synopsis

The health agency has accepted Capricor's Biologics License Application for full approval of deramiocel as a treatment for Duchenne muscular dystrophy cardiomyopathy.

Shares of Capricor Therapeutics (CAPR) closed Tuesday with modest gains and rose more than 3% in extended trading after the company announced a key regulatory milestone for its investigational therapy, deramiocel.

The U.S. Food and Drug Administration has accepted Capricor’s Biologics License Application (BLA) for full approval of deramiocel as a treatment for Duchenne muscular dystrophy (DMD) cardiomyopathy. 

The agency also granted the application Priority Review, setting a Prescription Drug User Fee Act (PDUFA) target action date of August 31, 2025. 

Capricor noted the FDA had not yet identified any review issues with the application.

The BLA submission is backed by cardiac data from Capricor’s earlier trials, in which results were compared to natural history data from an FDA-funded study on DMD cardiomyopathy and related biomarkers. 

The FDA has yet to decide whether an Advisory Committee meeting will be necessary for the review.

The Priority Review designation shortens the standard review period from 10 months to six, potentially accelerating the therapy’s path to market.  

The company highlighted that this was a rolling BLA submission, meaning Capricor had multiple interactions with the FDA throughout 2024. 

A rolling submission allows regulators to begin reviewing sections of the application before final submission, which could further expedite the decision-making process.

Retail sentiment on Stocktwits turned ‘extremely bullish’ following the announcement, with message volume surging 300% by the end of Tuesday’s session. 

Early results from a poll on the platform show 84% of retail investors expect a full approval of the drug. 

CAPR poll sentiment and message volume as on March 4 | source: Stocktwits

One user emphasized the significance of the rolling BLA process, noting that Capricor had met with the FDA nine times last year and submitted the first module of the application in October 2024.

Another user expressed surprise that the stock did not react more strongly to the news but anticipated further movement as Capricor prepares to release its fourth-quarter earnings. 

In January, Capricor announced it would receive a $10 million milestone payment from its distribution partner, Nippon Shinyaku Co., under their U.S. commercialization and distribution agreement for deramiocel.

Capricor shares are down more than 2% year-to-date but have surged nearly 190% over the past 12 months.

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