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Patient enrollment of phase III tolebrutinib trials paused in the U.S.

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Patient enrollment of phase III tolebrutinib trials paused in the U.S.

 June 30, 2022: “The U.S.FDA has placed Phase 3 studies of tolebrutinib in multiple sclerosis (MS) and myasthenia gravis on partial clinical hold.

As a result, new enrollment in the United States (U.S.) is paused, and participants in the U.S. who have been in the trial for fewer than 60 days shall suspend study drug. Importantly, U.S. participants who have completed at least 60-days in the trial should continue treatment.

The FDA action was based on a limited number of cases of drug-induced liver injury that have been identified with tolebrutinib exposure in Phase 3 studies.

The majority of the impacted patients were determined to have concurrent complications known historically to predispose to drug-induced liver injury.

Importantly, the elevations of laboratory values used for monitoring liver injury were reversible after drug discontinuation for all cases.

Following earlier dialog with FDA about these cases, study protocols were revised in May 2022 to update the monitoring frequency, and enrollment criteria were revised to exclude preexisting risk factors for hepatic dysfunction.

Enrollment in the clinical program continues with the revised study protocols and enhanced safety monitoring in countries outside of the U.S. Sanofi is working closely with the independent data monitoring committee members and investigators around the world to evaluate the effectiveness of safety measures.

The program in MS has been enrolling patients since 2019 and includes more than two-thousand patients currently on tolebrutinib therapy with durations of treatment as long as 3 years.

Sanofi remains confident in the future of tolebrutinib as a potentially transformative oral treatment option for people living with MS.

About tolebrutinib

Tolebrutinib is an investigational brain-penetrant and bioactive Bruton’s tyrosine kinase (BTK) inhibitor that achieves CSF concentrations needed for targeting B lymphocytes and microglial cells.

Tolebrutinib is being evaluated in Phase 3 clinical trials for the treatment of relapsing forms of MS (RMS), non-relapsing secondary progressive MS (nrSPMS), primary progressive MS (PPMS), and myasthenia gravis (MG) and its safety and efficacy have not been evaluated by any regulatory authority worldwide.

For more information on tolebrutinib clinical trials, please visit www.clinicaltrials.gov.

About the tolebrutinib Phase III trials

GEMINI 1 (EFC16033): RMS Study of BTKi tolebrutinib
GEMINI 2 (EFC16034): RMS Study of BTKi tolebrutinib
PERSEUS (EFC16035): PPMS Study of BKTi tolebrutinib
HERCULES (EFC16645): Non-relapsing SPMS Study of BTKi tolebrutinib
URSA (EFC17262): Efficacy and Safety of Tolebrutinib in Adult Participants With Generalized Myasthenia Gravis”

https://www.sanofi.com/en/media-room/press-releases/2022/2022-06-30-05-30-00-2471767

AZ’s New study indicates that CKD is present in one out of ten adults

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AZ’s New study indicates that CKD is present in one out of ten adults

 June 30, 2022: “New results from one of the largest real-world evidence studies of chronic kidney disease (CKD) reveal the high burden of the disease on patients and healthcare systems, with an estimated disease prevalence of 10% of the adult population.

Results from the CArdioREnal and MEtabolic (CaReMe) CKD study were published today in The Lancet Regional Health – Europe.

The multinational study of 2.4 million CKD patients across 9 countries in Europe, plus Israel and Canada estimates the prevalence, outcomes and cost of CKD.

While CKD is estimated to be one of the most common diseases affecting one in ten adults, it was found that two out of three patients identified to have CKD in the study were not diagnosed, putting them at high risk of morbidity and mortality and constituting a significant burden on healthcare providers and systems.

Professor Navdeep Tangri, MD PhD, Department of Medicine and Community Health Sciences, University of Manitoba, Winnipeg, Canada, said: “To date, estimates of the prevalence, impact and costs of CKD varied widely due to limited studies of the disease.

The CaReMe CKD study is one of the largest, longest and broadest studies assessing this chronic disease and adding to the body of evidence for CKD.

The results highlight the considerable public health impact of CKD and the importance of early detection and disease management to improve patients’ lives and reduce healthcare costs.”

Alexander de Giorgio-Miller, Senior Vice President, Global Medical, AstraZeneca, said: “We know there remains significant unmet need in chronic kidney disease, with millions more patients yet to be diagnosed.

Real world evidence studies like this are critical to build our understanding of the gaps in diagnosis and clinical care pathways, and to set ambitious quality standards to give patients better access to medicines with the potential to prevent disease progression, disability and premature death.”

CKD is a growing global health burden with increasing contribution to total mortality, and substantial financial costs and impact on healthcare providers.

The study found that between 6-9% of patients with CKD die each year, and the leading cause of hospital visits and healthcare costs were CKD events and co-morbidities such as heart failure (HF). 

he impact of CKD is expected to rise in the years ahead, with both the total number of CKD cases and costs for managing CKD projected to increase even further.1,5

Notes

CKD
CKD is a serious, progressive condition, affecting nearly 850 million people worldwide.6 CKD is 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 advanced and severe stage, 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 are more likely to die from CV causes before reaching ESKD.

CaReMe CKD
The CaReMe CKD study, authored by experts from more than 15 research organisations in Europe and Canada and sponsored by AstraZeneca, is one of the largest and most contemporary (years 2018-2021) CKD studies, including data from 2.4 million CKD patients across 11 countries including Belgium, Canada, Germany, Israel, The Netherlands, Norway, Portugal, Spain, Sweden, Switzerland, and the United Kingdom.

