persistent fatigue or lack of energy
  • Persistent vomiting
  • Persistent pain in the abdomen
  • Unusual and persistent cool, pale skin
  • Fainting can happen after getting injectable vaccines, including Pfizer-BioNTech COVID-19 Vaccine or COMIRNATY® (COVID-19 Vaccine, mRNA). Sometimes people who faint can fall and hurt themselves.
    For this reason, your vaccination provider may ask the vaccine recipient to sit or lie down for 15 minutes after receiving the vaccine
  • Some people with weakened immune systems may have reduced immune responses to Pfizer-BioNTech COVID-19 Vaccine or COMIRNATY® (COVID-19 Vaccine, mRNA)
  • Additional side effects include rash, itching, hives, swelling of the face, injection site pain, tiredness, headache, muscle pain, chills, joint pain, fever, injection site swelling, injection site redness, nausea, feeling unwell, swollen lymph nodes (lymphadenopathy), decreased appetite, diarrhea, vomiting, arm pain, and fainting in association with injection of the vaccine and irritability

These may not be all the possible side effects of the vaccine. Call the vaccination provider or healthcare provider about bothersome side effects or side effects that do not go away.”

https://www.pfizer.com/news/press-release/press-release-detail/pfizer-and-biontech-announce-updated-covid-19-vaccine-data

BrainVectis receives clearance to conduct clinical trial in France for early-stage Huntington’s Disease

admin
-
0
BrainVectis receives clearance to conduct clinical trial in France for early-stage Huntington’s Disease

August 23, 2022: “Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, has received clearance to conduct a Phase I/II trial for its novel Huntington’s Disease (HD) gene therapy, BV-101, in France through its subsidiary BrainVectis.

This authorization, provided by the National Agency for Safety of Medicines and Health Products (ANSM), the country’s governing drug authority, along with the approval of the trial protocol by the Ethics Committee in charge, enables the company to begin recruiting participants.

BV-101 is a novel, exclusively designed adeno-associated virus (AAV) gene therapy vector that simultaneously addresses the metabolic dysfunction of diseased neurons as well as contributes to the clearance of the mutant huntingtin protein.

BV-101 is administered through MRI-guided neurosurgical techniques directed to target tissues in the basal structures of the brain.

In preclinical studies in mice, BV-101 demonstrated the ability to repair the essential cholesterol pathway, provide neuroprotection, and restore physical performance by delivering CYP46A1, a crucial enzyme in the brain which is reduced in people with Huntington’s Disease.

BV-101 was granted orphan drug designation in the European Union in 2019 by the European Medicines Agency.

“Unlike other attempts to treat Huntington’s Disease, BV-101 aims to restore cholesterol metabolism, reduce mutant huntingtin and to improve neuronal function.

Importantly, BV-101 does not affect the levels of normal huntingtin protein in cells,” said Nathalie Cartier-Lacave, MD, founder of BrainVectis and now Vice President, Sector Lead Neurobiology, at AskBio.

“If this proves successful, we have the potential to change the course of a devastating disease that causes severe functional and cognitive decline.”

Currently there are no approved disease modifying therapies for HD, a rare inherited neurodegenerative disease that, based on information from the Committee for Orphan Medicinal Products (COMP), affects approximately 62,000 people in the European Union.

The disease is caused by anomalous repeating mutations in the huntingtin gene leading to abnormal protein aggregates in nerve cells.

This results in a range of progressive symptoms, leading to complete physical and mental deterioration, with symptoms usually beginning in adults ages 30 to 50, but which can also occur at an earlier age.

“The approval of this trial in France marks a major milestone to potentially treat one of the world’s most devastating genetic diseases,” added Sheila Mikhail, JD, MBA, CEO and Co-Founder of AskBio.

“If successful, this novel approach for treating Huntington’s Disease may impact how we treat many other neurodegenerative diseases in the future.”

About the BV-101 Clinical Trial
The BV-101 clinical trial will be an open-label, dose-escalation study to assess the safety, tolerability, and preliminary efficacy of administration of BV-101 in adult subjects with early-stage Huntington’s Disease (HD).

