- Sarepta Therapeutics on Monday won conditional approval from the Food and Drug Administration for its Duchenne muscular dystrophy (DMD) treatment, after a months-long delay left the company and DMD patients in regulatory limbo.
- Sarepta will be required to carry out a clinical trial to confirm the drug's clinical benefit, and the Food and Drug Administration made clear a failure to verify efficacy could lead the regulator to withdraw approval for the treatment.
- Shares in Sarepta skyrocketed by over 90% at one point Monday morning, as the FDA's decision resolved a long-standing question of whether the company would be sent back to the drawing board. The drug, now known as Exondys 51 (eteplirsen), is the first approved treatment for DMD.
The FDA has signed off on approval of Sarepta's DMD drug after several years of back and forth with the community. While the drug did pass muster for a conditional approval, the decision is a contentious one. Regulators had been originally expected to make a decision on the drug in May.
Advocates for the community have been particularly outspoken and criticized the FDA for passing over drugs from several other companies, including PTC Therapeutics and BioMarin. An advisory committee to the FDA voted against approval of eteplirsen earlier this year, raising further ire from the community.
The regulatory agency and its experts have long claimed that while the unmet need is very high in this case, the drug candidates in development have not shown strong efficacy.
DMD is a genetic disease that affects young boys, many of which die before reaching adulthood. The disease is characterized by muscle wasting and most of these boys lose the ability to walk and eventually succumb to respiratory failure. Advocacy groups — composed largely of parents of the children — have been major supporters of DMD developers and clinical trials seeking to test disease-modifying drugs.
Several companies have pulled out of the space after the FDA made clear there was no path forward for approval for their drugs. BioMarin was forced to announced this spring that it would end pursuit of approval of its drug, Kyndrisa (drisapersen).