The study assessed individual-level data obtained from digital healthcare systems to determine the prevalence of each stage of CKD and to detail patient characteristics, risks, clinical outcomes and costs associated with CKD across the participating countries.

Patients either had a confirmed diagnosis of CKD or measured CKD based on their urine albumin-creatinine ratio (uACR) value (i.e. level of albumin in the urine) or estimated glomerular filtration rate (eGFR) value (i.e. kidney function).

The CKD study adds to a growing body of real-world evidence from additional CaReMe studies across other cardio renal and metabolic diseases including heart failure and type 2 diabetes.

AstraZeneca in CVRM
Cardiovascular, Renal and Metabolism (CVRM), part of BioPharmaceuticals, forms one of AstraZeneca’s main disease areas and is a key growth driver for the Company.

By following the science to understand more clearly the underlying links between the heart, kidneys and pancreas, AstraZeneca is investing in a portfolio of medicines for organ protection and improving outcomes by slowing disease progression, reducing risks and tackling co-morbidities.

The Company’s ambition is to modify or halt the natural course of CVRM diseases and potentially regenerate organs and restore function, by continuing to deliver transformative science that improves treatment practices and CV health for millions of patients worldwide.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2022/new-chronic-kidney-disease-ckd-study-indicates-that-ckd-is-present-in-one-out-of-ten-adults.html

FDA Introduces Innovative Proposal to Advance Consumer Access to Nonprescription Drugs

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FDA Introduces Innovative Proposal to Advance Consumer Access to Nonprescription Drugs

June 27, 2022: “The U.S. FDA issued a proposed rule titled, “Nonprescription Drug Product with an Additional Condition for Nonprescription Use,” which is intended to broaden the range of marketed nonprescription drugs available to consumers, empowering them to self-treat certain common conditions and improving public health. 

“Nonprescription drug products play a vital role in America’s healthcare system, as millions of people use them to self-manage health conditions every day,” said FDA Commissioner Robert M. Califf, M.D.

“As part of the FDA’s ongoing efforts to improve public health, this proposal can broaden the types of drugs that can be approved as nonprescription—increasing availability of drugs that would otherwise only be available by prescription.”

Nonprescription drug products are used by consumers without the supervision of a health care professional and require the ability of the consumer to determine that they have the condition for which the drug is to be used, and to appropriately use the drug.

Consumers are likely familiar with the “Drug Facts Labeling” on nonprescription drug packaging as the primary source for information about a product’s intended use, directions for use, and important safety information, all designed in understandable language that is tested for consumer comprehension. 

The proposed rule, if finalized, would expand options for consumers by establishing the requirements for a drug company that submits a new application to bring a nonprescription drug product to market with an additional condition for nonprescription use.

Under the proposed rule, when the FDA finds that labeling alone is not sufficient to ensure that the consumer can appropriately self-select and use a drug product in a nonprescription setting, an applicant may submit an application proposing an additional condition for nonprescription use that a consumer must successfully fulfill to obtain the nonprescription drug product.   

An additional condition for nonprescription use is one or more FDA-approved conditions that an applicant of a nonprescription drug product must implement to ensure appropriate self-selection or appropriate actual use, or both, by consumers of the nonprescription drug product.

For example, an applicant could propose an additional condition for nonprescription use that requires a consumer to respond with specific answers to a set of questions on a self-selection test available by either a phone “app” or an automated telephone response system in order to purchase the nonprescription drug product.

Under the proposed rule, if the FDA finds that the additional condition for nonprescription use will permit appropriate self-selection and/or appropriate actual use of the product, and consumers can use the product safely and effectively without the supervision of a healthcare practitioner, it may approve the product for nonprescription use with the additional condition for nonprescription use.

As with all proposals, the safety of patients remains a top priority.

The FDA encourages public comment for this proposed rule. The comment period will end 120 days after the date of publication in the Federal Register.

After the comment period closes, the FDA will review and consider comments as it develops the final rule. The timing of the final rule will depend on the number and substance of the comments.

For more information on the proposed rule, visit the Nonprescription Drug Product with an Additional Condition for Nonprescription Use Proposed Rule webpage. 
 

Related Information

  • Guidance: Innovative Approaches for Nonprescription Drug Products
  • Guidance: Self-Selection Studies for Nonprescription Drug Products”

https://www.pfizer.com/news/press-release/press-release-detail/pfizer-and-biontech-announce-new-agreement-us-government

Pfizer and BioNTech Announce New Agreement with U.S. Government to Provide Additional Doses of COVID-19 Vaccine

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Pfizer and BioNTech Announce New Agreement with U.S. Government to Provide Additional Doses of COVID-19 Vaccine

 June 29, 2022: “Pfizer Inc. and BioNTech SE announced a new vaccine supply agreement with the U.S. government to support the continued fight against COVID-19.

Under the agreement, the U.S. government will receive 105 million doses (30 µg, 10 µg and 3 µg).

This may include adult Omicron-adapted COVID-19 vaccines, subject to authorization from the U.S. Food and Drug Administration (FDA).

The doses are planned to be delivered as soon as late summer 2022 and continue into the fourth quarter of this year.This press release features multimedia.

View the full release here: https://www.businesswire.com/news/home/20220629005833/en/

The U.S. government will pay the companies $3.2 billion upon receipt of the first 105 million doses. Under this agreement, the U.S. government also has the option to purchase up to 195 million additional doses, bringing the total number of potential doses to 300 million.

