Solid eyes accelerated approval as DMD gene therapy faces FDA uncertainty

As FDA’s Center for Biologics Evaluation and Research heads toward another leadership turnover next month, Duchenne muscular dystrophy gene therapy developers could be among those most affected. New Phase I/II data could put Solid Biosciences on a path toward accelerated approval, but whether that path remains open may depend on who replaces Vinay Prasad.

Solid Biosciences Inc. (NASDAQ:SLDB) reported that its gene therapy SGT-003 led to higher levels of microdystrophin expression and a higher percentage of microdystrophin-positive fibers than those achieved by marketed gene therapy Elevidys delandistrogene moxeparvovec from Sarepta Therapeutics Inc. (NASDAQ:SRPT), though there were differences in the patient populations across the trials. ...