The trial will include 12-18 participants and is expected to begin in Paris in Q4, 2022. The trial will be led by principal investigator, Alexandra Durr, MD, PhD, Professor Genetics, Reference Centre for Rare diseases-Neurogenetics.”

https://media.bayer.com/baynews/baynews.nsf/id/BrainVectis-a-subsidiary-AskBio-receives-clearance-conduct-Phase-I-II-clinical-trial-France-novel?Open&parent=news-overview-category-search-en&ccm=020

AstraZeneca aims to transform cancer outcomes with new data across industry-leading portfolio at ESMO 2022

admin
-
0
AstraZeneca aims to transform cancer outcomes with new data across industry-leading portfolio at ESMO 2022

August 24, 2022: “AstraZeneca will present new data supporting its ambition to redefine cancer care at the European Society for Medical Oncology (ESMO) Congress 2022, 9 to 13 September 2022.

A total of 15 approved and potential new medicines from AstraZeneca will be featured across more than 75 abstracts in 13 tumour types.

Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: “At ESMO this year, new evidence will demonstrate how our medicines are prolonging patient survival across several cancers. Data from the SOLO-1 and PAOLA-1 Phase III trials will reinforce the long-term survival benefits of PARP inhibition with Lynparza in advanced ovarian cancer, and new data for Imfinzi combinations in liver, biliary tract and lung cancers will show the potential to improve outcomes for patients in these areas of high unmet need.”

Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca, said: “The momentum will continue for Enhertu at ESMO with new data across tumour types, including results from the DESTINY-Lung02 Phase II trial in HER2-mutant metastatic non-small cell lung cancer which formed the basis for the recent FDA approval.

Additionally, we’re excited to advance the science of CTLA-4 inhibition with new analyses presented from two Phase III trials of Imfinzi plus tremelimumab, HIMALAYA in liver cancer and POSEIDON in lung cancer, and for MEDI5752, our bispecific antibody targeting both PD-1 and CTLA-4 in lung cancer.”

Transforming outcomes across tumours over time
Mature disease-free survival (DFS) data from the ADAURA Phase III trial will be featured in a late-breaking presentation detailing two years of additional follow-up in patients with early-stage (Stage IB-IIIA) EGFR-mutated non-small cell lung cancer (NSCLC) treated with adjuvant Tagrisso (osimertinib). 

Tagrisso is the only targeted treatment option approved in this setting. The presentation will also report updated results for patterns of recurrence and central nervous system DFS.

A late-breaking presentation will feature landmark five-year overall survival (OS) data from the externally sponsored PAOLA-1 Phase III trial of Lynparza (olaparib) in combination with bevacizumab in 1st-line advanced ovarian cancer, including patients with homologous recombination deficiency (HRD) positive disease.

This is the longest follow up for a PARP inhibitor in combination with standard of care in this setting.

In addition, seven-year OS data from the SOLO1 Phase III trial of Lynparza for 1st-line maintenance therapy in BRCA-mutated (BRCAm) advanced ovarian cancer will be presented. This is the longest follow-up for any PARP inhibitor in newly diagnosed advanced ovarian cancer.

Data will also include updated OS results at two years from the TOPAZ-1 Phase III trial of Imfinzi (durvalumab) plus standard-of-care chemotherapy (gemcitabine plus cisplatin) in 1st-line unresectable or advanced biliary tract cancer, as well as an analysis of immune-mediated adverse events.

TOPAZ-1 is the first Phase III trial to show improved OS with an immunotherapy combination versus chemotherapy alone in this setting.

Extending the benefit of antibody drug conjugates (ADCs) to more patients
Several presentations will demonstrate the clinical potential of Enhertu (trastuzumab deruxtecan) treatment across HER2-targetable lung, gastric and breast cancers.

A late-breaking presentation will feature interim results from the DESTINY-Lung02 Phase II trial investigating Enhertu in patients with HER2-mutant (HER2m) metastatic NSCLC (mNSCLC) who have progressed following one or more systemic therapies. 

