Sarepta resumes shipping of gene therapy Elevidys to patients who can walk

Sarepta Therapeutics said on Monday it will resume shipping of its gene therapy Elevidys to patients with a rare muscular disorder who can walk, after the FDA recommended the removal of a voluntary hold that was placed on the therapy.

However, the use of the gene therapy remains on hold for Duchenne Muscular Dystrophy patients who cannot walk, the FDA said, adding that it is continuing to work with the company while investigating the death of two patients.

The FDA’s recommendation for ambulatory patients — those who can walk — followed a probe that showed the death of an eight-year-old boy in Brazil, was unrelated to the gene therapy, the agency said.

Roche, which has partnered with Sarepta for commercialization outside the U.S., had previously said the patient’s death was not related to the therapy, according to the reporting physician’s assessment.

Sarepta is also facing intense scrutiny following the death of two non-ambulatory teenage boys associated with Elevidys, as well as a 51-year-old man who had received its experimental gene therapy SRP-9004.