Patient dies in Sarepta gene therapy trial, adding to safety concerns
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BioCentury first reported the man's death.
The death will likely further stoke safety concerns around Sarepta's gene therapies, as well as raise questions around the company's transparency.
Although they deliver different genes, Elevidys and the company's experimental limb girdle gene therapies rely on the same engineered virus to ferry the gene to patients' muscles. In all three cases, patients suffered acute liver failure after treatment.
Sarepta did not disclose the patient's death Wednesday, when it announced 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.
Asked on a Wednesday investor call if the decision was driven by any safety concerns, CEO Doug 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.
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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.
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.
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