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FDA Panel Says BioMarin DMD Drug Data Isn’t Persuasive Enough

Statistical significance in the late-stage study is not convincing

A panel of outside advisers to the FDA has indicated that efficacy data from BioMarin Pharmaceutical Inc. on its experimental drug for treating a muscle wastage disorder was not persuasive enough to warrant approval.

According to Reuters, the FDA panel did not officially vote on an approval of the drug, but 15 out of 17 members were of the opinion that the lack of statistical significance in BioMarin's late-stage study weakened the findings from two earlier studies. The drug, drisapersen, is being developed to treat Duchenne muscular dystrophy (DMD), a disease that affects one in 3,600 newborn boys and causes rapid muscle degeneration.

The FDA is expected to decide on the drug by December 27 after considering the panel's comments.

The FDA staff had raised concerns about the drug that included potentially fatal long-term side effects. "I don't think they [BioMarin) will necessarily be able to convince the FDA staff to change their mind," said Heather Behanna, an analyst at Wedbush Securities, noting that additional data on the drug could help BioMarin's case. But RBC Capital Markets analysts said the health regulator could still find BioMarin's drug approvable given overall risks and benefits.

The panel focused on the data and its interpretation after hearing 26 speakers, mostly parents who showed videos of their boys benefiting from drisapersen. Only one of the parents said the side effects were not manageable.

BioMarin's strategy going forward could be talking with the FDA about what else it could do, some analysts noted.

Rival drugmaker Sarepta Therapeutics Inc. is also developing a DMD treatment. The drug eteplirsen, like BioMarin's drisapersen, skips a faulty section of the gene to produce dystrophin, the lack of which causes DMD.

"Sarepta's review will also be tough. Their long-term prospects because of better safety data are pretty good," Wedbush's Behanna said. The fate of Sarepta's drug, which will go through a similar vote in January, will depend on how the FDA looks at dystrophin levels as a biological indicator for DMD, Behanna added.

Source: Reuters, November 25, 2015.

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