FDA and EMA accept regulatory submission for Marstacimab to treat hemophilia A and B

  • The submission builds on positive data from the Phase 3 BASIS trial, which was presented at the American Society of Hematology (ASH) annual meeting this past weekend

  • If approved in the U.S. and EU, marstacimab could become the first once-weekly subcutaneous treatment for people with hemophilia B and the first fixed-dose treatment for people with hemophilia A or B

New York, December 11, 2023(BUSINESS WIRE)–Pfizer Inc. (NYSE: PFE) today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for its anti-tissue factor pathway inhibitor (anti-TFPI) candidate marstacimab, for patients with hemophilia A or hemophilia B who do not have factor VIII (FVIII) or factor IX (FIX) inhibitors. The European Marketing Authorization Application (MAA) for marstacimab has also been validated and is currently under review by the European Medicines Agency (EMA).

The FDA has set a Prescription Drug User Fee Act (PDUFA) action date for the fourth quarter of 2024, and the European Commission is expected to make a decision in the first quarter of 2025. If approved in the U.S. and EU, a decision on marstacimab is expected in the first quarter of 2025. Becomes the first weekly subcutaneous injection treatment for people with hemophilia B and the first fixed-dose treatment for people with hemophilia A or B.

“Marstacimab has demonstrated that, if approved, it could be an effective treatment option for appropriate patients when administered subcutaneously via an auto-injector pen at a fixed dose once weekly. This is critical as patients with these diseases People who typically require intravenous infusions said James Rusnak, M.D., Ph.D., senior vice president and chief development officer, Internal Medicine and Infectious Diseases Research and Development at Pfizer. Bringing this important medicine to patients around the world. “

For more than fifty years, the most common treatment for hemophilia A and B has been factor replacement therapy, which replaces missing clotting factors to promote proper blood clotting.I Marstacimab is a novel investigational treatment for hemophilia designed to restore hemostasis by inhibiting TFPI. In appropriate patients with hemophilia A and B, the goal of this treatment is to prevent potentially life-threatening bleeding with once-weekly subcutaneous fixed-dose administration.

The submission of marstacimab is based on efficacy and safety data from the Phase 3 BASIS trial (NCT03938792). Key study results were recently presented at the American Society of Hematology (ASH) Annual Meeting & Expo on December 9, 2023. Enrollment for the inhibitor cohort of the BASIS trial has been completed and is expected to be announced as early as the end of 2024.

About Marstacimab

Marstacimab is a human monoclonal immunoglobulin G isotype 1 subclass (IgG1) that targets the Kunitz 2 domain of tissue factor pathway inhibitor (TFPI), a natural anticoagulant protein that prevents thrombosis. role. Marstacimab is being developed as a prophylactic treatment to prevent or reduce the frequency of bleeding episodes in individuals with hemophilia A or hemophilia B with or without inhibitors.

In September 2019, the U.S. FDA granted fast track designation to marstacimab for routine prophylaxis to prevent or reduce the frequency of bleeding episodes with hemophilia A inhibitors or hemophilia B inhibitors.

Pfizer has three Phase 3 programs investigating treatments for hemophilia patients: marstacimab, fidanacogene elaparvovec (hemophilia B) and giroctocogene fitelparvovec (hemophilia A).

About basic research

BASIS is a global Phase 3, open-label, multicenter study evaluating marstacimab, an investigational new subcutaneous treatment option, over 12 months in approximately 145 adolescent and adult participants aged 12 to less than 75 years. Annual bleeding rate (ABR) in severe hemophilia A (defined as FVIII <1%) or moderate-to-severe hemophilia B (defined as FIX activity 2%), with or without inhibitors. Approximately 15% of participants are adolescents (12 to under 18 years old). This study compared the treatment of patients receiving factor replacement or bypass therapy during a six-month observation period with a run-in period of treatment to a 12-month active treatment phase during which participants received prophylaxis (300 mg subcutaneously loading dose of marstacimab), followed by 150 mg subcutaneous injection once weekly), with the possibility of increasing the dose to 300 mg once weekly.

Pfizer is also conducting BASIS KIDS, an open-label study investigating marstacimab in children 1 to 18 years younger with severe hemophilia A or moderate to severe hemophilia B, with or without an inhibitor. safety and effectiveness. The study will compare 12 months of historical standard of care with marstacimab prophylaxis.

About hemophilia

Hemophilia is a rare genetic blood disorder caused by a deficiency of a clotting factor (FVIII in hemophilia A, FIX in hemophilia B) that prevents normal blood clotting. Hemophilia is diagnosed in early childhood and affects more than 400,000 people worldwide.two, Failure of the blood to clot properly increases the risk of painful bleeding within the joints, which can lead to scarring and damage to the joints. People with hemophilia may suffer permanent joint damage after repeated bleeding.one, two

For decades, the most common treatment for hemophilia A and B was factor replacement therapy, which replaces missing clotting factors. Factor replacement therapy increases the amount of clotting factors in the body to levels that improve clotting, thereby reducing bleeding.three, four Approximately 25-30% of patients with hemophilia A and 3-5% of patients with hemophilia B cannot continue on factor replacement therapy because they develop FVIII and FIX inhibitors.five, six

In a study of patients receiving prophylaxis for hemophilia A or B in the United States, nearly one-third of those treated with high adherence (defined as taking 75% or more of their prescribed infusions) reported that prophylaxis was The time-consuming nature of treatment is the most significant challenge of this regimen.seven, eight Among patients who received fewer infusions than prescribed, nearly 60% cited time commitment as the primary reason for missing infusions.

