Duchenne muscular dystrophy
The FDA found that data from a single Phase II study were “insufficient” to justify an accelerated approval review for sevasemten in Becker muscular dystrophy.
The FDA is assessing the need for “further regulatory action” on Sarepta’s Duchenne muscular dystrophy gene therapy in the aftermath of two patient deaths, though the regulator has not yet specified what action this could be.
Sarepta’s shares crashed 41% in premarket trading Monday morning to $21.01 after the biotech reported a second death from acute liver failure, a known side effect of adeno-associated virus-based gene therapies.
Avidity has been given the go-ahead by the FDA to request accelerated approval of delpacibart braxlosiran—potentially the first disease-modifying treatment for facioscapulohumeral muscular dystrophy—with an application in the second half of 2026.
After a major shareholder pushed back, Keros is returning half of its capital to investors in a move that Guggenheim analysts called “a positive step forward.”
Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according to BMO Capital Markets analysts.
Since Elevidys’ accelerated approval in 2023, experts have been clamoring for more data, particularly in older and non-ambulatory children. New results, presented Friday, show mobility improvements in 8- to 9-year-old patients after one year of follow-up.
One day after the European Medicines Agency requested that three clinical trials of Elevidys be placed on hold after the death of a U.S. teenager, a data monitoring committee concluded that they should continue unchanged.
Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death of a patient taking Sarepta’s Elevidys raises important safety questions.
WVE-N531, an oligonucleotide, elicited significant functional benefit and reversal of muscle damage in the Phase II FORWARD-53 trial. Wave plans to file for accelerated approval of the candidate in 2026.
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