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FDA panel says BioMarin DMD drug data not persuasive enough

Written by: | | Dated: Tuesday, November 24th, 2015

A panel of outside advisers to the U.S. Food and Drug Administration 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.

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 will decide on the approval of the drug by Dec. 27, after considering the panel’s comments.

The vote comes after FDA staff on Friday 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,” Heather Behanna, an analyst at Wedbush Securities said, noting that additional data on the drug could help making 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 on Tuesday focused on the data and its interpretation after hearing out 26 speakers, mostly parents who showed videos of their boys benefiting from taking drisapersen.

Only one of the parents said the side effects were not manageable.

BioMarin’s strategy going forward could be discussing with the FDA on what else it could do, some analysts noted, after the health regulator’s staff on Friday posted their preliminary review.

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.

BioMarin’s stock was placed on halt for all of Tuesday.

Sarepta shares were down about 1 percent at $36.98 after the bell while shares of PTC Therapeutics Inc, which is also developing a treatment for DMD, fell 5 percent in after-hours trading to $30.



(Reporting by Amrutha Penumudi and Vidya L Nathan in Bengaluru; , editing by G Crosse and Anil D’Silva)

Source: Reuters Health

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