Duchenne muscular dystrophy
After initially refusing to suspend Elevidys distribution after two deaths, Sarepta has now given in to the FDA’s request, noting the need to maintain a good working relationship with the regulator.
In light of recent patient deaths, the FDA has also revoked its platform designation for Sarepta’s AAVrh74 technology. The designation, granted last month, was the first of its kind to be announced publicly.
The rollercoaster week for Sarepta Therapeutics continued, with shares of the embattled gene therapy-turned-siRNA biotech down 37% Friday afternoon as media outlets reported FDA plans to request a stop to all shipments of the Duchenne muscular dystrophy therapy Elevidys following a third patient death linked to the underlying platform.
The patient, who was being treated with an investigational gene therapy for limb-girdle muscular dystrophy, died of acute liver failure, the same complication responsible for the deaths of two boys taking Sarepta’s Duchenne muscular dystrophy treatment Elevidys.
Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta Therapeutics adds a black box warning to the gene therapy for acute liver injury and failure and parts with more than a third of employees.
Around 3,500 FDA employees received termination emails; FDA Commissioner Marty Makary suggests lowering industry user fees and tying review times to drug prices; the regulator opens its trove of complete response letters in the name of transparency; and two companies receive rejections for rare disease therapies.
In its complete response letter, the FDA cited insufficient evidence establish deramiocel’s effectiveness for cardiomyopathy associated with Duchenne muscular dystrophy. The decision comes after CBER Director Vinay Prasad canceled an advisory committee meeting for the therapy.
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.
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