Sarepta Reports Death of DMD Patient Following Elevidys Gene Therapy Treatment

A 16-year-old male patient with DMD died of acute liver failure after receiving Elevidys gene therapy12.
This is the first reported death among over 800 patients treated with Elevidys in clinical trials or commercially13.
Acute liver injury is a known potential side effect of Elevidys and other AAV gene therapies, but death from liver failure had not previously been reported13.
The patient had a recent cytomegalovirus (CMV) infection, which may have been a contributing factor12.
Sarepta plans to update Elevidys' prescribing information to reflect this event13.
Sarepta's stock price dropped 22-25% following the announcement56.
The FDA had previously expanded approval of Elevidys to a wider DMD patient population in 20244.
Analysts suggest this event may make doctors more cautious about prescribing Elevidys, given its modest clinical benefits6.
Sarepta maintains that the overall benefit-risk profile of Elevidys remains positive13.

Sources:

1. https://www.genengnews.com/topics/genome-editing/dmd-patient-dies-after-treatment-with-sarepta-gene-therapy/

2. https://dmdwarrior.com/sixteen-year-old-dies-after-receiving-sareptas-duchenne-gene-therapy-elevidys/

3. https://musculardystrophynews.com/news/sarepta-reports-death-dmd-patient-elevidys-gene-therapy/

4. https://www.biocentury.com/article/652823/cardiologists-voice-safety-concerns-about-sarepta-s-elevidys

5. https://www.biospace.com/business/sareptas-elevidys-death-fdas-vaccine-move-weldon-whiplash-pdufas-in-cardio

6. https://www.biopharmadive.com/news/sarepta-elevidys-duchenne-gene-therapy-death-liver-failure/742765/