The Food and Drug Administration has canceled an advisory committee meeting for Capricor Therapeutics' cell therapy for Duchenne muscular dystrophy, marking a significant setback for the company amid a broader leadership shake-up at the agency's biologics division.
Vinay Prasad, director of the FDA's Center for Biologics Evaluation and Research (CBER), made the unilateral decision to cancel the meeting due to skepticism about the treatment's efficacy, according to sources familiar with the matter. The cancellation came immediately before the ouster of Nicole Verdun, the FDA's chief regulator of cell and gene therapies, who had originally scheduled the advisory committee review.
Market Impact and Investor Response
The regulatory uncertainty has taken a severe toll on Capricor's stock price, with shares falling $4.72, or 40%, to $7.22 in afternoon trading following the announcement. The dramatic decline reflects investor concerns about the therapy's regulatory pathway and the company's prospects for approval.
Leadership Tensions at FDA
The cancellation of Capricor's advisory committee meeting appears to be connected to broader tensions within the FDA's biologics division. Sources indicate that there had been long-simmering disagreements over Verdun's management style, which may have contributed to her placement on administrative leave.
The timing of events suggests a direct correlation between the disagreement over the Duchenne therapy review and Verdun's removal from her position. Verdun had been serving as the chief regulator overseeing cell and gene therapies, a rapidly growing and critically important therapeutic area.
Implications for Cell and Gene Therapy Regulation
The leadership change and canceled meeting raise broader questions about the FDA's approach to evaluating innovative cell and gene therapies. The disagreement between senior FDA officials over the review process highlights potential internal conflicts regarding regulatory standards and approval pathways for these emerging treatments.
The cancellation of the advisory committee meeting represents a significant procedural step backward for Capricor, as these meetings typically provide crucial external expert input on the risk-benefit profile of investigational therapies. Without this external review, the company faces increased uncertainty about its regulatory timeline and approval prospects.
