WASHINGTON (AP) — Sarepta Therapeutics stated Tuesday {that a} affected person died whilst taking its carefully watched gene remedy for muscular dystrophy, sending corporate stocks plummeting greater than 25%.The younger guy died of acute liver damage, a identified facet impact, Sarepta stated in a commentary. However the corporate stated the “severity” of the affected person’s case had no longer in the past been observed with the remedy, referred to as Elevidys. It’s the primary identified affected person dying with the remedy, which has been utilized in greater than 800 sufferers, the corporate stated.In 2023, Elevidys won expedited U.S. approval in spite of issues from some Meals and Drug Management scientists about its effectiveness in treating Duchenne muscular dystrophy. It’s the primary gene remedy licensed within the U.S. for the uncommon muscle-wasting situation, which reasons weak point, lack of mobility and early dying in men.
The FDA granted complete approval remaining 12 months for Duchenne’s sufferers with a specific genetic mutation, increasing its use to sufferers 4 and older, irrespective of whether or not they’re nonetheless in a position to stroll. Up to now it was once handiest to be had for more youthful sufferers who had been nonetheless cellular.
Sarepta stated in a commentary the affected person who died had a up to date an infection that may have contributed to the liver damage. The corporate stated it plans to replace the prescribing knowledge for Elevidys to replicate the case.Stocks of the Cambridge, Massachusetts-based corporate fell greater than 27% Tuesday to near at about $73 every.
Elevidys makes use of a disabled virus to insert a alternative gene for generating dystrophin into affected person cells. It prices $3.2 million for a one-time infusion.Sarepta has won FDA sped up acclaim for threeother Duchenne’s medication since 2016. None has but been showed to paintings; research designed to protected complete FDA approval are ongoing.___The Related Press Well being and Science Division receives toughen from the Howard Hughes Clinical Institute’s Science and Tutorial Media Team and the Robert Picket Johnson Basis. The AP is just liable for all content material.
