A panel of outside advisers to the U.S. Food and Drug Administration indicated that efficacy data from BioMarin Pharmaceutical Inc (BMRN.O) on its experimental drug for treating a muscle wastage disorder was not persuasive enough to warrant approval to treat the fatal disease called Duchenne muscular dystrophy.
Duchenne muscular dystrophy is a disorder occurring in about 1 in 3,500 boys. It is often fatal by the time the boy reaches his 20s or early 30s. It is typically first noticeable in children from 3 to 5 years old, with muscle weakness becoming progressive. Children with Duchenne begin to lose their ability to walk in their teens, and can lose respiratory function and begin to have severe cardiac problems.
‘FDA advisory panel concluded that a new drug from BioMarin Pharmaceutical Inc. hasnt been proven to effectively treat the fatal disease called Duchenne muscular dystrophy.’
The drug, drisapersen, is being developed to treat Duchenne muscular dystrophy (DMD). The FDA will decide on the approval of the drug by Dec. 27, after considering the panel's comments.
Efficacy has not been established," said Glen Nuckolls of the National Institutes of Health. But committee Chairman G. Caleb Alexander, an associate professor of epidemiology and medicine at the Johns Hopkins Bloomberg School of Public Health, said, "There was some suggestion that the drug could be helpful in some individuals."
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.
The panel 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.
During a final discussion of the totality of the evidence, some panelists noted that DMD involves significant heterogeneity, and that the drug may have benefits for some individuals -- getting a better understanding of which patients may stand to benefit more could be helpful.
Drisapersen is the first of two novel drugs for DMD that are before the FDA. Many had expected eteplirsen, developed by Sarepta Therapeutics, to be evaluated in conjunction with drisapersen, but that drug will be evaluated separately in January.