BioMarin's Duchenne MD drug fails to gain support from US FDA reviewers

The reviewers at the US Food and Drug Association (FDA) are not convinced by the data for biotech company BioMarin Pharmaceuticals’ (Nasdaq: BMRN) drisapersen, to treat Duchenne muscular dystrophy (DMD) and said the clinical trial data does not show substantial evidence necessary for approval.

The staffers at the regulatory body said it is “greatly concerning” that a number of biomarker studies suggest that, contrary to initial published reports, drisapersen has little effect on increasing dystrophin levels, the putative mechanism of action, in the briefing statement. The staff also raised concerns over the safety of the treatment.

The reviewers said the study encountered severe toxicity across many organ systems in the non-clinical studies, and appeared to predict a number of the adverse events that were subsequently observed in the clinical studies.