Enhertu was recently approved in the US in this setting as the first HER2-directed treatment for these patients. Detailed data will also be shared from the DESTINY-Lung01 Phase II trial, both in this setting and in patients with HER2-overexpressing mNSCLC.

Another presentation will feature updated data from the DESTINY-Gastric02 Phase II trial in HER2-positive metastatic gastric cancer, the first Enhertu trial in Western patients with gastric cancer.

Data will also include a subgroup analysis of the DESTINY-Breast03 Phase III trial of Enhertu by disease history and prior treatments in patients with HER2-positive metastatic breast cancer previously treated with trastuzumab and a taxane.

Patient-reported outcomes from the DESTINY-Breast04 Phase III trial will also highlight quality of life data for patients treated with Enhertu in HER2-low unresectable and/or metastatic breast cancer. 

Enhertu was recently approved in the US as the first HER2-directed therapy for patients with HER2-low metastatic breast cancer based on this trial.

Additional posters will describe trials evaluating the TROP2-directed ADC datopotamab deruxtecan in patients with hormone-receptor positive, HER2-negative breast cancer (TROPION-Breast01 Phase III trial) and in a platform trial in combination with Tagrisso in patients with advanced NSCLC who have experienced disease progression (ORCHARD Phase II trial).

There are currently no approved TROP2-directed therapies for patients in these settings.

Advancing the science of CTLA-4 inhibition
A new analysis from the positive HIMALAYA Phase III trial will show the impact of viral aetiology on outcomes in unresectable liver cancer for patients treated with a single priming dose of tremelimumab, an anti-CTLA-4 antibody, added to Imfinzi (STRIDE regimen).

In addition, a poster will describe the EMERALD-3 Phase III trial evaluating tremelimumab added to Imfinzi and transarterial chemoembolisation with or without lenvatinib in unresectable liver cancer patients eligible for embolisation.

A late-breaking presentation of the positive POSEIDON Phase III trial in mNSCLC will feature four-year OS outcomes in patients treated with a limited course of tremelimumab added to Imfinzi plus chemotherapy.

Another late-breaking presentation will share initial data for MEDI5752 plus chemotherapy in patients with treatment-naïve Stage IIIB-IV non-squamous NSCLC. MEDI5752 is a novel bispecific antibody that simultaneously targets the immune checkpoint proteins PD-1 and CTLA-4.

Bispecific antibodies are a promising approach in immuno-oncology that combines the potential benefits of two medicines into one antibody without the increased toxicity seen with administration of two separate medicines.

Reinforcing the robust benefits of PARP inhibitors across a broad range of tumour types
In addition to data from PAOLA-1 and SOLO1, an oral presentation will share updated efficacy analyses across biomarker subgroups from the PROpel Phase III trial of Lynparza plus abiraterone in patients with newly diagnosed metastatic castration-resistant prostate cancer (mCRPC) treated with the combination with or without homologous recombination repair (HRR) gene mutations. 

Lynparza is the first PARP inhibitor to demonstrate a significant improvement in radiographic progression-free survival in combination with abiraterone versus abiraterone alone in 1st-line mCRPC irrespective of biomarker status.

Additionally, final OS data will be presented from the MEDIOLA Phase II trial of Lynparza and Imfinzi in germline BRCAm platinum-sensitive relapsed ovarian cancer and from the OPINION Phase IIIB trial of Lynparza maintenance monotherapy in patients with platinum-sensitive relapsed ovarian cancer without a germline BRCA1/BRCA2 mutation.

Data will also include an extended OS analysis from the POLO Phase III trial of Lynparza in germline BRCA-mutated metastatic pancreatic cancer, a disease in which no other PARP inhibitor is approved.

Collaboration in the scientific community is critical to improving outcomes for patients.

AstraZeneca is collaborating with Daiichi Sankyo Company Limited to develop and commercialise Enhertu and datopotamab deruxtecan, and with MSD (Merck & Co., Inc. in the US and Canada) to develop and commercialise Lynparza.

