The Top Line

Inside the commercial growing pains of hemophilia gene therapies


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With Pfizer discontinuing Beqvez and BioMarin scaling back the commercial focus of Roctavian, the curative promise of hemophilia gene therapies is tempered by significant barriers that discourage widespread adoption.

In this week’s episode of “The Top Line,” Fierce Pharma’s Zoey Becker speaks with Glenn Pierce, M.D., Ph.D., vice president of medical at the World Federation of Hemophilia, about the complexities behind these innovative therapies and the multifaceted reasons for their slow uptake. Pierce discusses the competitiveness of the current hemophilia gene therapy market, why Pfizer’s product couldn’t keep up, and the patient populations that risk being left behind.

To learn more about the topics in this episode:  

  • As Pfizer backs out of hemophilia gene therapy space, CSL hopes Hemgenix is here to stay 

  • Pfizer discontinues hemophilia treatment Beqvez, emptying its gene therapy portfolio 

  • BioMarin downsizes Roctavian efforts but keeps hemophilia gene therapy for 3 markets 

See omnystudio.com/listener for privacy information.

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