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FDA Issues Safety Alert for Squamous Cell Carcinoma and Various Lymphomas in Scar Tissue around Breast Implants

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FDA Issues Safety Alert for Squamous Cell Carcinoma and Various Lymphomas in Scar Tissue around Breast Implants

September 08, 2022: “The U.S. Food and Drug Administration issued a safety communication informing patients and providers about reports of squamous cell carcinoma (SCC) and various lymphomas located in the capsule or scar tissue around breast implants.

After an initial extensive review, we currently believe that the risk of SCC and other lymphomas occurring in the tissue around breast implants is rare.

However, in this case, and when safety risks with medical devices are identified, we wanted to provide clear and understandable information to the public as quickly as possible. 

In some reported cases, patients were diagnosed years after having breast implants and presented with findings such as swelling, pain, lumps or skin changes.

These emerging reports of lymphoma in scar tissue are different from Breast Implant Associated Anaplastic Large Cell Lymphoma (BIA-ALCL), which the FDA began communicating about as a potential risk more than a decade ago.   

The FDA’s work in the area of patient-centered risk communication for these devices has accelerated in recent years, including convening stakeholders to share perspectives that have informed the FDA’s regulatory oversight and implementation of new requirements for manufacturers.

We continue to engage top cancer experts and are consulting with our Oncology Center of Excellence to ensure a coordinated approach informed by leaders in the field.

Additionally, the agency continues to closely monitor various data sources, such as the scientific literature, adverse event reports submitted to the agency and is soliciting information from manufacturers regarding any reports they may have regarding SCC and other lymphomas related to the tissue around an implant. 

We know that breast implants are not lifetime devices, and that the longer a patient has breast implants, the more likely they will need to be removed or replaced.

We also understand that information regarding breast implant risks can be overwhelming for a patient.

For this reason, we encourage review of our website with attention to patient labeling, which has easy to understand information in the patient brochure. 

Right now, we do not have enough information to say whether breast implants cause these cancers or if some implants pose higher risk than others. For this reason, instances of SCC, lymphoma and any cancer located in the scar tissue around breast implants should be reported to the FDA.

Our collective understanding has advanced significantly because of the efforts to study, communicate and act when needed.

As the agency moves further into adopting modernized approaches to our regulatory responsibilities to promote faster science-based decision-making, accurate data is crucial. 

If a patient with breast implants is experiencing a problem, or there is a case of SCC, lymphoma or any other cancer of the breast implant capsule identified, the FDA strongly encourages reporting this through MedWatch, the FDA Safety Information and Adverse Event Reporting program.

Reporting strengthens our ability to work with manufacturers and others to improve safety.

Today’s safety communication underscores our commitment to sharing the information that we regularly gather and analyze so that patients may fully consider and thoughtfully discuss implant risks with their doctors.

We will continue to collaborate with other regulatory authorities, clinical and scientific experts, breast implant registries and patients as a part of our commitment to educate and enhance evidence generation on these potential new risks.

Looking ahead, the FDA will soon complete a thorough literature review and continue our partnershipExternal Link Disclaimer with the American Society of Plastic Surgeons as we work to identify ways to collect more detailed information regarding patient cases where cancer in the breast implant capsule has been reported.

As we learn more about these cases, we hope to better understand the patient risk and communicate findings to the public.  

The safety signal issued today is an emerging issue that will require steadfast, ongoing evaluation and communication with patients, healthcare providers and manufacturers.

We remain committed to informing the public of important and emerging medical device safety risks and appropriately take action on behalf of patients and public health. “

https://www.fda.gov/news-events/press-announcements/fda-issues-safety-alert-squamous-cell-carcinoma-and-various-lymphomas-scar-tissue-around-breast

Novartis Kisqali® adds one more year of survival benefit for broadest set of patients

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Novartis Kisqali® adds one more year of survival benefit for broadest set of patients

September 9, 2022: “Novartis today announced results from a new pooled exploratory analysis across the entire MONALEESA Phase III program, confirming nearly one year of additional overall survival (OS) benefit in a subgroup of patients with aggressive forms of hormone receptor-positive, human epidermal growth factor receptor-2 negative (HR+/HER2-) advanced breast cancer (aBC).