Key AstraZeneca presentations during ESMO 2022

Lead authorAbstract titlePresentation details
Antibody drug conjugates
Goto, KTrastuzumab Deruxtecan (T-DXd) in Patients (Pts) With HER2-Mutant Metastatic Non-Small Cell Lung Cancer (NSCLC): Interim Results From the Phase 2 DESTINY-Lung02 TrialPresentation #LBA55Mini Oral Session11 September 202210:15am (CEST)
Ueno, NTPatient-Reported Outcomes (PROs) From DESTINY-Breast04, a Randomized Phase 3 Study of Trastuzumab Deruxtecan (T-DXd) vs Treatment of Physician’s Choice (TPC) in Patients (pts) With HER2-Low Metastatic Breast Cancer (MBC)Presentation #217OProffered Paper Session11 September 20229:30am (CEST)
Ku, GYUpdated Analysis of DESTINY-Gastric02: a Phase 2 Single-Arm Trial of Trastuzumab Deruxtecan (T-DXd) in Western Patients (Pts) With HER2-Positive (HER2+) Unresectable/Metastatic Gastric/Gastroesophageal Junction (GEJ) Cancer Who Progressed on or After Trastuzumab-Containing RegimenPresentation #1205MOMini Oral Session10 September 20223:45pm (CEST)
Cortés, JSubgroup Analysis by Disease History and Prior Treatments of Patients (pts) With HER2-Positive (HER2+) Metastatic Breast Cancer (MBC) From DESTINY-Breast03, a Randomized Phase 3 Study of Trastuzumab Deruxtecan (T-DXd) vs Trastuzumab Emtansine (T-DM1)Presentation #236Pe-Poster10 September 2022
Li, BTPhase 2 Trial of Trastuzumab Deruxtecan (T-DXd) in Patients (Pts) With HER2-Mutated (HER2m) Metastatic Non-Small Cell Lung Cancer (NSCLC): Registrational Data From DESTINY-Lung01Presentation #976Pe-Poster12 September 2022
Bardia, ADatopotamab deruxtecan (Dato-DXd), a TROP2 antibody-drug conjugate, vs investigators’ choice of chemotherapy (ICC) in previously-treated, inoperable or metastatic hormone-receptor (HR) positive, HER2-negative (HR+/HER2–) breast cancer: TROPION-Breast01Presentation #274TiPTrial in Progress10 September 2022
De Langen, JORCHARD platform study: osimertinib + datopotamab deruxtecan (Dato-DXd) cohort in patients (pts) with advanced NSCLC (aNSCLC) who have progressed on first-line (1L) osimertinibPresentation #1188TiPTrial in Progress12 September 2022
Immuno-oncology
Johnson, ML.Durvalumab (D) ± tremelimumab (T) + chemotherapy (CT) in 1L metastatic (m) NSCLC: overall survival (OS) update from POSEIDON after median follow-up (mFU) of approximately 4 years (y)Presentation #LBA59Mini Oral Session11 September 202211:05am (CEST)
Ahn, MJMEDI5752 or pembrolizumab (P) plus carboplatin/pemetrexed (CP) in treatment-naïve (1L) non-small cell lung cancer (NSCLC): a Phase 1b/2 trialPresentation #LBA56Mini Oral Session11 September 202210:20am (CEST)
Spicer, JPlatform study of neoadjuvant durvalumab (D) alone or combined with novel agents in patients (pts) with resectable, early-stage non-small-cell lung cancer (NSCLC): pharmacodynamic correlates and circulating tumor DNA (ctDNA) dynamics in the NeoCOAST studyPresentation #929MOMini Oral Session12 September 20223:15pm (CEST)
Oh, DYUpdated overall survival (OS) from the Phase 3 TOPAZ-1 study of durvalumab (D) or placebo (PBO) plus gemcitabine and cisplatin (+ GC) in patients (pts) with advanced biliary tract cancer (BTC)Presentation #56Pe-Poster12 September 2022
Antonuzzo, LImmune-mediated adverse event (imAE) incidence, timing and association with efficacy in the Phase 3 TOPAZ-1 study of durvalumab (D) or placebo (PBO) plus gemcitabine and cisplatin (+ GC) in advanced biliary tract cancer (BTC)Presentation #57Pe-Poster12 September 2022
