FDA reviewers question efficacy of BioMarin's DMD therapy

In briefing documents released ahead of a Nov. 24 advisory committee meeting, FDA reviewers expressed doubts that Kyndrisa drisapersen from BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is effective to treat Duchenne muscular dystrophy (DMD). The reviewers intimated that the agency will use the meeting of the Peripheral and Central Nervous System Drugs Advisory Committee to get panelists' critical appraisal of available efficacy data rather than recommendations on approvability.

"The current thinking of the primary review team is that evidence supporting the effectiveness of drisapersen is inconsistent," wrote Division of Neurology Products Clinical Team Leader Ronald Farkas in an introductory memo to the committee. "The decision about approvability is necessarily step-wise, requiring first that the drug be found by FDA to be effective prior to any consideration of benefit-risk." ...