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The Food and Drug Administration will request Sarepta Therapeutics stop all shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, according to a source familiar with the matter.
The move follows the death of two teenagers who were administered the treatment, as well as the death of a 51-year-old man who received a similar product in a clinical trial for a different form of muscular dystrophy.
Sarepta had already paused shipments of Elevidys for all patients who use wheelchairs — most boys and young adults over the age of 12. But the product had remained on the market for younger patients who could still walk.
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Sarepta’s stock was down 38 percent in afternoon trading. The FDA’s plan to request shipments be halted was first reported by Reuters.
It’s a stunning turnaround for a therapy that provided immense hope for many in the Duchenne community, but had never delivered clear and decisive efficacy results in clinical trials. And it comes as Sarepta CEO Doug Ingram and his executive team face withering criticism for failing to promptly disclose some of the deaths and additional cases of severe liver failure that have led to patient hospitalizations.
Sarepta did not disclose the death of the 51-year-old man -- who died last month -- when it announced Wednesday that it was restructuring and laying off 500 employees. The company revealed it was discontinuing all but one of its limb girdle programs — it was developing treatments for six different subtypes — but described it as a financially motivated decision, as all subtypes are quite rare.
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Asked on a Wednesday investor call if the decision was driven by any safety concerns, Ingram said the company had made “a risk-adjusted net present value” calculation and determined the financial opportunity didn’t justify the costs of developing the limb girdle treatments.
A Sarepta spokesperson said Thursday the company “prioritized disclosing to clinicians, regulators, and the community.” Sarepta informed the patient community about the fatal case earlier Thursday, weeks after the man’s death. It’s not clear when regulators and investigators were informed.
“Our deepest condolences go to the family and all those involved in his care,” the spokesperson said. “Advancing the science of genetic medicine is incredibly challenging — especially in ultra-rare diseases like the limb-girdle subtypes. While we do everything possible to ensure patient safety, there is inherent risk in clinical trials, and we are grateful to the courageous patients and families who participate.”
The news will likely be devastating to limb girdle patients and advocates. Their conditions have long been overlooked because they are generally regarded as less deadly than other types of muscular dystrophy, even though they are debilitating and some subtypes can significantly shorten life. They are also too rare to interest most for-profit drug developers.
A few gene therapy companies are targeting individual subtypes, but Sarepta had promised to develop a suite of different treatments, building on the approval of its Duchenne therapy.
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The company said it still plans to file for approval later this year for one subtype, called LGMD 2E. But the three deaths could give regulators pause. Sarepta has said it will present data from its LGMD 2E therapy in October, at the World Muscle Society conference.
The company also detailed on Wednesday plans to test, in a six-month trial, whether a new immune-modulating drug can mitigate the risk of liver toxicity from Elevidys. For now the drug remains off the market for all Duchenne patients who rely on wheelchairs — most patients over the age of 12.