Chan, LSImpact of viral aetiology in the Phase 3 HIMALAYA study of tremelimumab (T) plus durvalumab (D) in unresectable hepatocellular carcinoma (uHCC)Presentation #714Pe-Poster12 September 2022
Özgüroğlu, MPhase 3 trial of durvalumab combined with domvanalimab following concurrent chemoradiotherapy (cCRT) in patients with unresectable stage III NSCLC (PACIFIC-8)Presentation #971TiPTrial in Progress10 September 2022
Abou-Alfa, GKA randomised Phase 3 study of tremelimumab (T) plus durvalumab (D) with or without lenvatinib combined with concurrent transarterial chemoembolisation (TACE) versus TACE alone in patients (pts) with locoregional hepatocellular carcinoma (HCC): EMERALD-3Presentation #727TiPTrial in Progress12 September 2022
DNA damage response
Ray-Coquard, ILFinal overall survival (OS) results from the Phase III PAOLA-1/ENGOT-ov25 trial evaluating maintenance olaparib (ola) plus bevacizumab (bev) in patients (pts) with newly diagnosed advanced ovarian cancer (AOC)Presentation #LBA29Proffered Paper Session9 September 20222:00pm (CEST)
DiSilvestro, POverall survival (OS) at 7-year (y) follow-up (f/u) in patients (pts) with newly diagnosed advanced ovarian cancer (OC) and a BRCA mutation (BRCAm) who received maintenance olaparib in the SOLO1/GOG-3004 trialPresentation #517OProffered Paper Session9 September 20222:10pm (CEST)
Guo, CA Phase (Ph) I/II trial of the CXCR2 antagonist AZD5069 in combination with enzalutamide (ENZA) in patients (pts) with metastatic castration resistant prostate cancer (mCRPC)Presentation #454OProffered Paper Session10 September 202211:15am (CEST)
Saad, FBiomarker analysis and updated results from the Phase III PROpel trial of abiraterone (abi) and olaparib (ola) vs abi and placebo (pbo) as first-line (1L) therapy for patients (pts) with metastatic castration-resistant prostate cancer (mCRPC)Presentation #1357OProffered Paper Session11 September 20229:30am (CEST)
Banerjee, SPhase II study of olaparib plus durvalumab with or without bevacizumab (MEDIOLA): final analysis of overall survival in patients with non-germline BRCA-mutated platinum-sensitive relapsed ovarian cancerPresentation #529MOMini Oral Session11 September 20225:00pm (CEST)
Poveda Velasco, AMMaintenance olaparib monotherapy in patients (pts) with platinum-sensitive relapsed ovarian cancer (PSR OC) without a germline BRCA1/BRCA2 mutation (non-gBRCAm): final overall survival (OS) results from the OPINION trialPresentation #531Pe-Poster11 September 2022
Hammel, PExtended overall survival results from the POLO study of active maintenance olaparib in patients with metastatic pancreatic cancer and a germline BRCA mutationPresentation #1298Pe-Poster12 September 2022
Tumour drivers and resistance
Tsuboi, MOsimertinib as adjuvant therapy in patients (pts) with resected EGFR-mutated (EGFRm) stage IB–IIIA non-small cell lung cancer (NSCLC): updated results from ADAURAPresentation #LBA47Proffered Paper Session11 September 20228:30am (CEST)
Piotrowska, ZELIOS: A multicentre, molecular profiling study of patients (pts) with epidermal growth factor receptor-mutated (EGFRm) advanced NSCLC treated with first-line (1L) osimertinibPresentation #LBA53Proffered Paper Session11 September 20223:05pm (CEST)
Nakamura, AFinal results and biomarker analysis of a randomized phase II study of osimertinib plus bevacizumab versus osimertinib monotherapy for untreated patients with non-squamous non-small-cell lung cancer harboring EGFR mutations; WJOG9717L studyPresentation #982Pe-Poster12 September 2022