“As the virus evolves, this new agreement will help ensure people across the country have access to vaccines that may provide protection against current and future variants,” said Albert Bourla, Chairman and Chief Executive Officer, Pfizer.

“Vaccines have been and will remain critical to protecting people of all ages against COVID-19.

We remain proud of our long-term partnership with the U.S. government in helping to address this pandemic, and of the ongoing impact of vaccination efforts in the U.S. and around the world.”

“This agreement will provide additional doses for U.S. residents and help cope with the next COVID-19 wave.

Pending regulatory authorization, it will also include an Omicron-adapted vaccine, which we believe is important to address the rapidly spreading Omicron variant,” said Sean Marett, Chief Business and Chief Commercial Officer of BioNTech.

“We appreciate the continued partnership of the U.S. government in our shared goal to help end this pandemic.”

On June 25, 2022, Pfizer and BioNTech reported pivotal data demonstrating the safety, tolerability and immunogenicity of two Omicron-adapted vaccine candidates.

These data have been shared with regulators, including the FDA, and a request for U.S. Emergency Use Authorization is planned.

The companies have begun manufacturing the Omicron-adapted vaccine candidates at risk so that they can begin deliveries rapidly upon authorization or approval and subsequent recommendation by the U.S. Centers for Disease Control and Prevention’s (CDC), if received, and as directed by the U.S. government.

Eligible U.S. residents will continue to receive the vaccine for free, consistent with the U.S. government’s commitment for free access to COVID-19 vaccines.

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 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.

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 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 16 years of age and older.

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

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

Primary Series

  • a 2-dose primary series to individuals 12 through 15 years of age
  • 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 formulations 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 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 bleeding disorder or is on a blood thinner
  • is immunocompromised or is 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 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 the vaccine recipient 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 adolescent males and adult 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 or difficulty breathing
  • 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, feeling weak or lack of energy, 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, 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.

  • You should always ask your healthcare providers for medical advice about adverse events. Report vaccine side effects to the US Food and Drug Administration (FDA) and the Centers for Disease Control and Prevention (CDC) Vaccine Adverse Event Reporting System (VAERS).”

    https://www.pfizer.com/news/press-release/press-release-detail/pfizer-and-biontech-announce-new-agreement-us-government

GSK selects Target the Future grant recipient in innovation challenge supporting multiple myeloma community

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GSK selects Target the Future grant recipient in innovation challenge supporting multiple myeloma community

June 29, 2022: “GSK plc announced the first recipient of the Target the Future Think Tank Challenge £70,000 (equivalent to approximately $100,000) grant to the HealthTree Foundation, a non-profit organisation helping patients learn more about their health and become their own best advocates.

Their proposal, the “HealthTree Equity and Diversity for Multiple Myeloma Program,” will improve access, education and support for underserved communities and minority patients. 

Tania Small, Vice President, Global Head of Oncology Medical Affairs and Head of R&D Diversity, Equity and Inclusion at GSK, said: “The response to this challenge showcased an abundance of innovation, creativity and ingenuity, which we need to address patients’ unmet needs.

We see great potential in the HealthTree Foundation’s proposal, which goes beyond the science to improve access, equitable care and quality of life for the multiple myeloma community.”

GSK’s grant will help support the HealthTree Foundation as it develops digital delivery tools to distribute information on treatments and trials, facilitate digital communities and expand outreach efforts to support, educate and provide more equitable care to multiple myeloma patients.

Jenny Ahlstrom, Founder and CEO of the HealthTree Foundation, said: “I am alive today because I had the right information and treatments, and we must extend that access to communities of colour.

GSK saw problems and created a programme to target a better future.

Because of GSK’s support, we can implement a strategy to help our community by providing necessary tools to broaden awareness. We’re ready to make a difference in this underserved population suffering from multiple myeloma.” 

Specifically, the Target the Future grant from GSK will aid in the expansion of two of the following initiatives at the HealthTree Foundation:

  • “Black Myeloma Health”: Outreach from the HealthTree Foundation will expand to spotlight Black patients with multiple myeloma and their experiences through video journaling and distribution of new educational brochures with information about relevant treatments and resources.
  • It will also create distinct communities online for Black patients and caregivers to come together and support each other.
  • “HealthTree for Mieloma Multiple”: The organisation’s efforts to support multiple myeloma patients will increase through deployment of Spanish-speaking patient navigators who will help people get answers along their treatment journey in their native language.
  • It will also create referral forms in Spanish at clinics and translate existing HealthTree Foundation materials so more patients can access and understand information relevant to their care.

About the Target the Future Think Tank Challenge

Target the Future is an international, multi-year initiative dedicated to advancing innovation and addressing key needs in the multiple myeloma community. The Think Tank Challenge issued a clarion call for innovative ideas to support patients.

GSK assembled a multidisciplinary advisory group of people personally and professionally connected to the multiple myeloma community and evaluated the top 20 submissions from around the world.

Entries came in from patients, caregivers, healthcare professionals, developers, researchers, advocates and non-profits. Following finalist presentations, the panel evaluated ideas on novelty, feasibility to execute and ability to address unmet needs. The panel unanimously voted in favour of the inaugural winner.

GSK will share detailed updates as the HealthTree Foundation’s idea comes to life and report on results of this effort within the community.”

https://www.gsk.com/en-gb/media/press-releases/gsk-selects-target-the-future-grant-recipient-in-innovation-challenge-supporting-multiple-myeloma-community/

Patient enrollment of phase III tolebrutinib trials paused in the U.S.

