Gene Therapy Approval Standards: Available Treatments Influence US FDA’s View On Durability

15 Oct 2020

CBER’s Peter Marks says agency assesses the importance of durability of effect differently for a gene therapy that treats a disease that has no other available therapies versus a condition for which there are multiple approved treatments. Marks’ comments shed more light on the complete response letter for BioMarin’s hemophilia gene therapy Roctavian.

two benches in a park in autumn that point in different directons — US FDA's view on gene therapy effect durability depends upon the disease and treatment landscape. • Source: Shutterstock

The US Food and Drug Administration’s view of the importance of durability for a gene therapy depends, in part, on the condition being treated and the existence of other approved therapies.

Speaking at the virtual Cell & Gene Meeting on the Mesa on 14 October, Center for Biologics Evaluation and Research...