Sarepta Therapeutics Faces FDA Crackdown after Third Patient Death; George Tidmarsh Named Top FDA Drug Regulator; Multiple Drugs Rejected; Pharma Invests Billions in US Manufacturing

A third patient death linked to Sarepta Therapeutics’ gene therapy programs has been confirmed by the company and FDA, leading to heightened regulatory scrutiny.1234

The most recent fatality was a 51-year-old man with limb-girdle muscular dystrophy (LGMD) who died due to acute liver failure after receiving a Sarepta investigational gene therapy in a clinical trial.12

Prior deaths occurred in pediatric Duchenne muscular dystrophy (DMD) patients treated with Sarepta’s approved AAVrh74-based gene therapy, Elevidys.12

In response, the FDA has halted Sarepta's Elevidys distribution and placed clinical trials for multiple Sarepta gene therapies on hold, citing unreasonable and significant patient safety risk.34

The FDA also revoked Sarepta’s platform technology designation, further elevating regulatory barriers for the company.3

The FDA has demonstrated a firm approach, with Commissioner stating commitment to swift action on safety—signaling a new era of regulatory vigilance.3

Sarepta’s stock fell more than 33% immediately after the news, reversing prior gains.1

As part of broader industry shifts, the FDA recently installed George Tidmarsh as the new top drug regulator, coinciding with a wave of drug rejections and significant new industry investments in US manufacturing infrastructure—with multiple pharma firms committing billions to reshoring production (recent news on these points not detailed in the current search excerpts but align with ongoing trends described in the query).

Sources:

1. https://www.fiercebiotech.com/biotech/3rd-patient-dies-following-treatment-sarepta-gene-therapy-reports

2. https://www.fda.gov/vaccines-blood-biologics/safety-availability-biologics/fda-investigating-deaths-due-acute-liver-failure-following-treatment-sareptas-aavrh74-gene-therapies

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

4. https://www.neurologylive.com/view/third-patient-death-leads-significant-concerns-sarepta-gene-therapy-program