The approval comes almost five months after the Food and Drug Administration and a panel of outside advisers panned the drug, saying there was little evidence that it helped.
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Duchenne muscular dystrophy drug – the first treatment for the degenerative disease – accelerated approval.
It’s the first drug to gain FDA approval for the condition. Under the accelerated approval pathway, the FDA is requiring Sarepta Therapeutics to conduct a clinical trial to confirm the drug’s clinical benefit. While no clinical benefit was found, approval was granted based on the disorder’s potential risks, its life-threatening nature and the lack of any other available therapy, the FDA said. About 13% of the 35,000 DMD patients in the U.S. and Europe have a mutation that might theoretically respond to a drug such as eteplirsen.
Duchenne’s muscular dystrophy is a rare disease, affecting about 1 of every 3,600 boys worldwide and usually causing death by age 25, according to the National Institutes of Health.
Pat Furlong, a patient advocate who lost two sons to the disease, called the announcement “an extraordinary win”. The drug acts on a protein called dystrophin, which plays a role in the growth of muscle fibers.
Until now there have been no FDA-approved drugs for DMD, and pressure has been increasing on the regulator to swiftly approve treatments.The FDA issued a scathing review of the drug in April, suggesting its perceived efficacy in a small number of patients could be due to chance because the company had not conducted a randomized, controlled clinical trial. The FDA is not required to follow the advice of its advisory panels, though it often does.
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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 gain gives Cambridge, Massachusetts-based Sarepta a market value of about $2.4 billion, and the positive FDA news could make it a much more appetizing – and expensive – takeover target.
