US FDA’s Marks Willing To Accept Gene Therapies Occasionally Not Confirming Benefit

CBER director does not want to hold back potentially beneficial products, but also warned that patients will have difficult treatment decisions to make since an immune response might prevent a patient from receiving another gene therapy using the same vector ever again.

gene therapy laboratory — Patients will have to decide whether to accept gene therapy treatment using their own risk tolerance, Marks said. • Source: Shutterstock

Some gene therapies that reach the market through accelerated approval may not ultimately show clinical benefit, but that’s acceptable, said Peter Marks, director of the US Food and Drug Administration’s Center for Biologics Evaluation and Research.

However, he also said the consequences for patients could be substantial in those cases and that they must understand the risks as

US FDA’s Marks Willing To Accept Gene Therapies Occasionally Not Confirming Benefit

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