This subgroup analysis found that patients with visceral metastases—including liver metastases and multiple metastatic sites, which are typically associated with a poor prognosis—who were treated with Kisqali® (ribociclib) plus endocrine therapy in the first-line setting, achieved a median OS of 62.7 months compared to 52.1 months for those treated with endocrine therapy alone (HR=0.79; 95% CI: 0.65-0.97).

Data from this analysis will be presented at the European Society of Medical Oncology (ESMO) Congress in Paris, France.

“Patients who have visceral metastases typically have a worse prognosis and often demonstrate resistance to treatment, so as a clinician it is encouraging to see significant survival benefit with ribociclib in the first-line setting in patients with more aggressive disease,” said Denise A. Yardley, MD, Senior Investigator, Breast Cancer Research Program, Sarah Cannon Research Institute at Tennessee Oncology, USA.

“Ribociclib is the only CDK4/6 inhibitor to show a consistent overall survival benefit in combination with endocrine therapy, while also maintaining quality of life across the Phase III program.”

Those with liver metastases on Kisqali plus endocrine therapy in the first-line achieved 44.2 months median OS compared to 38.1 months for those on endocrine therapy alone (HR=0.77; 95% CI: 0.55-1.07).

For patients with visceral metastases in three or more organs, first-line
treatment with Kisqali-endocrine therapy achieved 57.7 months median OS compared to 49.3 months for those on endocrine therapy alone (HR=0.81; 95% CI: 0.63-1.03).

“The goal for advanced breast cancer treatment is to help people live longer, and we are proud that Kisqali continues to deliver a significant survival benefit while also maintaining quality of life, even for those with harder-to-treat disease,” said Jeff Legos, Executive Vice President, Global Head of Oncology and Hematology at Novartis.

“We are committed to demonstrating what makes Kisqali a unique CDK4/6 inhibitor, thus providing patients and oncologists confidence in this therapeutic option.”

HARMONIA head-to-head CDK4/6 inhibitor trial design
Also at ESMO, the trial design will be presented for HARMONIA, the first prospective, head-to-head Phase III trial of CDK4/6 inhibitors being conducted in collaboration with SOLTI Innovative Cancer Research, to evaluate Kisqali vs. Ibrance®* (palbociclib) for patients with advanced HR+/HER2-, HER2-enriched subtype, ultimately exploring what makes Kisqali unique at a molecular level.

HARMONIA seeks to test if Kisqali improves the course of HR+/HER2- aBC by changing tumor biology to enable a better response to endocrine therapy as compared to Ibrance*, and could further substantiate differences seen among these CDK4/6 inhibitors.

HER2-enriched is an intrinsic subtype associated with a very poor prognosis and endocrine-resistance, as compared to luminal disease.

The global, multicenter, randomized, open-label, Phase III study has a primary outcome of progression-free survival (PFS), and secondary outcomes include OS and PFS2. HARMONIA is currently ongoing with an anticipated enrollment of 456 patients.

About Kisqali® (ribociclib)
Kisqali is the only CDK4/6 inhibitor with proven overall survival benefit across all its three pivotal Phase III advanced breast cancer trials, and is recognized by the National Comprehensive Cancer Network (NCCN) guidelines as the only CDK4/6 inhibitor with overall survival benefit in first-line HR+/HER2- advanced breast cancer.

Additionally, Kisqali has the highest rating of any CDK4/6 inhibitor on the ESMO Magnitude of Clinical Benefit Scale, achieving a score of five out of five for first-line premenopausal patients with HR+/HER2- advanced breast cancer.

Further, Kisqali in combination with either letrozole or fulvestrant has uniquely, among other CDK4/6i, received a score of four out of five for postmenopausal patients with HR+/HER2- advanced breast cancer treated in the first line16.