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2022/astrazeneca-aims-to-transform-cancer-outcomes-with-new-data-across-industry-leading-portfolio-at-esmo-2022.html

BMS Completes Acquisition of Turning Point Therapeutics, Expanding Precision Oncology Portfolio

admin
-
0
BMS Completes Acquisition of Turning Point Therapeutics, Expanding Precision Oncology Portfolio

AUG 17, 2022: “Bristol Myers Squibb  announced that it has successfully completed its acquisition of Turning Point Therapeutics, Inc. (“Turning Point”), in an all-cash transaction.

With the completion of the acquisition, Turning Point shares have ceased trading on the NASDAQ Global Select Market and Turning Point is now a wholly owned subsidiary of Bristol Myers Squibb.

“Turning Point has distinguished itself in the field of precision oncology, and this acquisition will further strengthen our leading oncology franchise,” said Elizabeth Mily, Executive Vice President, Strategy & Business Development, Bristol Myers Squibb.

“With Turning Point’s lead asset, repotrectinib, Bristol Myers Squibb will be positioned to address a significant unmet medical need for ROS1-positive non-small cell lung cancer patients.

We look forward to bringing this promising, innovative medicine to patients in the second half of 2023.”

Through the transaction, Bristol Myers Squibb gains a pipeline of investigational medicines designed to target the most common mutations associated with oncogenesis, including repotrectinib.

Repotrectinib is a next-generation, potential best-in-class tyrosine kinase inhibitor targeting the ROS1 and NTRK oncogenic drivers of non-small cell lung cancer (NSCLC) and other advanced solid tumors.

In the Phase 1/2 TRIDENT-1 clinical trial, longer duration of response has been observed in the landmark analysis with repotrectinib than with existing ROS1 agents in first-line NSCLC.

The asset has been granted three Breakthrough Therapy Designations from the U.S. Food and Drug Administration.

Bristol Myers Squibb expects repotrectinib to be approved in the U.S. in the second half of 2023 and become a new standard of care for patients with ROS1-positive NSCLC in the first-line setting.

Bristol Myers Squibb also plans to continue to explore the potential of Turning Point’s promising pipeline of novel compounds.

Bristol Myers Squibb’s previously announced tender offer for all outstanding shares of common stock of Turning Point for $76.00 per share expired at 5:00 p.m. Eastern Time on August 15, 2022.

Approximately 41,896,678 shares of Turning Point common stock were validly tendered, and not validly withdrawn from the tender offer, representing approximately 84% of Turning Point’s issued and outstanding shares of common stock.

In accordance with the terms of the tender offer, all shares that were validly tendered and not validly withdrawn have been accepted for payment and Bristol Myers Squibb expects to promptly pay for all such shares.

Following completion of the tender offer, Bristol Myers Squibb completed the acquisition of Turning Point through the merger of its wholly owned subsidiary, Rhumba Merger Sub Inc., with and into Turning Point, without a vote of Turning Point’s stockholders pursuant to Section 251(h) of the General Corporation Law of the State of Delaware.

As a result of the merger, each share of common stock of Turning Point issued and outstanding and not tendered in the tender offer was automatically converted into the right to receive an amount in cash equal to $76.00, without interest, subject to any applicable withholding of taxes, the same price offered in the tender offer.

Turning Point shareholders can direct questions regarding the tender offer to MacKenzie Partners, Inc., the information agent for the tender offer, toll free, at 1-800-322-2885.”

https://investors.bms.com/iframes/press-releases/press-release-details/2022/Bristol-Myers-Squibb-Completes-Acquisition-of-Turning-Point-Therapeutics-Expanding-Precision-Oncology-Portfolio/default.aspx

Mirena® from Bayer approved in the US for extended duration of use in contraception

admin
-
0
Mirena® from Bayer approved in the US for extended duration of use in contraception

August 18, 2022: “Bayer, a leader in women’s healthcare, announced today that the supplemental New Drug Application (sNDA) for the extended use up to eight years in the indication, contraception was approved for its levonorgestrel (LNG) releasing intrauterine system (IUS) Mirena® by the U.S.FDA.

This approval is based on the results of the Mirena Extension Trial evaluating the efficacy and safety of Mirena which demonstrated that contraceptive efficacy during years six to eight remains high.

