Untangling Sarepta’s Gene Therapy Fallout and Trust Deficit

Sarepta Therapeutics has faced mounting scrutiny following three patient deaths linked to its AAVrh74-based gene therapies:
two in non-ambulatory Duchenne muscular dystrophy (DMD) pediatric patients treated with Elevidys, and one in a 51-year-old adult with limb-girdle muscular dystrophy (LGMD) in a clinical trial using an investigational gene therapy2 3 5.

The FDA originally requested Sarepta to halt all shipments of Elevidys and placed its LGMD gene therapy trials on clinical hold due to safety concerns—a move prompted by these deaths tied to acute liver failure3 5.

Initially, Sarepta refused the FDA’s request, continuing to supply Elevidys for ambulatory DMD patients, arguing available evidence still supported its use in that group; this created a rare standoff between company and regulator and raised concerns over transparency and regulatory trust1 3.

Following further deaths and regulatory pressure, Sarepta ultimately paused shipments of Elevidys, and its partner Roche stopped shipments in certain countries. The FDA also revoked Sarepta's platform technology designation3 5.

The FDA has since made a partial reversal, now backing Elevidys again for ambulatory DMD patients, while broader safety and regulatory issues, including a deficit of trust and demands for improved transparency, continue to reverberate through the gene therapy industry4.

Sarepta’s stock price fell more than 33% after the third reported death and subsequent regulatory actions, highlighting the significant market and industry impact2 3.