Sarepta Stands Firm: FDA Request to Halt Elevidys Shipments Rejected

Sarepta Therapeutics is in the spotlight after three patient deaths linked to its gene therapy, Elevidys. Recently, two teenage boys died from acute liver failure while being treated for Duchenne muscular dystrophy. A 51-year-old man also faced the same fate during a trial for another condition.

Despite this troubling news, Sarepta has chosen to continue shipping Elevidys, citing its review of the data and arguing there are no new safety concerns for patients who can walk. However, the FDA asked Sarepta to halt shipments, worrying that the risk of removing the drug from the market has increased. Baird analyst Brian Skorney noted that the FDA’s request shows heightened risks for Elevidys’s ongoing availability.

The FDA’s scrutiny of Elevidys raises questions about its approval process. Some experts, including FDA Commissioner Marty Makary, are evaluating if the drug should still be on the market, especially after recent deaths. Historically, the FDA has faced criticism for rushing approvals, especially for treatments like Elevidys that didn’t show clear benefits in early trials.

In June, Sarepta paused shipments of Elevidys for patients who can no longer walk. This decision affected about 15% of those treated, as most patients are still able to walk at the time of treatment.

Elevidys has helped over 800 patients, becoming a major revenue source for Sarepta. However, the recent deaths could lead to hesitancy among families desperately seeking options for their children. Advocacy groups like CureDuchenne emphasize the need for clear communication on treatment risks and benefits. Debra Miller, the group’s founder, highlighted that families affected by Duchenne muscular dystrophy often lack alternatives.

As a result of these challenges, Sarepta announced job cuts and paused multiple projects, aiming to save around $400 million annually. The situation is creating a ripple effect within the patient community and the market, pushing Sarepta’s shares down significantly.

In summary, the ongoing concerns surrounding Elevidys reflect broader challenges in the regulatory landscape for gene therapies. Families facing devastating illnesses deserve transparency as they navigate their treatment options in a rapidly evolving field.

For further reading, you can explore this detailed report on gene therapy approvals by the FDA here.

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Duchenne muscular dystrophy, gene therapies, FDA Commissioner, Sarepta Therapeutics, Food and Drug Administration, Bloomberg, patient death, acute liver failure, Elevidys, Limb-girdle muscular dystrophy