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 CommercializationWEBINAR: Early Integrated Evidence Strategy for Cell & Gene TherapiesExploring an Expedited Regulatory Pathway for Cell and Gene TherapiesSuccess Factors for Commercializing Cell and Gene TherapiesSyneos 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.  If you want access to more future-focused, actionable insights to help biopharmaceutical companies better execute and succeed in a constantly evolving environment, visit the Syneos Health Insights Hub. The perspectives you’ll find there are driven by dynamic research and crafted by subject matter experts focused on real answers to help guide decision-making and investment. You can find it all at https://www.syneoshealth.com/insights-hub. Like what you’re hearing? Be sure to rate and review us!  We want to hear from you! If there’s a topic you’d like us to cover on a future episode, contact us at [email protected].