Kisqali has been approved in more than 95 countries worldwide, including by the United States Food and Drug Administration (FDA) and the European Commission, for the treatment of women with HR+/HER2- advanced or metastatic breast cancer in combination either with an aromatase inhibitor or with fulvestrant as initial endocrine-based therapy or following disease progression on endocrine therapy.

Kisqali in combination with fulvestrant is approved as initial endocrine-based therapy or following disease progression on endocrine therapy in men by the FDA.

Novartis is committed to continuing to study Kisqali in breast cancer.

NATALEE is a large Phase III clinical trial of Kisqali with endocrine therapy in the adjuvant treatment of HR+/HER2- early breast cancer being conducted in collaboration with Translational Research In Oncology (TRIO).

Additionally, Novartis is collaborating with the Akershus University Hospital in Norway on the NEOLETRIB trial, a neoadjuvant Phase II trial studying the effects of Kisqali in HR+/HER2- early breast cancer and to discover the potentially unique underlying mechanism of action.

Kisqali was developed by the Novartis Institutes for BioMedical Research (NIBR) under a research collaboration with Astex Pharmaceuticals.”

https://www.novartis.com/news/media-releases/novartis-kisqali-adds-one-more-year-survival-benefit-broadest-set-patients-including-those-aggressive-hrher2-advanced-breast-cancer

Pfizer Invites Public to View and Listen to Webcast of Pfizer Discussion at Healthcare Conference

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Pfizer Invites Public to View and Listen to Webcast of Pfizer Discussion at Healthcare Conference

September 06, 2022: “Pfizer Inc. invites investors and the general public to view and listen to a webcast of a discussion with William Pao, Chief Development Officer, Executive Vice President, and Aamir Malik, Chief Business Innovation Officer, Executive Vice President, at the Morgan Stanley 20th Annual Global Healthcare Conference on Monday, September 12, 2022 at 1:40 p.m. EDT.

To view and listen to the webcast, visit our web site at www.pfizer.com/investors. Information on accessing and registering for the webcast will be available at www.pfizer.com/investors beginning today.

The transcript and webcast replay of the discussion will be made available on our web site at www.pfizer.com/investors within 24 hours after the end of the live discussion and will be accessible for at least 90 days.

About Pfizer: Breakthroughs That Change Patients’ Lives
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives.

We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines.

Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time.

Consistent with our responsibility as one of the world’s premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us.”

https://www.pfizer.com/news/press-release/press-release-detail/pfizer-invites-public-view-and-listen-webcast-pfizer-11

New Dupixent data at EADV 2022 adds to body of evidence across multiple inflammatory skin diseases

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New Dupixent data at EADV 2022 adds to body of evidence across multiple inflammatory skin diseases

 September 6, 2022: “Twenty-five scientific abstracts evaluating Dupixent® (dupilumab) in moderate-to-severe atopic dermatitis from infancy to adulthood, prurigo nodularis and chronic spontaneous urticaria will be presented at the European Academy of Dermatology and Venereology (EADV) 2022 Congress from September 7 to 10.

The data add to a growing body of clinical and real-world evidence illustrating the potential benefit of Dupixent in targeting IL-4 and IL-13, key and central drivers of the type 2 inflammation that plays a role in these inflammatory skin diseases.

New analyses from the pivotal trial in children aged 6 months to 5 years with moderate-to-severe atopic dermatitis will be shared across eight posters.

Data presentations will show dupilumab plus low-potency topical corticosteroids significantly improved quality of life and sleep quality for both children and their caregivers.

Presentations will also show that children aged 6 months to 5 years treated with dupilumab experienced improvement in skin pain and that dupilumab treatment was associated with lower overall infections and non-herpetic skin infections in this age group.

The safety results of this pivotal trial were generally consistent with the known safety profile of dupilumab in atopic dermatitis.

Data in patients with moderate-to-severe atopic dermatitis aged 6 years and older will also be presented. Presentations of analyses from an open-label trial evaluating skin barrier function (BALISTAD) in patients 12 years and older will show Dupixent normalized skin barrier function for many patients and was associated with improvements in disease signs and symptoms and quality of life.

