The evidence supporting BioMarin's application for drisapersen is weak and that the drug doesn't appear to be effective in Duchenne muscular dystrophy (DMD).

‘FDA advisory panel concluded that a new drug from BioMarin Pharmaceutical Inc. hasn’t 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.
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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.
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Source-Medindia