Sarepta继续Elevidys的发货，尽管FDA要求停止

Despite an FDA request to halt shipments, Sarepta Therapeutics (NASDAQ:SRPT) announced late Friday that the company will continue to ship its gene therapy Elevidys, developed with Roche (OTCQX:RHHBY), for ambulant patients with Duchenne muscular dystrophy, a rare neuromuscular disorder.

The Cambridge, Massachusetts-based Sarepta said that the FDA informally requested the company to voluntarily halt shipments on Friday after three patient deaths were linked to Elevidys and another gene therapy developed by the company this year.

Previously, Sarepta (NASDAQ:SRPT) paused Elevidys shipments to non-ambulant patients with DMD pending an update to the drug’s label after two patient deaths were linked to the treatment early this year.

“Based on our comprehensive scientific interpretation of the data, which shows no new or changed safety signals in the ambulant patient population, we will continue to ship ELEVIDYS to the ambulant population,” the company said.

However, Sarepta (NASDAQ:SRPT) drew public scrutiny on Thursday after a third patient who received SRP-9004, another gene therapy developed by the company for limb-girdle muscular dystrophy, died from liver failure in an early-stage trial.

“The LGMD study participant who passed away was not treated with ELEVIDYS, and the dosing for the SRP-9004 trial had concluded at the time of his death,” SRPT noted, adding that it will continue discussions and share information with the FDA to ensure patient safety and access to care.