By MATTHEW PERRONE, AP Well being Author
WASHINGTON (AP) — Sarepta Therapeutics stated Tuesday {that a} affected person died whereas taking its intently watched gene remedy for muscular dystrophy, sending firm shares plummeting in morning buying and selling.
The younger man died of acute liver harm, a recognized aspect impact, Sarepta stated in a press release. However the firm stated the “severity” of the affected person’s case had not beforehand been seen with the remedy, known as Elevidys. It’s the primary recognized affected person demise with the remedy, which has been utilized in greater than 800 sufferers, the corporate stated.
In 2023, Elevidys obtained expedited U.S. approval regardless of considerations from some Meals and Drug Administration scientists about its effectiveness in treating Duchenne muscular dystrophy. It’s the primary gene remedy permitted within the U.S. for the uncommon muscle-wasting situation, which causes weak point, lack of mobility and early demise in males.
The FDA granted full approval final 12 months for Duchenne’s sufferers with a specific genetic mutation, increasing its use to sufferers 4 and older, no matter whether or not they’re nonetheless capable of stroll. Beforehand it was solely out there for youthful sufferers who have been nonetheless cellular.
Sarepta stated in a press release the affected person who died had a current an infection that might have contributed to the liver harm. The corporate stated it plans to replace the prescribing info for Elevidys to replicate the case.
Shares of the Cambridge, Massachusetts-based firm fell greater than 23% to about $78 per share in morning buying and selling.
Elevidys makes use of a disabled virus to insert a substitute gene for producing dystrophin into affected person cells. It prices $3.2 million for a one-time remedy.
Sarepta has obtained FDA accelerated approval for threeother Duchenne’s medicine since 2016. None has but been confirmed to work; research designed to safe full FDA approval are ongoing.
The Related Press Well being and Science Division receives help from the Howard Hughes Medical Institute’s Science and Instructional Media Group and the Robert Wooden Johnson Basis. The AP is solely chargeable for all content material.
Initially Printed:
