Pfizer’s HYMPAVZI Gains FDA Nod for Hemophilia A and B Without Inhibitors

Pfizer Inc. announced the FDA approval of HYMPAVZI™ (marstacimab-hncq) for routine prophylaxis aimed at preventing or reducing the frequency of bleeding episodes. This approval covers both adult and pediatric patients aged 12 years and older who have hemophilia A (congenital factor VIII deficiency) without factor VIII (FVIII) inhibitors, or hemophilia B (congenital factor IX deficiency) without factor IX (FIX) inhibitors. This milestone underscores Pfizer’s commitment to providing innovative therapies for patients with bleeding disorders.

Publish Date: 14-10-2024 Source: Pfizer Inc.

Hemophilia is a severe hereditary hemorrhagic disorder, with significant types of hemophilia A (classic hemophilia or factor VIII deficiency) and hemophilia B (also known as Christmas disease or factor IX deficiency). The disorder is inherited and is characterized by a deficiency of clotting factors in the blood. Hemophilia C is a rare condition caused by a deficiency of clotting factor XI. Approximately 25% of hemophilia A cases are caused by gross gene rearrangements, with 5% due to gross gene deletions and 20% to inversions of a 500-600 kb segment that breaks intron 22 of the F8 gene. Hemophilia B cases due to gross rearrangements of F9 are less than 2%. F9 mutations occur more frequently in male germline than in female germline. Hemophilia can manifest in mild, moderate, and severe forms, depending on the blood level or amount of clotting factor and the patient’s symptoms. Mild hemophilia involves having 6-49% of the normal factor level, with bleeding typically occurring only after a significant injury, trauma, or surgery. Moderate hemophilia is characterized by having 1-5% of the normal factor level, with bleeding episodes, typically following injuries, major trauma, or surgery. Patients with moderate hemophilia may also experience occasional spontaneous bleeding episodes without an obvious cause. Differential diagnosis is required to exclude other conditions presenting with similar symptoms, such as von Willebrand disease, scurvy, diseases of platelet dysfunction, and deficiencies of other coagulation factors.

In the United States, the estimated prevalence of hemophilia is 12 cases per 100,000 U.S. males for hemophilia A and 3.7 cases per 100,000 U.S. males for hemophilia B.

However, the current Hemophilia treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (Emicizumab, SPK-8011, Efanesoctocog alfa (BIVV001), BAY2599023 (DTX201), etc..) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the Hemophilia treatment. The key companies in the advanced development stage are Hoffmann-La Roche, Spark Therapeutics, Inc., Bioverativ, a Sanofi company, Bayer, etc..

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Hemophilia to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2023 to 2033.

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