Sarepta’s gene therapy data present patients, FDA with familiar quandary

Parents of children with Duchenne muscular dystrophy find themselves in a familiar situation, grasping for a glimmer of hope to lighten the burden of a horrible disease. Disappointed by the results of a Phase III trial of Sarepta’s gene therapy Elevidys, they are not ready to give up on the possibility that it could help their children lead better lives.

The plight of DMD patients has touched FDA leaders deeply, prompting them to make a series of approval decisions about exon-skipping drugs and the gene therapy Elevidys delandistrogene moxeparvovec that rank-and-file agency staff believed were not justified. Based on this history, the DMD community’s views could have a great deal of influence over the decisions FDA makes about the sBLA that Sarepta Therapeutics Inc. (NASDAQ:SRPT) will soon file to seek a label expansion and conversion from accelerated to full approval for Elevidys. ...