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Sarepta Therapeutics stated on Monday it’s going to pause all shipments of Elevidys in the US after two teenage boys with a uncommon situation known as Duchenne muscular dystrophy, who had obtained the corporate’s gene remedy, died of liver failure this yr.
The U.S. Meals and Drug Administration on Friday requested Sarepta to voluntarily halt shipments of its gene remedy.
The corporate responded that it could proceed offering the remedy to ambulatory sufferers, whereas sustaining a halt it carried out June 15 for non-ambulatory sufferers.
Sarepta’s shares are down 8.6% to $12.17 after the bell. The inventory plummeted on Friday after it disclosed the demise of a 3rd affected person who had obtained an experimental gene remedy, deepening investor considerations over the corporate’s remedies.
Whereas the 51-year-old man with limb girdle muscular dystrophy who died most not too long ago was not taking Elevidys, the experimental remedy he took was primarily based on comparable gene know-how, the FDA stated.
The FDA put Sarepta’s scientific trials for limb girdle muscular dystrophy on maintain on account of security considerations.
All shipments of Elevidys for Duchenne muscular dystrophy shall be quickly paused by shut of enterprise on Tuesday, Sarepta stated Monday.
The corporate stated that the pause will permit it the “time to reply to any requests for data and permit Sarepta and FDA to finish the Elevidys security labeling complement course of.”
Sarepta stated it anticipates a evaluate and dialogue with the FDA.
The FDA didn’t instantly reply to a Reuters request for remark.
After the primary two deaths, Sarepta in June suspended using Elevidys for sufferers whose illness had progressed to the purpose the place they wanted a wheelchair.
Elevidys has full FDA approval for youngsters with DMD who’re nonetheless capable of stroll, and conditional approval for non-ambulatory sufferers.
Final week, Sarepta stated it was including a warning on Elevidys’ label for the danger of acute liver harm and liver failure in DMD sufferers who can stroll. It additionally introduced 500 job cuts and halted improvement of a number of gene therapies for limb girdle muscular dystrophy.
Individually, the Kids’s Hospital Los Angeles stated on Monday it has paused utilization of Elevidys in all sufferers with muscular dystrophy.