Pfizer secures CDSCO panel nod to study Hemophilia drug Marstacimab
New Delhi: In a significant development, drug firm Pfizer has got a green signal from the Subject Expert Committee (SEC) functional under the Central Drugs Standard Control Organization (CDSCO) to conduct a Phase III clinical study of Marstacimab prophylaxis in participants with severe hemophilia a and b with or without inhibitors.
This came after the firm presented their proposal for a Phase III Clinical Study of Marstacimab Prophylaxis before the committee.
Marstacimab, an anti-tissue factor pathway inhibitor (anti-TFPI), was developed by Pfizer. Marstacimab is a human monoclonal immunoglobulin G isotype, subclass 1 (IgG1) that targets the Kunitz 2 domain of tissue factor pathway inhibitor (TFPI). Marstacimab is in development as a prophylactic treatment to prevent or reduce the frequency of bleeding episodes in individuals with severe hemophilia A or B with or without inhibitors.
Hemophilia is a genetic haematological rare disease that results in a deficiency of a protein that is required for normal blood clotting—clotting factor VIII in hemophilia A and clotting factor IX in hemophilia B.
In September 2019, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to marstacimab for use in combination with inhibitors as a potential treatment for hemophilia A and B.
At the recent SEC meeting for Oncology & Hematology, the committee thoroughly reviewed the proposal presented by Pfizer for conducting a Phase III Clinical Trial of Marstacimab prophylaxis.
While analysing the risk versus benefit to the patients, the committee noted that the safety profile of the study drug from preclinical and clinical studies justified the conduct of the trial.
Further, in line with the innovation vis-a-vis existing therapeutics, the committee observed that the aim of the study is to evaluate the long-term safety, tolerability, and efficacy of marstacimab prophylaxis in participants with severe hemophilia a and b, with or without inhibitors.
In accordance with the unmet medical need in the country, the committee minutes added, "The test drug is used in severe Hemophilia a and b with or without inhibitors."
After detailed deliberation, the committee recommended the grant of permission to conduct the proposed Phase III clinical study.