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Patient enrollment of phase III tolebrutinib trials paused in the U.S.

 June 30, 2022: “The U.S.FDA has placed Phase 3 studies of tolebrutinib in multiple sclerosis (MS) and myasthenia gravis on partial clinical hold.

As a result, new enrollment in the United States (U.S.) is paused, and participants in the U.S. who have been in the trial for fewer than 60 days shall suspend study drug.

Importantly, U.S. participants who have completed at least 60-days in the trial should continue treatment.

The FDA action was based on a limited number of cases of drug-induced liver injury that have been identified with tolebrutinib exposure in Phase 3 studies.

The majority of the impacted patients were determined to have concurrent complications known historically to predispose to drug-induced liver injury. Importantly, the elevations of laboratory values used for monitoring liver injury were reversible after drug discontinuation for all cases.

Following earlier dialog with FDA about these cases, study protocols were revised in May 2022 to update the monitoring frequency, and enrollment criteria were revised to exclude preexisting risk factors for hepatic dysfunction.

Enrollment in the clinical program continues with the revised study protocols and enhanced safety monitoring in countries outside of the U.S. Sanofi is working closely with the independent data monitoring committee members and investigators around the world to evaluate the effectiveness of safety measures.

The program in MS has been enrolling patients since 2019 and includes more than two-thousand patients currently on tolebrutinib therapy with durations of treatment as long as 3 years.

Sanofi remains confident in the future of tolebrutinib as a potentially transformative oral treatment option for people living with MS.

About tolebrutinib

Tolebrutinib is an investigational brain-penetrant and bioactive Bruton’s tyrosine kinase (BTK) inhibitor that achieves CSF concentrations needed for targeting B lymphocytes and microglial cells.

Tolebrutinib is being evaluated in Phase 3 clinical trials for the treatment of relapsing forms of MS (RMS), non-relapsing secondary progressive MS (nrSPMS), primary progressive MS (PPMS), and myasthenia gravis (MG) and its safety and efficacy have not been evaluated by any regulatory authority worldwide.

For more information on tolebrutinib clinical trials, please visit www.clinicaltrials.gov.

About the tolebrutinib Phase III trials

GEMINI 1 (EFC16033): RMS Study of BTKi tolebrutinib
GEMINI 2 (EFC16034): RMS Study of BTKi tolebrutinib
PERSEUS (EFC16035): PPMS Study of BKTi tolebrutinib
HERCULES (EFC16645): Non-relapsing SPMS Study of BTKi tolebrutinib
URSA (EFC17262): Efficacy and Safety of Tolebrutinib in Adult Participants With Generalized Myasthenia Gravis

https://www.sanofi.com/en/media-room/press-releases/2022/2022-06-30-05-30-00-2471767

CDC recommends Moderna COVID 19 Vaccine for childern and adolescent

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CDC recommends Moderna COVID 19 Vaccine for childern and adolescent

 June 24, 2022: “CDC Director Rochelle P. Walensky, M.D., M.P.H., endorsed the CDC Advisory Committee on Immunization Practices’ (ACIP) recommendation that Moderna’s COVID-19 vaccine be used as an option for children ages 6 through 17 years, in addition to its already recommended use in children 6 months through 5 years and adults 18 years and older.

This recommendation reinforces the use of Moderna’s COVID-19 vaccine as an important tool in the pandemic and provides another vaccine option for children and adolescents.

The ACIP recommendation comes after a thorough review of the scientific evidence demonstrating safety and efficacy, and supports the use of the vaccine among those 6 through 17 years of age.

The following is attributable to CDC Director Dr. Rochelle P. Walensky:

“It is critical that we protect our children and teens from the complications of severe COVID-19 disease.

Today, we have expanded the options available to families by recommending a second safe and effective vaccine for children ages 6 through 17 years.

Vaccinating this age group can provide greater confidence to families that their children and adolescents participating in childcare, school, and other activities will have less risk for serious COVID-19 illness.”

https://www.cdc.gov/media/releases/2022/s0623-moderna-children.html

New Novartis extension phase data show nearly 80% of RMS patients treated with Kesimpta had NEDA-3

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New Novartis extension phase data show nearly 80% of RMS patients treated with Kesimpta had NEDA-3

June 27, 2022: “Novartis announced new data from the Phase 3 ASCLEPIOS I/II trials and the ALITHIOS open-label extension showing continuous treatment with Kesimpta® (ofatumumab) significantly increased the odds of achieving no evidence of disease activity (NEDA-3) versus switching from teriflunomide.

These data were presented at the European Academy of Neurology (EAN) Annual Meeting being held in Vienna, Austria and virtually on June 25–28, 2022.

These data show that after four years of treatment, 78.8% of those who continuously received Kesimpta achieved NEDA-3 (defined as having no MS relapses, no disability worsening and no MRI activity) versus only 51.8% of those who switched from teriflunomide to Kesimpta in the extension phase (odds ratio: 3.89; p<0.001).

These data build on the previously presented efficacy data from ASCLEPIOS I/II and ALITHIOS showing sustained differences in cumulative relapses, MRI lesion activity and the risk of disability worsening between those who were continuously treated with Kesimpta versus those who switched at a later date.

“Early initiation of high-efficacy therapies for the treatment of relapsing multiple sclerosis has been shown to improve long-term outcomes versus escalating from lower efficacy therapies,” said Professor Ludwig Kappos, University Hospital Basel.

