Sarepta Reports Third Patient Death in Gene Therapy Studies for Limb-Girdle Muscular Dystrophy

A 51-year-old man enrolled in a Phase 1 clinical trial for limb-girdle muscular dystrophy died from acute liver failure after receiving Sarepta Therapeutics' investigational SRP-9004 gene therapy134.

This is the third reported death associated with Sarepta's gene therapies; the previous two occurred in teenagers with Duchenne muscular dystrophy using the approved gene therapy Elevidys, also linked to acute liver failure134.

All fatal cases shared a common feature:
use of adeno-associated virus (AAV) vectors, which have been tied to liver complications, particularly at high doses required for neuromuscular conditions13.

The FDA has placed Sarepta's clinical trials for limb-girdle muscular dystrophy on clinical hold and requested the suspension of Elevidys distribution following these safety concerns and new potential risks considered significant4.

Sarepta did not initially disclose the limb-girdle trial death during recent corporate communications but later confirmed the incident to media and regulators, stating it was not a new safety signal and that the SRP-9004 program would not be moving forward1.

Sarepta’s stock price dropped sharply—down by double digits—following public confirmation of the new death and regulatory actions13.

Sources:

1. https://www.biopharmadive.com/news/sarepta-limb-girdle-gene-therapy-death/753419/

3. https://www.biospace.com/drug-development/sarepta-reports-a-third-gene-therapy-related-patient-death

4. https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold