Capricor hit with Form 483 following FDA inspection at San Diego manufacturing facility

Capricor Therapeutics announced key regulatory updates for its Duchenne muscular dystrophy program, including the completion of a pre-license inspection (PLI) by the FDA for its San Diego manufacturing facility supporting deramiocel.

The inspection resulted in a Form 483 with several observations related to documentation and quality systems. Capricor has submitted responses, none of which required material changes to the cGMP process or facility, and expects the site will meet FDA requirements for licensure and, if approved, commercial launch.

“This inspection outcome is a major regulatory milestone, particularly in a field where standards are exceptionally high,” Capricor CEO Linda Marbán said in a statement. “It reflects the strength of our manufacturing capabilities and positions us well as we advance toward potential approval.”

The FDA has tentatively scheduled an advisory committee meeting for July 30 to evaluate the Biologics License Application (BLA) for deramiocel. A mid-cycle review was recently completed with no significant issues reported, and a late-cycle meeting is planned for mid-July. The BLA remains under priority review, with a PDUFA target action date of August 31.

Deramiocel (CAP-1002) is an allogeneic cell therapy composed of cardiosphere-derived cells, designed to preserve muscle function in patients with DMD. It has received RMAT, Orphan Drug, and rare pediatric disease designations in the U.S., and ATMP designation in Europe.

Capricor has partnered with Nippon Shinyaku for exclusive commercialization and distribution of deramiocel in the U.S. and Japan, pending regulatory approval.