“NEDA-3 is an important endpoint for physicians to consider when deciding to initiate high efficacy therapy, with this latest data from ALITHIOS we can clearly see the benefit of starting Kesimpta early versus switching to it later from teriflunomide.”

About Multiple Sclerosis
Multiple sclerosis (MS) is a chronic inflammatory disease of the central nervous system characterized by myelin destruction and axonal damage in the brain, optic nerves and spinal cord.

MS, which affects approximately 2.3 million people worldwide, can be characterized into four main types: clinically isolated syndrome (CIS), relapsing-remitting (RRMS), secondary progressive (SPMS) and primary progressive (PPMS).

The various forms of MS can be distinguished based on whether a patient experiences relapses (clearly defined acute inflammatory attacks of worsening neurological function), and/or whether they experience progression of neurologic damage and disability from the onset of the disease.

About Kesimpta® (ofatumumab)
Kesimpta is a targeted, precisely dosed and delivered B-cell therapy that provides the flexibility of self-administration for adults with relapsing forms of multiple sclerosis (RMS).

It is an anti-CD20 monoclonal antibody (mAb) self-administered by a once-monthly injection, delivered subcutaneously.

Initial doses of Kesimpta are at Weeks 0, 1 and 2, with the first injection performed under the guidance of a healthcare professional.

As shown in preclinical studies, Kesimpta is thought to work by binding to a distinct epitope on the CD20 molecule inducing potent B-cell lysis and depletion.

The selective mechanism of action and subcutaneous administration of Kesimpta allows precise delivery to the lymph nodes, where B-cell depletion in MS is needed, and preclinical studies have shown that it may preserve the B-cells in the spleen.

Once-monthly dosing of Kesimpta differs from other anti-CD20 therapies as it allows faster repletion of B-cells, offering more flexibility in MS management.

Ofatumumab was originally developed by Genmab and licensed to GlaxoSmithKline. Novartis obtained rights for ofatumumab from GlaxoSmithKline in all indications, including RMS, in December 2015.

Ofatumumab has been approved for the treatment of relapsing forms of multiple sclerosis in the United States, European Union, United Kingdom, Canada, China, Switzerland, Singapore, Australia, Japan, Argentina, United Arab Emirates, Albania, and India etc.”

https://www.novartis.com/news/media-releases/new-novartis-extension-phase-data-show-nearly-80-rms-patients-treated-kesimpta-ofatumumab-had-no-evidence-disease-activity-neda-3

FDA Provides Update on Efforts to Increase Supply and Availability of Safe and Nutritious Infant Formula

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FDA Provides Update on Efforts to Increase Supply and Availability of Safe and Nutritious Infant Formula

June 22, 202: “The U.S. Food and Drug Administration is providing an update on the steps it has taken, and will continue to take, to ensure that American consumers have access to safe and nutritious infant formula in the coming weeks and months. 

“The FDA is working night and day to ensure that parents and caregivers can readily find safe and nutritious formula products for any child who needs it,” said FDA Commissioner Robert M. Califf, M.D.

“I have personally spoken with infant formula manufacturers over the past several weeks and all have significantly increased their production efforts, which is resulting in more supply that will be available on stores shelves moving forward.”

FDA Flexibilities Have Resulted in Approximately 365 Million Bottles Worth of Infant Formula

In the past month, the FDA has issued enforcement discretion letters for the importation of the infant formula products from six countries, with a total estimated quantity of 17 million cans, or about 365 million full-size, 8-ounce bottles.

These products have already started to hit the U.S. market and more will appear on store shelves over the coming weeks and months.

Consumers should have confidence that the infant formula that is being imported to the U.S. through this process involved a thorough review of the information provided by the companies, including details about the product’s nutritional adequacy and safety, microbiological testing results, labeling information, and importantly, details about the manufacturing facility’s food safety production practices and inspection history.

Agency Continues Regular Inspections of Infant Formula Facilities

The FDA takes its responsibility seriously to ensure the foods we eat are safe and meet our rigorous standards for quality and safety – this is particularly true for infant formula.

While the FDA Food Safety Modernization Act (FSMA) requires the agency to inspect domestic food facilities at least once every 3 to 5 years, the FDA has a policy of annually inspecting infant formula manufacturers because the products serve as the sole source of nutrition for some of our most vulnerable consumers. 
 
This goal of annual surveillance inspections of these facilities has consistently been met over the years with the exception of 2020 during the COVID-19 pandemic. However, even throughout the COVID-19 pandemic, the FDA continued to inspect facilities, including infant formula facilities.

In fact, we inspected six firms in fiscal year 2020 (Oct. 1, 2019 – Sept. 30, 2020), including several during the height of the pandemic in the summer and fall of 2020.

In order to develop a prioritized list of firms to inspect throughout 2021, the FDA evaluated the firms that we did not inspect in 2020 to focus on firms that met certain risk criteria, including a firm’s inspection history, hazard signals such as consumer complaints, and other factors. 

While the agency postponed certain food surveillance inspections during the pandemic, the FDA resumed its annual surveillance inspection schedule for infant formula facilities beginning in July 2021.

In fiscal year 2021 (Oct. 1, 2020 – Sept. 30, 2021), we prioritized inspections of infant formula firms, conducting 16 domestic inspections throughout the year; including Abbott, Mead Johnson, Gerber, and Nestle. Since the start of fiscal year 2022 (Oct. 1, 2021), we have conducted 10 domestic infant formula inspections and one foreign, including Abbott, Mead Johnson, and Gerber. 

