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Hemophilia Gene Therapies Should Have REMS For Approval, National Hemophilia Foundation Says

 
• By Brenda Sandburg

Agency should require physician training, facility certification, and patient registry as a condition for approving BioMarin's and CSL Behring’s investigational gene therapies, the advocacy group says. It calls for treatment to be limited to the population studied with no-off label use.

gene therapy
National Hemophilia Foundation wants restrictions on use of gene therapies to mitigate risk • Source: Shutterstock

The National Hemophilia Foundation is urging the US Food and Drug Administration to impose restrictions on the use of gene therapies for hemophilia as a condition of their approval in order to assure their safe use.

The patient advocacy group submitted a citizen petition to the agency asking it to require a Risk Evaluation and Mitigation Strategy (REMS) if it approves BioMarin Pharmaceutical Inc

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