Health
Sarepta Seeks Broader Approval for Gene Therapy Even After Drug Trial Fizzled
- Therapy for kids’ muscle disease hasn’t shown clear benefit
- FDA’s accelerated approval pathway remains under scrutiny
A technician runs a test at a lab.
Photographer: Lauryn Ishak/BloombergThis article is for subscribers only.
Normally when a drug fails to show a clear effect in a final-stage trial, the company developing it goes back to the drawing board. But not Sarepta Therapeutics Inc.
Late Monday, the company said a trial of its $3.2 million gene therapy for Duchenne muscular dystrophy failed to clearly slow the disease in a year-long trial of 125 young kids with the progressive muscle-wasting disease. The company’s stock declined 37% Tuesday, wiping out $3.76 billion in market value.