Importantly, it is a firm’s responsibility to ensure the consistent quality and safety of the products they produce.

In addition to our oversight work, the FDA stresses the importance of a company’s quality systems and culture. Ultimately, when problems are found it is the responsibility of the firm to correct those issues to keep consumers safe.

Latest Update on Certain Specialty, Metabolic Infant Formula Being Released by Abbott on a Case-by-Case Basis

The FDA is continuing to work to ensure infants and individuals with medical conditions who rely on certain specialty, amino acid-based and metabolic infant formula products have access to these life-sustaining products.

Some of these products have been available on a case-by-case basis from Abbott Nutrition over the last several months because the risk of not having access to them could significantly worsen underlying medical conditions and in some cases pose life-threatening risks.

To date, this case-by-case release has provided life-sustaining products to more than 2,411 infants and individuals in need, and more than 280,000 containers of additional product remain accessible to those in need.

The products are available on a case-by-case basis to patients, hospitals and institutions by calling Abbott Nutrition at 1-800-881-0876. 

Abbott Nutrition tested certain product lots that Abbott has held in storage since the FDA’s Feb. 17 warning and the company’s voluntary recall due to concerns that they were manufactured under insanitary conditions.

These products have now undergone enhanced batch testing and the company reports none of the batches tested positive for Cronobacter, a bacterium that can cause severe foodborne illness primarily in infants.

The enhanced testing provides further safety assurances to those seeking access to these products.

It’s important to note that even with the enhanced batch product testing being completed, the agency continues to recommend that parents and caregivers seeking access to these products first work with their child’s medical provider to determine whether comparable products or other changes to feeding practices may be an appropriate substitute. 

If comparable, alternative products are not available or appropriate, parents and caregivers using these products should consider following the FDA and CDC’s most current advice on how to reduce the possibility of a Cronobacter infection.

Because powdered formula is not sterile and can also be contaminated in homes, it is advised to wash hands with soap and water, especially before preparing bottles and feeding.

Ensuring all surfaces and feeding items are clean when preparing infant formula will also reduce the potential for possible contamination. 

Abbott’s Sturgis Facility Update and Prior Inspections at the Facility

Abbott recently announced that recent severe weather and rainfall resulted in flooding in areas of the Sturgis facility on Monday, June 13.

While this is an unfortunate setback and a reminder that natural weather events can also cause unforeseen supply chain disruptions, Abbott is working quickly to assess the damage and will be reporting its progress to the agency in the days ahead.

We will return to the facility and work closely with Abbott so that the Sturgis facility can restart producing safe and quality formula products as quickly as possible.
 
Making sure that parents and caregivers have access to both safe and available infant formula remains a top priority for the FDA, and our teams are working night and day to help make that happen.

Separately, given the overwhelming public interest in the FDA’s prior inspections of the Sturgis facility, the FDA is also releasing establishment inspection reports for several previous Abbott inspections.

As previously reported, during our 2019 and 2021 inspections, the agency collected and tested samples of finished products.

These samples tested negative for pathogens. During these inspections, it was determined the firm had found batches of Cronobacter contaminated finished product and had taken the appropriate action to destroy three batches, two in 2019 and one in 2020, before distribution. 

Our most recent inspection in January 2022 was a for cause inspection due to consumer complaints of infant illnesses following consumption of infant formula produced at Abbott’s Sturgis facility. 

Agency Review of Abbott Infant Formula Consumer Complaints  

The work the FDA does to make sure the foods we eat are safe is constant. This work includes ongoing surveillance of every consumer complaint it receives about the products the agency regulates.

To date, the FDA has reviewed and investigated a total of 129 complaints associated with Abbott Nutrition formula products. Of these, 119 complaints were reported after Abbott voluntarily recalled product on February 17.

The FDA has previously reported its review of complaints related to nine infant deaths.

Only two were associated with the Abbott Nutrition Sturgis plant investigation, and despite extensive investigation the evidence does not rule in or rule out a definitive link between these infant deaths and the product produced at Abbott Nutrition’s Sturgis plant.

The agency was notified of one additional consumer complaint on June 10, 2022, that resulted in an infant death in January 2022.

The agency has initiated an investigation, given that the complaint referenced that the infant had consumed an Abbott product. However, the investigation of this most recent consumer complaint is in its preliminary stages and the agency will provide an update as it learns more.”

https://www.fda.gov/news-events/press-announcements/fda-provides-update-efforts-increase-supply-and-availability-safe-and-nutritious-infant-formula

FDA Denies Authorization to Market JUUL Products

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FDA Denies Authorization to Market JUUL Products

June 23, 2022: “The U.S. Food and Drug Administration issued marketing denial orders (MDOs) to JUUL Labs Inc. for all of their products currently marketed in the United States.

As a result, the company must stop selling and distributing these products. In addition, those currently on the U.S. market must be removed, or risk enforcement action.

The products include the JUUL device and four types of JUULpods: Virginia tobacco flavored pods at nicotine concentrations of 5.0% and 3.0% and menthol flavored pods at nicotine concentrations of 5.0% and 3.0%.

Retailers should contact JUUL with any questions about products in their inventory.

“Today’s action is further progress on the FDA’s commitment to ensuring that all e-cigarette and electronic nicotine delivery system products currently being marketed to consumers meet our public health standards,” said FDA Commissioner Robert M. Califf, M.D.

“The agency has dedicated significant resources to review products from the companies that account for most of the U.S. market.

We recognize these make up a significant part of the available products and many have played a disproportionate role in the rise in youth vaping.”

