The Implications of Accelerated Approval of Gene Therapies

Session Description: 

The focus of the August 2023 Advocacy Grand Rounds will be on the recent accelerated approval of Sarepta’s gene therapy drug, Elevidys, for Duchenne’s muscular dystrophy. This drug was seemingly unilaterally decided by FDA CBER Director, Dr. Peter Marks despite the recommendation from his own staff to not approve the drug. Our AGR session will unpack the concerns and implications of Sarepta’s approval and discuss ways to address those concerns.