FDA Approves Pfizer's Second Hemophilia Drug With Six Months

On Friday, the FDA approved Pfizer Inc’s PFE Hympavzi (marstacimab-hncq) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients with hemophilia A (congenital factor VIII deficiency), or hemophilia B (congenital factor IX deficiency).

Hympavzi is the first and only anti-tissue factor pathway inhibitor (anti-TFPI) approved in the U.S. for hemophilia A or B and the first hemophilia medicine approved in the U.S. to be administered via a pre-filled, auto-injector pen.

Hympavzi can offer a subcutaneous treatment option with a once-weekly dosing schedule and minimal preparation required for each individual administration.

Hemophilia is a family of rare genetic blood diseases caused by a clotting factor deficiency (FVIII in hemophilia A, FIX in hemophilia B), impacting more than 800,000 people globally.

Results from the Phase 3 BASIS trial (NCT03938792) supported the FDA approval of Hympavzi.

In the study, Hympavzi reduced the annualized bleeding rate (ABR) for treated bleeds by 35% and 92% after a 12-month active treatment period compared to routine prophylaxis (RP) and on-demand (OD) treatment, respectively, in patients with hemophilia A or B without inhibitors.

The Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion for marstacimab for the routine prophylaxis of bleeding episodes in adults and adolescents with severe hemophilia A or hemophilia B.

Reuters, citing some analysts, highlights that Hympavzi sales are expected to reach $300 million by 2030.

Earlier this year, FDA approved Pfizer’s Beqvez (fidanacogene elaparvovec-dzkt) for moderate to severe hemophilia B in adult patients who currently use factor IX (FIX) prophylaxis therapy or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var capsid. 

Beqvez is a one-time treatment designed to enable hemophilia B patients to produce FIX themselves rather than the current standard of care, which requires regular intravenous infusions of FIX that are often administered multiple times a week or multiple times a month.

In July, Pfizer, in collaboration with Sangamo Therapeutics Inc, released results from the Phase 3 AFFINE study of giroctocogene fitelparvovec, an investigational gene therapy for adult patients with moderately severe to severe hemophilia A.

Price Action: PFE stock is down 0.14% at $29.12 during the premarket session at last check Monday.

Photo via Shutterstock

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