Capricor cited in FDA plant inspection

 

• U.S. FDA successfully completed Pre-License Inspection; Company expects facility will meet all requirements to support licensure
• Advisory Committee meeting scheduled for July 30, 2025
• Mid-cycle meeting recently completed with no significant issues or major deficiencies; late-cycle meeting planned for mid-July
• Biologics License Application remains under priority review with PDUFA target action date of August 31, 2025

Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced the successful completion of the U.S. Food and Drug Administration’s (FDA) Pre-License Inspection (PLI) of its San Diego manufacturing facility for Deramiocel, the Company’s lead cell therapy candidate with a Biologics License Application (BLA) under FDA review for potential approval in the treatment of Duchenne Muscular Dystrophy (DMD). The inspection concluded with a Form 483 containing several observations. The Company has submitted its responses to the FDA, none of which required material changes to the cGMP process or facility. The observations were primarily related to routine quality systems and documentation practices. The Company is confident that the facility will meet the necessary requirements to support product licensure and, pending approval, commercial launch.

“This inspection outcome is a major regulatory milestone, particularly in a field where standards are exceptionally high,” said Linda Marbán, Ph.D., Capricor’s Chief Executive Officer. “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.”

The FDA informed Capricor of its intent to hold the Advisory Committee meeting on July 30, 2025, although that date is pending confirmation by the FDA. At the time of the mid-cycle review, no significant issues or major deficiencies were noted. A late-cycle meeting is planned for mid-July 2025. The BLA for Deramiocel remains under priority review with a Prescription Drug User Fee Act (PDUFA) action date of August 31, 2025.

https://www.globenewswire.com/news-release/2025/06/11/3097631/0/en/Capricor-Therapeutics-Announces-Key-Regulatory-Updates-for-its-Duchenne-Muscular-Dystrophy-Program.html