In a separate Phase 4 randomized, placebo-controlled trial in adults assessing the effect of Dupixent on sleep (DUPISTAD), analyses will be presented showing that Dupixent treatment improved sleep quality, daytime sleepiness, symptoms of anxiety and depression, health-related quality of life measures and itch, including in patients who switched to Dupixent from placebo.

The safety results of this pivotal trial were generally consistent with the known safety profile of Dupixent in atopic dermatitis.

Late-breaking results from a Phase 3 dupilumab trial showing improvement in signs and symptoms in adults with prurigo nodularis will also be presented.

The potential uses of dupilumab in prurigo nodularis, chronic spontaneous urticaria and bullous pemphigoid are currently under clinical development, and the safety and efficacy have not been fully evaluated by any regulatory authority.

Data to be presented at the EADV 2022 Congress

Investigation of Dupilumab in Prurigo Nodularis

  • Late-breaking oral presentation (4:45-5:00 pm CEST):
    • #3583: Dupilumab Significantly Improves Itch and Skin Lesions in Patients with Prurigo Nodularis: Results from a 2nd Phase 3 Trial (LIBERTY-PN PRIME), Gil Yosipovitch

Investigation of Dupilumab in Chronic Spontaneous Urticaria

  • Poster #P1754: Dupilumab Significantly Reduces Itch and Hives in Patients With Chronic Spontaneous Urticaria Irrespective of Baseline IgE Level: Results From a Phase 3 Trial (LIBERTY-CSU CUPID Study A), Marcus Maurer

Study Design for Dupilumab in Bullous Pemphigoid

  • Poster #P0404: The Study Design of a Trial of Dupilumab in Adult Patients With Bullous Pemphigoid: LIBERTY-BP ADEPT, Dedee Murrell

Clinical Efficacy and Safety of Dupilumab in Infants and Young Children with Atopic Dermatitis

  • Poster #P0322: Dupilumab Treatment Improves Health-Related Quality of Life in Children Aged 6 Months to 5 Years with Moderate-to-Severe Atopic Dermatitis and Their Caregivers, Amy Paller
  • Poster #P0321: Dupilumab Treatment Improves Sleep Quality in Children Aged 6 Months to 5 Years With Moderate-to-Severe Atopic Dermatitis and Their Caregivers, Amy Paller
  • Poster #P0297: Dupilumab Treatment Is Not Associated With an Increased Overall Risk of Infections in Patients Aged 6 Months to 5 Years With Moderate-to-Severe Atopic Dermatitis, Michael Cork
  • Poster #P0320: Dupilumab Treatment Reduces Skin Pain in Infants and Young Children With Moderate-to-Severe Atopic Dermatitis Aged 6 Months to 5 Years, Amy Paller
  • Poster #P0290: Dupilumab Treatment Shows Rapid and Consistent Improvement in Atopic Dermatitis in All Anatomical Regions in Patients Aged 6 Months to 5 Years, Andreas Wollenberg
  • Poster #P0356: Laboratory Safety From a 16-Week Phase 3 Study of Dupilumab in Patients Aged 6 Months to 5 Years With Moderate-to-Severe Atopic Dermatitis, Amy Paller
  • Poster #P0354: Clinically Meaningful Within-Patient Change Threshold for the Children’s Dermatology Life Quality Index and Infants’ Dermatitis Quality of Life Index Instruments in Patients Aged 6 Months to <6 Years With Atopic Dermatitis, Amy Paller
  • Poster #P0199 Meaningful Change Threshold for the Dermatitis Family Impact (DFI) Questionnaire for Parents/Caregivers of Children Aged 6 Months to <6 Years With Atopic Dermatitis (AD), Amy Paller

BALISTAD Skin Barrier Trial of Dupilumab in Atopic Dermatitis

  • Poster #P0319: Dupilumab Treatment Normalizes Skin Barrier Structure and Function and Improves Quality of Life in Adult and Adolescent Patients With Moderate-to-Severe Atopic Dermatitis, Robert Bissonnette
  • Poster #P0317: Dupilumab Treatment Normalizes Skin Barrier Function and Improves Clinical Outcomes in Patients With Atopic Dermatitis, Robert Bissonnette

