Pfizer Discontinues Hemophilia Gene Therapy Beqvez As Interest Wanes

Benzinga
02-22

According to multiple media reports on Thursday, Pfizer Inc. (NYSE:PFE) has terminated the global development and commercialization of its hemophilia B gene therapy, Beqvez.

In April 2024, the FDA approved Beqvez for moderate to severe hemophilia B in adult patients who currently use factor IX 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. 

Also Read: uniQure/ CSL Behring’s Hemophilia Gene Therapy Shows Sustained Efficacy and Safety At Four Years

In a statement to Nikkei Asia, Pfizer said the decision was due to "the limited interest patients and their doctors have demonstrated in hemophilia gene therapies.”

The U.S. drugmaker said it would focus on different treatment for the disorder, citing low interest.

In December 2024, Pfizer walked away from its hemophilia A co-development pact with Sangamo Therapeutics Inc (NASDAQ:SGMO) giroctocogene fitelparvovec, an investigational gene therapy product candidate.

According to a Reuters report, Pfizer will continue to invest resources in Hympavzi (marstacimab-hncq), which is approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients with hemophilia A or hemophilia B.

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.

Earlier this month, Novo Nordisk A/S (NYSE:NVO) announced interim results from the phase 3 FRONTIER3 trial of 70 children (aged 1-11 years old) with hemophilia A with and without inhibitors.

Novo Nordisk expects Mim8 regulatory submission during 2025. Data from the ongoing phase 3 FRONTIER program will be disclosed at upcoming congresses and in publications in 2025 and 2026.

In September 2024, Pfizer voluntarily withdrew all lots of Oxbryta (voxelotor) for sickle cell disease (SCD) in all markets where it is approved.

Pfizer's decision is based on the totality of clinical data indicating that the overall benefit of Oxbryta no longer outweighs the risk in the approved sickle cell patient population.

Price Action: PFE stock is up 0.87% at $26.13 at the last check on Friday.

Read Next:

  • Apple Expands Apple TV+ to Android, Eyes Subscription Growth and Ecosystem Expansion, Analyst Says

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?

  • PFIZER (PFE): Free Stock Analysis Report

This article Pfizer Discontinues Hemophilia Gene Therapy Beqvez As Interest Wanes originally appeared on Benzinga.com

© 2025 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

免责声明:投资有风险,本文并非投资建议,以上内容不应被视为任何金融产品的购买或出售要约、建议或邀请,作者或其他用户的任何相关讨论、评论或帖子也不应被视为此类内容。本文仅供一般参考,不考虑您的个人投资目标、财务状况或需求。TTM对信息的准确性和完整性不承担任何责任或保证,投资者应自行研究并在投资前寻求专业建议。

热议股票

  1. 1
     
     
     
     
  2. 2
     
     
     
     
  3. 3
     
     
     
     
  4. 4
     
     
     
     
  5. 5
     
     
     
     
  6. 6
     
     
     
     
  7. 7
     
     
     
     
  8. 8
     
     
     
     
  9. 9
     
     
     
     
  10. 10