January 21, 2016
By Mark Terry, BioSpace.com Breaking News Staff
Cambridge, Mass.-based Sarepta Therapeutics, Inc. announced today that, because of the impending snowstorm expected to blast the east coast, the U.S. Food and Drug Administration (FDA)’s meeting to review the company’s New Drug Application (NDA) for eteplirsen has been postponed.
Investors have been waiting for the results of the FDA’s Peripheral and Central Nervous System Advisory Committee’s findings on eteplirsen for the treatment of Duchenne Muscular Dystrophy (DMD). Just last Friday, the FDA announced that it did not approve San Rafael, Calif.-based BioMarin Pharmaceutical Inc. ’s application for Kyndrisa (drissapersen) for DMD.
The FDA argued that Kyndrisa didn’t show enough benefit. There has been some controversy over how the original FDA panel approached evaluating the drug, and advocacy groups have been arguing that even a drug that doesn’t work terribly well is better than no drug at all for a disease like DMD.
DMD is a muscle wasting disease caused by mutations in the dystrophin gene. The disease is progressive and typically causes death in early adulthood, with serious complications that include heart or respiratory-related problems. It mostly affects boys, about 1 in ever 3,500 to 5,000 male children.
There were reports last week that the FDA turned down Sarepta’s eteplirsen as well. In the case of last week’s story, it appears that the advisory committee did not recommend approval, but the final decision regarding the NDA will be decided by the committee, when its meeting will be rescheduled.
Early evaluation’s of Sarepta’s eteplirsen have criticized the trial design, efficacy, dystrophin measurement methods, and statistical analysis. In particular, Sarepta’s trial included only 12 patients. “It’s a different leap of faith you have to take with Sarepta,” Ira Loss, an analyst with Washington Analysis, told BloombergBusiness last week. “They do have patients who are still walking around after three years. The risk is you don’t have a lot of patients.”
Despite that, WBB Securities analyst Stephen Brozak told Reuters, “I’m not writing the Sarepta drug off yet.” It has been pointed out that the issues with Sarepta’s data doesn’t seem to be about whether the drug works—it appears to—but more about how well, how reliably, and what side effects there might be when the trial size was so small.
“I think there’s still a chance (of approval),” said Wedbush Securities analyst Heather Behanna to Reuters, “but a small chance.”
The rescheduled meeting will be announced in the Federal Register. The Prescription Drug User Fee Act (PDUFA) date is currently scheduled for Feb. 26.
Sarepta took a short hop this morning at the news, currently trading for $12.86 per share. Earlier in the day it traded for $9.50. Shares had a yearly high of $41.79 on Oct. 5, 2015, dropped to $24.06 on Oct. 30, then went back up to $38.67 on Dec. 30 before its most recent drop.
