Gene Therapy for Hemophilia

Last year the FDA approved the first gene therapy for hemophilia B. This year, the first gene therapy may be approved in the US for hemophilia A. What does the gene therapy revolution mean for patients, payers, and pharma?

Ankita Chowdhury, Senior Engagement Manager, Commercial Advisory at Syneos Health®, joins the podcast to discuss the landscape of recent hemophilia gene therapy approvals, the state of upcoming additional therapies to market, and key considerations for developers to address in order to optimize access, pricing and reimbursement for patients impacted by this rare disease.

For more of our insights on rare disease and gene therapies, check out:

The Importance of the Right Go-to-Market in Cell Therapy Commercialization

WEBINAR: Early Integrated Evidence Strategy for Cell & Gene Therapies

Exploring an Expedited Regulatory Pathway for Cell and Gene Therapies

Success Factors for Commercializing Cell and Gene Therapies

Syneos Health Podcast: The Value of Registries

NOTE: At the time of recording, Biomarin’s hemophilia A product was expected to receive FDA approval in March of this year. Since then, news broke that FDA review will be delayed until June.

The views expressed in this podcast belong solely to the speakers and do not represent those of their organization.

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