DUPISTAD Sleep Trial of Dupilumab in Atopic Dermatitis

  • Poster #P0328: Dupilumab ameliorates sleep disturbance and relieves itch in adults with moderate-to-severe atopic dermatitis over 24 weeks, Joseph Merola
  • Poster #P0331: Improvement in sleep quality anxiety and depression in adults with moderate-to-severe atopic dermatitis with dupilumab treatment, Joseph Merola
  • Poster #P0332: Improvement in symptoms of atopic dermatitis (AD) and AD-related quality of life with dupilumab treatment in adults: 24-week results of the DUPISTAD study, Joseph Merola

Clinical and Real-World Efficacy and Safety of Dupilumab in Atopic Dermatitis

  • Oral Presentation (September 8, 10:45-10:55 am CEST):
    • #FC02.04: Real-world treatment outcomes for up to 2 years in patients aged less than 12 years with inadequately controlled moderate-to-severe atopic dermatitis, Amy Paller
  • Poster #P0323: Dupilumab 16-Week Efficacy and Safety Is Robust and Consistent in Adults Over 60 Years of Age With Moderate-to-Severe Atopic Dermatitis, Johnathan Silverberg
  • Poster #P0312: Dupilumab Treatment Leads to Rapid, Progressive, and Sustained Reduction in Lichenification in Children, Adolescents, and Adults With Atopic Dermatitis, Emma Guttman-Yassky
  • Poster #P0313: Dupilumab Treatment Leads to Rapid, Progressive and Sustained Reduction in Lichenification in White, African American/Black and Asian Patients With Atopic Dermatitis, Emma Guttman-Yassky.”

https://www.sanofi.com/en/media-room/press-releases/2022/2022-09-06-05-00-00-2509982

Forxiga approved in China for the treatment of chronic kidney disease

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Forxiga approved in China for the treatment of chronic kidney disease

September 05, 2022: “AstraZeneca’s Forxiga (dapagliflozin), a sodium-glucose cotransporter 2 (SGLT2) inhibitor, has been approved in China to reduce the risk of sustained estimated glomerular filtration rate (eGFR) decline, end-stage kidney disease (ESKD), cardiovascular (CV) death and hospitalisation for heart failure (hHF) in adults with chronic kidney disease (CKD) at risk of progression with and without type-2 diabetes (T2D).

The approval by China’s National Medical Products Administration (NMPA) is based on positive results from the DAPA-CKD Phase III trial.

CKD is a serious, progressive condition defined by decreased kidney function and is often associated with an increased risk of heart disease or stroke.

The condition affects 850 million people worldwide. However, diagnosis rates remain low and up to 90% of patients are unaware they have the disease.

Member of the DAPA-CKD Executive Committee, Fan Fan Hou, Southern Medical University, Guangzhou, China, said: “With unprecedented results of DAPA-CKD, dapagliflozin becomes the first SGLT2 inhibitor approved in China for the treatment of chronic kidney disease.

This transformational milestone brings great hope to the 120 million subjects suffering from chronic kidney disease in China.”

Sir Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca said: “In addition to AstraZeneca’s commitment to drive increased awareness, prevention and earlier diagnoses, this approval marks another important step forward in our ambition to stop, reverse and ultimately cure chronic kidney disease globally.

We are thrilled at the opportunity to bring Forxiga to millions of patients across the country and improve patient outcomes.”

The DAPA-CKD Phase III trial demonstrated that Forxiga, on top of standard-of-care (SoC) treatment with an angiotensin-converting enzyme inhibitor or an angiotensin receptor blocker, reduced the relative risk of worsening of renal function, onset of ESKD, or risk of CV or renal death by 39%, the primary composite endpoint, compared to placebo (absolute risk reduction [ARR]=5.3%, p<0.0001) in patients with CKD Stages 2-4 and elevated urinary albumin excretion. Forxiga also significantly reduced the relative risk of death from any cause by 31% (ARR=2.1%, p=0.0035) compared to placebo.

