A second patient death has been reported in connection with Sarepta Therapeutics’ gene therapy Elevidys, which treats muscular dystrophy. The therapy, approved in 2023 for children with a specific genetic variant of Duchenne’s muscular dystrophy, is believed to cause liver injuries in some patients.
Sarepta announced it would pause shipments of the therapy for older patients who cannot walk, citing increased use of immune-system suppressing drugs as part of new safety protocols. The FDA has been cooperating with the company on these changes.
Shares of Sarepta plummeted by 42% after the announcement, as Wall Street analysts speculated that the FDA might impose more restrictions or even block the therapy’s use in non-ambulatory patients.
Elevidys is the first gene therapy approved for muscular dystrophy and costs $3.2 million per treatment. The therapy uses a disabled virus to insert a replacement gene into patient cells.
Source: https://apnews.com/article/sarepta-muscular-dystophy-duchennes-gene-therapy-255d04aa3db9bd171c9bf006328ba5d3