At Duchenne Panel, Parents Plead With Experts, FDA Decision Looms
Summary
Instead, panelists were asked to assess the strength of the evidence accrued in the clinical trials of drisapersen, a Duchenne drug from San Rafael, CA-based BioMarin Pharmaceutical (NASDAQ: BMRN).The panelists did so, and their commentary did not seem favorable, which stood out all the more because of the emotional pleas for the drug’s approval that came from many of the parents of Duchenne patients—and the patients themselves—who showed up at the meeting in Silver Spring, MD.Justin Zivin, a professor from UC San Diego, noted that after reading the FDA’s briefing materials on drisapersen, “it was clear that this wasn’t going to get approved at this point.The drisapersen panel is as close as they’ve been to an FDA approved therapy, and many parents—and some young patients themselves—came to the hearing to make an impassioned plea for the drug before the panelists.Today’s meeting on drisapersen will also likely affect the fortune of a rival drug from Sarepta Therapeutics (NASDAQ: SRPT), eteplirsen, that will be the subject of an FDA advisory panel in January.