Capricor shares turn red as FDA plans deramiocel adcomm, likely the first under new commissioner

# Capricor's Deramiocel FDA Review:
Market Reaction to Advisory Committee Plans

Market Response and FDA Development

Capricor Therapeutics experienced a significant market downturn, with shares falling 13% by the end of trading on Monday following the announcement that its investigational cell therapy deramiocel would face an FDA advisory committee meeting. This committee review is notable as it appears to be the first drug expected to undergo this process under the FDA's new commissioner, Dr. Marty Makary.

Despite the negative market reaction, Capricor recently completed its mid-cycle review meeting with the FDA regarding its Biologics License Application (BLA) for deramiocel, with the agency confirming that no significant deficiencies have been identified in the review process1. The company remains on track for its previously established Prescription Drug User Fee Act (PDUFA) target action date of August 31, 20251.

## Deramiocel:
A Potential Breakthrough Treatment

Deramiocel is being developed as a first-in-class treatment for Duchenne muscular dystrophy (DMD) cardiomyopathy, a condition for which there are currently no approved therapies4. If approved, it would be administered quarterly as a lifelong treatment and has the potential to be widely adopted across the DMD-cardiomyopathy treatment landscape4.

The FDA granted priority review status to deramiocel's application in March 2025, shortening the standard review period from 10 months to six months, indicating the therapy's potential to provide significant benefits in treating this serious condition4. The BLA was originally submitted to the FDA in January 20255.

Commercial and Financial Outlook

If approved, deramiocel would represent a significant milestone for Capricor Therapeutics. The company has already received a $10 million milestone payment from Nippon Shinyaku and reportedly maintains a strong cash balance of approximately $152 million, which is expected to support operations until 20273.

Capricor is actively preparing for the commercial launch of deramiocel while also expanding its manufacturing capabilities in anticipation of regulatory approval in both the U.S. and Europe, where the therapy has received Orphan Drug and Advanced Therapy designations from the European Medicines Agency3.

Sources:

1. https://www.capricor.com/investors/news-events/press-releases/detail/311/capricor-therapeutics-announces-completion-of-mid-cycle

3. https://www.nasdaq.com/articles/capricor-therapeutics-announces-fda-acceptance-biologics-license-application-deramiocel

4. https://musculardystrophynews.com/news/fda-priority-review-granted-deramiocel-dmd-heart-disease/

5. https://www.biospace.com/press-releases/capricor-therapeutics-completes-submission-of-biologics-license-application-to-the-u-s-fda-for-deramiocel-for-the-treatment-of-duchenne-muscular-dystrophy