The safety and tolerability of Forxiga were consistent with the well-established safety profile of the medicine.

Forxiga (known as Farxiga in the US) is now approved in 100 countries around the world including the US, the European Union and Japan for the treatment of CKD in adults with and without T2D.

Notes

CKD
CKD is a serious, progressive condition defined by decreased kidney function (shown by reduced estimated glomerular filtration rate (eGFR) or markers of kidney damage, or both, for at least three months). 

The most common causes of CKD are diabetes, hypertension and glomerulonephritis. CKD is associated with significant patient morbidity and an increased risk of CV events, such as heart failure (HF) and premature death.

In its most severe form, known as ESKD, kidney damage and deterioration of kidney function have progressed to the point where dialysis or kidney transplantation are required. 

The majority of patients with CKD will die from CV causes before reaching ESKD. Currently in China, up to 120 million people are living with CKD.

DAPA-CKD
DAPA-CKD was an international, multi-centre, randomised, double-blinded Phase III trial in 4,304 patients designed to evaluate the efficacy of Forxiga 10mg, compared with placebo, in patients with CKD Stage 2-4 and elevated urinary albumin excretion, with and without T2D. 

Forxiga was given once daily in addition to SoC. The primary composite endpoint was worsening of renal function or risk of death (defined as a composite of an eGFR decline ≥50%, onset of ESKD or death from CV or renal cause).

The secondary endpoints included the time to first occurrence of the renal composite (sustained ≥50% eGFR decline, ESKD or renal death), the composite of CV death or hospitalisation for HF (hHF), and death from any cause.

The trial was conducted in 21 countries.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2022/forxiga-approved-in-china-for-ckd.html

Manus Bio receives additional funding to fight malaria

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Manus Bio receives additional funding to fight malaria

August 31, 2022: “Manus Bio, the leading biomanufacturer of natural products, has received a fourth award from the Bill & Melinda Gates Foundation to develop a scalable production route for the potential antimalarial drug, artemisinin. 

The $2 million award will enable Manus Bio to begin scaling up the unique biological process it has developed towards the key chemical intermediate, dihydroartemisinic acid.

Economical and scalable access to this compound will enable process simplification for large-scale, commercial manufacturing of artemisinin.

“We are grateful for the longstanding support from the Bill & Melinda Gates Foundation on developing a sustainable and low-cost manufacturing route for artemisinin,” says Dr. Christine Santos, Chief Technology Officer at Manus Bio.

“Ready access to this life-saving drug is such an important tool in the global fight against malaria.

The additional funding we have received will enable us to translate a robust technology built with our BioAssemblyLineTM cell factory engineering platform into a fully scaled process.”

A key time to beat malaria…

Malaria remains one of the world’s biggest public health issues, with rising numbers of both cases and deaths globally in the last couple years, thought to be attributable to disruption from the COVID-19 pandemic.

However, it is a preventable and curable disease with sufficient access to key antimalarial medicines.

Artemisinin is an effective compound in malaria treatments. The World Health Organization (WHO) recommends artemisinin or one of its derivatives formulated in combination therapies as frontline treatments for all cases of malaria.

It has traditionally been extracted from the Artemisia annua plant – or “sweet wormwood”. However, availability of the plant is subject to agricultural instabilities and vulnerabilities.

The artemisinin precursor, dihydroartemisinic acid, can be produced using fermentation, which provides a more stable and sustainable source for making artemisinin than through agricultural extraction.

…using robust biotechnology-based solutions

Manus Bio has already made significant progress in applying its pioneering BioAssemblyLine™ cell engineering platform to develop a fermentation-based approach for artemisinin production through the support of the foundation.

“Our aim is to complete the development process within a significantly accelerated timeframe by leveraging our experience, know-how, and track record in scaling next-generation biotechnology solutions,” adds Dr. Santos.

https://www.manusbio.com/manus-bio-receives-additional-funding-to-fight-malaria-through-biotechnology/

Avacta group announces Phase I clinical study of AVA6000

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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

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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

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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

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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

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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

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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

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