These MDOs only pertain to the commercial distribution, importation and retail sales of these products, and do not restrict individual consumer possession or use—the FDA cannot and will not enforce against individual consumer possession or use of JUUL products or any other tobacco products. 

After reviewing the company’s premarket tobacco product applications (PMTAs), the FDA determined that the applications lacked sufficient evidence regarding the toxicological profile of the products to demonstrate that marketing of the products would be appropriate for the protection of the public health.

In particular, some of the company’s study findings raised concerns due to insufficient and conflicting data – including regarding genotoxicity and potentially harmful chemicals leaching from the company’s proprietary e-liquid pods – that have not been adequately addressed and precluded the FDA from completing a full toxicological risk assessment of the products named in the company’s applications. 

To date, the FDA has not received clinical information to suggest an immediate hazard associated with the use of the JUUL device or JUULpods.

However, the MDOs issued today reflect FDA’s determination that there is insufficient evidence to assess the potential toxicological risks of using the JUUL products.

There is also no way to know the potential harms from using other authorized or unauthorized third-party e-liquid pods with the JUUL device or using JUULpods with a non-JUUL device.

The FDA recommends against modifying or adding substances to tobacco products. JUUL users are encouraged to report any unexpected health problems or product problems to the FDA through the Safety Reporting Portal and to seek medical attention as necessary.

“The FDA is tasked with ensuring that tobacco products sold in this country meet the standard set by the law, but the responsibility to demonstrate that a product meets those standards ultimately falls on the shoulders of the company,” said Michele Mital, acting director of the FDA’s Center for Tobacco Products.

“As with all manufacturers, JUUL had the opportunity to provide evidence demonstrating that the marketing of their products meets these standards.

However, the company did not provide that evidence and instead left us with significant questions.

Without the data needed to determine relevant health risks, the FDA is issuing these marketing denial orders.” 

Any products subject to an MDO may not be offered for sale or distributed in the United States, or the FDA may take enforcement action. 

In addition to ensuring that JUUL complies with this order, as with unauthorized products generally, the FDA intends to ensure compliance by distributors and retailers.

Specifically, the FDA notes that all new tobacco products on the market without the statutorily required premarket authorization are marketed unlawfully and are subject to enforcement action.  

As the FDA has stated in the past, unauthorized electronic nicotine delivery system (ENDS) products for which no application is pending, including for example, those with an MDO, are among our highest enforcement priorities.

Therefore, the FDA encourages retailers to discuss products in their inventory with their suppliers including the current status of any particular tobacco product’s marketing application or marketing authorization.

Manufacturers will be the best source of that information and retailers should rely on manufacturers directly to inform decisions about which products to continue selling.

There are many resources to help smokers who want to quit. Quitting all tobacco products is the best possible path to good health.

Some current JUUL users who will not have access to JUUL products following this action or current smokers who want to transition away from cigarettes and cigars may decide to switch to other ENDS products that have been reviewed and authorized by the FDA based on their potential to benefit adult smokers. 

To date, the FDA has authorized 23 ENDS products. Under the PMTA pathway, applicants must demonstrate to the agency, among other things, that permitting the marketing of the new tobacco product would be appropriate for the protection of the public health. 

The FDA continues to work to complete its review of the remaining pending applications for deemed products submitted by the Sept. 9, 2020, deadline.

Related Information

  • Tobacco Products Marketing Orders
  • Premarket Tobacco Product Applications
  • Quitting Smoking and Other Tobacco Public Health Resources
  • Safety Reporting Portal for Tobacco Products

https://www.fda.gov/news-events/press-announcements/fda-denies-authorization-market-juul-products

FDA Releases Action Plan for Rare Neurodegenerative Diseases, Including ALS

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FDA Releases Action Plan for Rare Neurodegenerative Diseases, Including ALS

June 23, 2022: “The U.S. Food and Drug Administration unveiled its Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis (ALS) – a five-year strategy for improving and extending the lives of people living with rare neurodegenerative diseases by advancing the development of safe and effective medical products and facilitating patient access to novel treatments. 

“The effects of rare neurodegenerative diseases are devastating, with very few effective therapeutic options available to patients.

We recognize the urgent need for new treatments that can both improve and extend the lives of people diagnosed with these diseases,” said FDA Commissioner Robert M. Califf, M.D.

“To face that challenge and to accelerate drug development, we need innovative approaches to better understand these diseases while also building on current scientific and research capabilities.

This action plan, especially including the use of public-private partnerships and direct involvement of patients, will ensure the FDA is working toward meeting the task set forth by Congress to enhance the quality of life for those suffering by facilitating access to new therapies.” 
 
The action plan is a blueprint for how the agency will move forward in aggressively tackling challenges in drug development for rare neurodegenerative diseases, including ALS, in order to improve patients’ health.

Specific actions include regulatory science initiatives, enhancements to existing programs and new policy initiatives.

The plan has been developed in accordance with the provisions of the Accelerating Access to Critical Therapies for ALS Act, or ACT for ALS, that President Biden signed into law on December 23, 2021. 

Within a five-year span, the plan will focus on bolstering scientific advancement and promoting innovation for rare neurodegenerative diseases by engaging in targeted activities including: 

  • Establishing the FDA Rare Neurodegenerative Diseases Task Force (FY 22).
  • Establishing the public-private partnership for rare neurodegenerative diseases (FY 22).
  • Developing disease-specific science strategies (FY 22 – FY 26).
  • Leveraging ongoing FDA regulatory science efforts.

