FDA Approves Pfizer's Second Hemophilia Drug With Six Months
On Friday, the FDA approved Pfizer Inc’s (NYSE: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
Read Next:
Up Next: Transform your trading with Benzinga Edge's one-of-a-kind market trade ideas and tools. Click now to access unique insights that can set you ahead in today's competitive market.
Get the latest stock analysis from Benzinga?
This article FDA Approves Pfizer's Second Hemophilia Drug With Six Months originally appeared on Benzinga.com
© 2024 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.