by I. Edwards
A affected person has died whereas receiving Elevidys, a gene remedy for Duchenne muscular dystrophy, marking the primary recognized dying linked to the remedy.
The corporate behind the drug, Sarepta Therapeutics, stated that the affected person suffered acute liver damage, a recognized facet impact, The Related Press reported. Nevertheless, the “severity” of this case had not been seen earlier than, the corporate famous. Elevidys has been utilized in greater than 800 sufferers, the corporate said.
Sarepta didn’t launch particulars concerning the affected person however stated a latest an infection could have performed a task within the liver damage. The remedy has been utilized in greater than 800 sufferers, the corporate stated.
The corporate plans to replace the prescribing data for Elevidys to replicate this case.
Shares of Sarepta, based mostly in Cambridge, Massachusetts, dropped greater than 27% on Tuesday, closing at about $73 per share.
Elevidys, which prices $3.2 million for a one-time infusion, was the primary gene remedy authorized within the U.S. for Duchenne muscular dystrophy, a uncommon situation that causes muscle weak point, mobility loss and early dying in males.
The FDA first granted the drug expedited approval in 2023, regardless of some scientists questioning its effectiveness.
In 2024, the company expanded full approval to incorporate sufferers 4 and older, no matter mobility standing.
The remedy works by utilizing a disabled virus to insert a gene that helps the physique produce dystrophin, a protein essential for muscle operate. Sarepta stated greater than 800 sufferers have obtained Elevidys thus far, The Related Press reported.
Sarepta has secured FDA accelerated approval for 3 different Duchenne medication since 2016. However none have but been confirmed to be efficient and additional research are nonetheless ongoing.
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Sarepta’s gene remedy underneath scrutiny after affected person dying (2025, March 19)
retrieved 19 March 2025
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