FDA and EMA Accept Marstacimab Regulatory Submissions for the Treatment of Hemophilia A and B - Seite 2

About Marstacimab

Marstacimab is a human monoclonal immunoglobulin G isotype, subclass 1 (IgG1) that targets the Kunitz 2 domain of tissue factor pathway inhibitor (TFPI), a natural anticoagulation protein that functions to prevent the formation of blood clots. Marstacimab is in development 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 in hemophilia A with inhibitors or hemophilia B with inhibitors.

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

About the BASIS study

BASIS is a global Phase 3, open-label, multicenter study evaluating annual bleed rate (ABR) through 12 months of treatment with marstacimab, an investigational, novel subcutaneous therapy option, in approximately 145 adolescent and adult participants ages 12 to <75 years with severe hemophilia A (defined as FVIII <1%) or moderately severe to severe hemophilia B (defined as FIX activity ≤2%) with or without inhibitors. Approximately 15% of participants are adolescents (ages 12 to <18 years old). This study is comparing treatment with a run-in period for patients prescribed factor replacement therapy or bypass therapy during a six-month Observational Phase with a 12-month Active Treatment Phase, during which participants receive prophylaxis (a 300 mg subcutaneous loading dose of marstacimab, followed by 150 mg subcutaneously once weekly) with potential for dose escalation to 300 mg once weekly.

Pfizer is also conducting BASIS KIDS, an open-label study investigating the safety and efficacy of marstacimab in children ages 1 to <18 years with severe hemophilia A or moderately severe to severe hemophilia B with or without inhibitors. The study will compare 12 months of historical standard treatment to marstacimab prophylaxis.

About Hemophilia

Hemophilia is a family of rare genetic blood diseases caused by a clotting factor deficiency (FVIII in hemophilia A, FIX in hemophilia B), which prevents normal blood clotting. Hemophilia is diagnosed in early childhood and impacts more than 400,000 people worldwide.ⁱⁱ The inability of the blood to clot properly can increase the risk of painful bleeding inside the joints, which can cause joint scarring and damage. People living with hemophilia can suffer permanent joint damage following repeated bleeding episodes.^i,ii