Capricor Announces Big Steps Forward In Duchenne Muscular Dystrophy Program With Key Regulatory Updates

Capricor Therapeutics, a biotechnology company focused on developing cell and exosome-based treatments for rare diseases, announced the successful completion of the U.S. Food and Drug Administration’s (FDA) Pre-License Inspection (PLI) of its San Diego manufacturing facility. The inspection was conducted as part of the regulatory review for Deramiocel, Capricor’s lead cell therapy candidate currently under FDA priority review for the treatment of Duchenne Muscular Dystrophy (DMD).

The inspection concluded with the issuance of a Form 483, which included several observations related primarily to standard quality systems and documentation practices. Capricor has submitted its formal responses to the FDA, and importantly, none of the observations required significant changes to the current good manufacturing practices (cGMP) or the facility itself. The company has expressed confidence that the facility will meet all regulatory requirements necessary to support the licensure and eventual commercial launch of Deramiocel, pending FDA approval.

Linda Marbán, Ph.D., Capricor’s Chief Executive Officer, said in a statement, “This inspection outcome is a major regulatory milestone, particularly in a field where standards are exceptionally high. It reflects the strength of our manufacturing capabilities and positions us well as we advance toward potential approval. With the FDA Advisory Committee meeting now scheduled, we look forward to the opportunity to present the totality of evidence supporting the approval of Deramiocel for the treatment of Duchenne muscular dystrophy. With all key review activities progressing on track, we remain focused on delivering this much-needed therapy to the Duchenne community.”

In addition to the inspection update, Capricor shared that the FDA has communicated its intent to hold an Advisory Committee meeting on July 30, 2025, though this date has not yet been confirmed. A late-cycle meeting with the agency is scheduled for mid-July 2025. According to Capricor, no significant issues or major deficiencies were identified during the mid-cycle review. The Biologics License Application (BLA) for Deramiocel continues to progress under priority review, with a Prescription Drug User Fee Act (PDUFA) target action date set for August 31, 2025.