The ALS Science Strategy is an element of the plan focused specifically on ALS.

It provides a forward leaning framework for FDA activities to assess key regulatory science priorities. The focus areas of the ALS Science Strategy are to:

  • Improve characterization of disease pathogenesis and natural history —including quantifying disease progression, predictive and prognostic biomarkers, and efficient translation and implementation of basic science discovery is needed.
  • Facilitate patient access to new drugs whenever possible and promote greater participation in clinical trials by reducing barriers and burdens faced by diverse populations; utilizing digital health technologies and decentralized trial designs; and ensuring mechanisms for expanded access (generally outside of a clinical trial) are appropriately integrated into development programs.
  • Enhance clinical trial infrastructure and agility to enable early selection of promising therapeutic candidates for further development, optimize clinical trial design, improve access to the trials, streamline clinical trial operations, and reduce the time and cost of drug development.

Key to the success of the FDA’s implementation of the ALS Science Strategy will be patient engagement, public workshops, research projects, coordination across FDA centers and offices and collaboration with the National Institutes of Health (NIH).

ACT for ALS also requires the Department of Health and Human Services (HHS) to implement a Public-Private Partnership for Neurodegenerative Diseases between NIH, FDA and one or more outside entities through cooperative agreements, contracts or other appropriate mechanisms to advance the understanding of neurodegenerative diseases and foster development of treatments for ALS and other rare neurodegenerative diseases.

It also directs the agency to award grants and contracts to public and private entities to cover the costs of research and development of interventions that are intended to prevent, diagnose, mitigate, treat or cure ALS and other rare neurodegenerative diseases.”

https://www.fda.gov/news-events/press-announcements/fda-releases-action-plan-rare-neurodegenerative-diseases-including-als

For Wilson disease, ALXN1840 shows rapid and sustained improvement in copper mobilisation from tissues

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For Wilson disease, ALXN1840 shows rapid and sustained improvement in copper mobilisation from tissues

June 23, 2022: “Detailed results from the positive FoCus Phase III trial in Wilson disease showed that ALXN1840, an investigational once-daily, oral medicine, met its primary endpoint demonstrating three-times greater copper mobilisation from tissues compared to the standard of care (SoC) arm (Least Square Mean Difference [LSM Diff] 2.18 µmol/L; p< 0.0001), including in patients who had been treated previously for an average of 10 years.1

In the trial, people taking ALXN1840 experienced rapid copper mobilisation, with a response at four weeks and sustained through 48 weeks.

Results from the trial will be presented on 23 June at the 2022 International Liver Congress (ILC) in London.

Wilson disease is a rare and progressive genetic condition in which the body’s pathway for removing excess copper is compromised.

This may result in the accumulation of copper in a person’s liver, brain or other vital organs. Damage from excess copper build-up in tissues and organs may lead to symptoms of liver, neurological and psychiatric diseases, which may be irreversible.

Even after SoC treatment is initiated, some patients experience worsening of disease, especially of neurologic symptoms.

Change in neurological scale scores and clinician-reported functional assessments with ALXN1840 treatment were also evaluated in a post-hoc analysis as secondary endpoints in the Phase III trial.  

In patients who were symptomatic at baseline, there were greater improvements in neurological scores for those treated with ALXN1840 compared to SoC (Unified Wilson Disease Rating Scale [UWDRS] part II symptomatic ALXN1840 -1.7, SoC -0.8; UWDRS Part III symptomatic ALXN1840 -2.91, SoC -1.31). However, there were no significant differences between treatment groups observed at 48 weeks.1  

Most patients in the trial had low symptom scores at baseline, so there was minimal room for total score improvement (UWDRS Part II ALXN1840 -0.6, SoC -0.3; UWDRS Part III ALXN1840 -2.20, SoC -1.02).

As people with Wilson disease experience a highly varied degree of symptoms4, this total score may not reflect the extent of disease severity.

ALXN1840 was well tolerated and the long-term safety and efficacy of ALXN1840 is being assessed in an up to 60-month extension period.1

Professor Karl Heinz Weiss, MD, Director of the Department of Internal Medicine at Salem Medical Centre Heidelberg and investigator in the FoCus Phase III trial, said: “These data from the largest global trial in Wilson disease to date show significant copper mobilisation from the tissues with ALXN1840, even in patients who were on standard of care for over a decade on average.

These results have the potential to reframe the way doctors can think about the disease given that current therapies focus on removing copper from the blood. 

We are also encouraged by initial neurological improvement with ALXN1840 in those who were symptomatic and believe that assessing individual patient experiences may provide a better understanding of the impact on daily life.”

Marc Dunoyer, Chief Executive Officer, Alexion, said: “Many people with Wilson disease continue to experience symptoms even after years of intervention with current therapies, illuminating an urgent need to re-evaluate the standard of care.

Applying our 30 years of experience in rare disease clinical development, Alexion has conducted rigorous scientific research to bring fresh thinking to Wilson disease around the importance of copper mobilisation from the tissues.

These data further our efforts to potentially introduce a novel treatment for patients who have gone decades without meaningful innovation.”

Summary of efficacy and safety results

The primary endpoint gauged the daily mean Area Under the Effect Curve (AUEC) for directly measured non-ceruloplasmin-bound copper (dNCC)ii over 48 weeks.

The dNCC parameter includes copper bound in an inert complex with ALXN1840.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2022/alxn1840-shows-rapid-and-sustained-improvement-in-copper-mobilisation-from-tissues.html

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