“This label extension underlines Bayer’s continued commitment to drive innovation in the area of women’s healthcare and to offer women a broad variety of contraceptive methods during their reproductive life”, said Dr. Michael Devoy, Chief Medical Officer at Bayer AG.

“As every woman is different, the personal decision for a specific contraceptive option is taken on the individual needs of a woman in close consultation with her healthcare professional.”

The duration of use for the second approved indication in the US (heavy menstrual bleeding for women who choose to use intrauterine contraception as their method of contraception) remains unchanged for up to five years.

At the beginning of 2022, Bayer also submitted the eight-year label extension for the indication contraception in the EU for the LNG-IUS Mirena.

LNG-IUS is one of the most effective birth control methods as it does not require user interventions such as daily dosing or monthly re-fills.

It can be removed by the doctor at any time and offers a rapid return to a woman’s natural level of fertility after removal. An LNG-IUS should only be inserted after a comprehensive discussion and evaluation of all contraceptive options and the individual woman’s needs.

About the Contraceptive Efficacy & Mirena Extension Trial
The contraceptive efficacy of Mirena for up to five years has been studied in five large clinical trials involving 3,330 women.

The Pearl Index was about 0.2 in the first year and cumulatively, contraception failed by about 0.7% over five years.

The contraceptive effectiveness of Mirena after more than five years (including years six to eight) was studied in the Mirena Extension Trial.

Based on 2 pregnancies (1 in Year 6 and 1 in Year 7) and 10,216 exposure cycles, the cumulative pregnancy rate at the end of the 3-year period of extended use (Years 6, 7 and 8) was 0.68% with a 95% upper confidence limit of 2.71%.

The study confirmed a positive benefit risk profile with no new or unexpected safety findings. The eight-year data from the extension trial were published in abstract form at the American College of Obstetricians and Gynecologists (ACOG) in 2022.

The full paper will be published in due course. Information about this trial can be found at https://clinicaltrials.gov/ct2/show/study/NCT02985541.

About Mirena®
Mirena is a T-shaped device containing 52 mg of synthetic levonorgestrel. Once placed in the uterus, it continuously releases small amounts of levonorgestrel directly into the uterus. Mirena is available in many countries over all continents.

The approved indications in the US are contraception (prevention of pregnancy) for up to eight years of use and treatment of heavy menstrual bleeding for women who choose to use intrauterine contraception as their method of contraception for up to five years.

Mirena is a long-acting reversible contraceptive (LARC) and can be removed at any time by a healthcare provider if a woman’s plans change.

About Women’s Healthcare at Bayer
Bayer is a recognized leader in the area of women’s healthcare, with a long-standing commitment to delivering science for a better life by advancing a portfolio of innovative treatments. Bayer offers a wide range of effective short- and long-acting birth control methods as well as therapies for menopause management and gynecological diseases. Bayer is also focusing on innovative options to address the unmet medical needs of women worldwide.

Today, Bayer’s research and development efforts focus on finding new treatment options for menopause as well as gynecological diseases and includes several compounds in various stages of pre-clinical and clinical development.

Together, these projects reflect the company’s approach to research, which prioritizes targets and pathways with the potential to alter the way that gynecological diseases are treated.

Additionally, Bayer intends to provide 100 million women in low-and-middle income countries by 2030 with access to family planning by funding multi-stakeholder aid programs and by ensuring the supply of affordable modern contraceptives.

This is part of the comprehensive sustainability measures and commitments from 2020 onwards and in line with the Sustainable Development Goals of the United Nations.”

https://media.bayer.com/baynews/baynews.nsf/id/Mirena-from-Bayer-approved-in-the-US-for-extended-duration-of-use-in-contraception?OpenDocument&sessionID=1661144809

1...111213...156Page 12 of 156

EDITOR PICKS

POPULAR POSTS

POPULAR CATEGORY

ABOUT US

LifeProNow provides guidance and high quality contents to help job seekers to get their dream Job. All the contents are written by experienced folks and peer reviewed by experts.

Contact us: [email protected]

FOLLOW US

© LifeProNow | All Rights Reserved