Sarepta Therapeutics will comply with an FDA request last week to halt shipments of its Duchenne muscular dystrophy gene therapy, an about-face after the company earlier refused to do so, reports Endpoints News, and coming hard on the heels of another tragedy linked to a separate treatment trial.
The treatment – Elevidys – has caused two patient deaths and been under investigation by the FDA for weeks. On Friday, the FDA said it had asked the company to halt shipments of the medication, but Sarepta had refused, setting up a showdown with the regulator.
On Monday night, Sarepta released a statement saying it had decided to halt the shipments, and that the pause would give it time to respond to questions from the FDA and to complete an ongoing review of updated safety labelling for the therapy.
It said the decision was a difficult but necessary step to preserve its working relationship with regulators.
Earlier in the day, the FDA said it would use its “full regulatory authority” to enforce its request. The agency didn’t immediately respond to a request for comment Monday night.
At least one large children’s hospital had already stopped treating patients. Children’s Hospital Los Angeles stopped treatments last week after the FDA’s announcement.
The company has been reeling from the Duchenne news, and also from the death of another patient in a trial testing a different gene therapy in a disease known as limb-girdle muscular dystrophy.
Sarepta had not disclosed the fatality during an investor call a day earlier, when executives outlined a sweeping restructuring plan that includes seeking partners for nearly all of its limb-girdle programmes.
The FDA has since placed those trials on clinical hold.
Endpoints News article – Sarepta halts US shipments of Duchenne therapy, bowing to FDA (Open access)
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Second death linked to muscular dystrophy gene therapy
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