Gene Therapies: US FDA Sticks With Bleeding Rate For Hemophilia Approval Endpoint

 
• By Sue Sutter

Although clotting factor activity levels may someday be validated as a surrogate endpoint, the currently recommended primary efficacy measure for traditional approval remains annualized bleeding rate, agency says in final guidance.

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US FDA recommends annualized bleeding rate as the primary efficacy endpoint for traditional approval of hemophilia gene therapies. • Source: Shutterstock

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