FDA Decision on Highly Anticipated DMD Drug Delayed
by Amurtha Penumudi
NEW YORK (Reuters Health) - The U.S. Food and Drug Administration deferred a highly anticipated decision on whether to approve Sarepta Therapeutics Inc's muscle-wasting disorder drug, a month after an advisory panel determined that the treatment was not effective.
Sarepta's shares were up 17.6 percent at $21.69 on Wednesday on hopes that the delay could mean that the drug, eteplirsen, may still be cleared for sale.
Eteplirsen was developed to treat Duchenne muscular dystrophy (DMD), a rare condition that typically emerges in boyhood, causing weakness in the arms and legs, and eventually the lungs and heart. Patients often lose the ability to walk in adolescence.
Patient groups and parents have been arguing passionately in favor of the drug, saying children had benefited from it. There is no other treatment on the market.
Sarepta said it had been told by the FDA that the agency was unable to finish its review by Thursday as planned but would try to complete it in "as timely a manner as possible."
However, analysts said the chance of approval was still low, noting that the FDA had also delayed a decision on BioMarin Pharmaceutical Inc's DMD drug before rejecting it.
"We continue to expect no near-term approval," SunTrust Robinson analyst Edward Nash wrote in a client note, adding that he expected the FDA to make its decision in mid-June.
The deferral, however, buys time for parents and DMD advocacy groups to put more pressure on the FDA.
CureDuchenne, a nonprofit organization which provided early funding to Sarepta for the development of eteplirsen, said it appreciated the care the regulator was taking in the review of the drug.
"We cannot afford to lose another generation of boys to Duchenne because there weren't treatment options. It is urgent that we get the right combination of approved treatments for our boys,” founder and CEO Debra Miller said.
An FDA advisory panel said last month that Sarepta's clinical trial of 12 patients did not provide substantial evidence that the drug was effective.
The panel announced its determination at an emotional public meeting at a hotel in Hyattsville, Maryland after more than 50 patients and family members pleaded for the drug's approval.
While most analysts see the panel vote as the end of the road for eteplirsen, the FDA has in the past ignored a negative recommendation from the panel in the face of strong advocacy.
Up to Wednesday's close, Sarepta's stock had fallen 42 percent since BioMarin's rival drug was rejected.
(Reporting by Amrutha Penumudi in Bengaluru; Editing by Don Sebastian and Ted Kerr)
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