Second Patient Dies After Gene Therapy for Duchenne Muscular Dystrophy: Company

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Sarepta, the company that makes the drug, is pausing it for non-ambulatory patients.

A second patient has died after receiving the gene therapy Elevidys, which was approved by the Food and Drug Administration (FDA) over concerns of staffers, according to the company that makes it.

The 15-year-old boy who died, and the 16-year-old boy whose death was reported earlier in the year, had both received Elevidys, the only gene therapy approved by the FDA for the treatment of a rare form of muscular dystrophy called Duchenne muscular dystrophy (DMD), Sarepta officials said in a call on June 16.

The boys were both non-ambulatory and both died within 90 days of treatment, according to Sarepta.

The 15-year-old died from acute liver failure, Sarepta said in a statement on Sunday. Acute liver injury is listed as a side effect of the product.

Sarepta said it was investigating the cases independently and in relation to each other to identify any common risk factors. The company suspended its Elevidys sales forecast for this year and said it would provide an update with its second-quarter results.

Sarepta officials said the company is pausing shipments of Elevidys for non-ambulatory patients and halting a clinical trial while it develops an โ€œenhanced immunosuppressive regimen,โ€ utilizing the sirolimus drug. They said there is no timeline right now for resuming dosing for the population and that they would try to speak soon with FDA officials about the situation.

โ€œOur paramount priority is the safety and well-being of the patients we serve. We are taking immediate, decisive steps to better understand and mitigate the risk of acute liver failure, including enhancing the immunosuppressive regimen, for those with Duchenne who are non-ambulatory,โ€ Louise Rodino-Klapac, chief scientific officer and head of research & development at Sarepta, said in a statement. โ€œWe are deeply saddened by the loss of a second patient and extend our heartfelt condolences to the patientโ€™s family and his care team during this incredibly difficult time.โ€

โ€œThe FDA is aware of the second reported fatal case of severe liver failure and are treating this situation with the highest level of concern. The FDA will take all appropriate regulatory actions to protect patients during our review of gene therapy products,โ€ an FDA spokesperson said in an email.

Byย Zachary Stieber

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