About Pfizer: Breakthroughs That Change Patients’ Lives

At Pfizer, we apply science and global resources to bring people treatments that extend and dramatically improve their lives. We work to set the standards for quality, safety and value in the discovery, development and manufacturing of healthcare products, including innovative medicines and vaccines. Pfizer colleagues work every day in developed and emerging markets to advance health, prevention, treatments and cures and challenge the most feared diseases of our time. As one of the world’s premier innovative biopharmaceutical companies, we have a responsibility to partner 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’ve been working to make a difference for everyone who relies on us. We regularly publish information that may be important to investors on our website www.pfizer.com. Additionally, for more information, please visit us at www.pfizer.com and follow us on Twitter: @Pfizer and @Pfizer_NewsLinkedIn, YouTube and like on Facebook: Facebook.com/Pfizer.

Disclosure Statement: The information contained in this press release is as of December 11, 2023. Pfizer assumes no obligation to update the forward-looking statements contained in this press release as a result of new information or future events or developments.

This press release contains forward-looking information about marstacimab, an investigational anti-tissue factor pathway inhibitor, and Pfizer’s fidanacogene elaparvovec and giroctocogene fitelparvovec hemophilia programs, including their potential benefits and pending applications for marstacimab from the FDA and EMA. Uncertainties involving significant risks that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, but are not limited to, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, clinical trial commencement and/or completion dates, regulatory submission dates, regulatory approval dates and/or launch dates and new the possibility of unfavorable clinical data and further analysis of existing clinical data; whether or when the inhibitor queue for the BASIS trial will be successful; the risk that clinical trial data will be interpreted and evaluated differently by regulatory authorities; whether regulatory authorities will respond to our are satisfied with the design and results of the clinical studies; if and when applications for marstacimab, fidanacogene elaparvovec, or giroctocogene fitelparvovec may be filed with regulatory authorities in specific jurisdictions; if and when pending marstacimab applications at the FDA and EMA may be approved, and Whether and when regulatory authorities can approve any other applications that may be pending or submitted for marstacimab, fidanacogene elaparvovec or giroctocogene fitelparvovec will depend on a number of factors, including a determination that the product’s benefits outweigh its benefits. Know the risks, determine the efficacy of the product, and whether marstacimab, fidanacogene elaparvovec, and giroctocogene fitelparvovec will be commercially successful if approved; actions made by regulatory agencies that affect labeling, manufacturing processes, safety, and/or other actions that may affect marstacimab, fidanacogene elaparvovec, and giroctocogene fitelparvovec and giroctocogene fitelparvovec’s availability or commercial potential; uncertainty about the impact of COVID-19 on our business, operations and financial performance; and competitive developments.

For a further description of risks and uncertainties, see Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2022 and its subsequent reports on Form 10-Q, including the headings “Risk Factors” and “Risk Factors” part. “Forward-Looking Information and Factors That Could Affect Future Results,” and subsequent reports on Form 8-K, all of which have been filed with the Securities and Exchange Commission and are available at: www.sec.gov and www.pfizer.com.

I Franchini M, Manucci PM. The past, present and future of hemophilia: a narrative review. Orphanet J Rare Dis 7, 24 (2012). https://doi.org/10.1186/1750-1172-7-24.
two, Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd ed.; 2020. Hemophilia, 26(S6), 1158. https://doi.org/10.1111/hae.14046.
three, Centers for Disease Control and Prevention. hemophilia. Last reviewed: April 2023. https://www.cdc.gov/ncbddd/hemophilia/.
No. 4 Weyand AC, Pipeline SW. New treatments for hemophilia. Blood 2019;133(5):389398. doi: https://doi.org/10.1182/blood-2018-08-872291.
v Centers for Disease Control and Prevention. Inhibitors and hemophilia. Last reviewed: April 2023. https://www.cdc.gov/ncbddd/hemophilia/inhibitors.html.
six Peyvandi F, Garagiola I, Seregni S. The future of clotting factor replacement therapy. J Throm Haemost. 2013;11(Suppl 1):8498.
seven Thornburg CD, Duncan NA. Treatment compliance in hemophilia. Patients prefer compliance. 2017;11:1677-1686
https://doi.org/10.2147/PPA.S139851.
eight Hacker MR, Geraghty S, Manco-Johnson M. Barriers to compliance with preventive treatment in hemophilia. Hemophilia 2001;7(4):392-6.

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