Capricor's Lead Duchenne Therapy Hits FDA Regulatory Roadblock

Capricor Therapeutics Inc. (NASDAQ:CAPR) stock is trading lower on Friday.

The company received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its Biologics License Application (BLA) for Deramiocel, the company’s lead cell therapy candidate for cardiomyopathy associated with Duchenne muscular dystrophy (DMD).

In the CRL, the FDA stated that it had completed its application review but could not approve the BLA in its current form.

The agency specifically cited that the BLA does not meet the statutory requirement for substantial evidence of effectiveness and that additional clinical data is needed.

Also Read: Capricor Reports 4-Year Data For Deramiocel, Shows Sustained Benefit In Duchenne Patients

The CRL also referenced certain outstanding items in the application’s Chemistry, Manufacturing and Controls (CMC) section, most of which Capricor says it has addressed in prior communications to the FDA.

However, the FDA did not review these materials due to the timing of the CRL issuance.

The FDA confirmed that it will restart the review clock upon resubmission. In addition, the agency offered the company the opportunity to request a Type A meeting to discuss the path forward.

Capricor plans to engage further with the FDA to determine the appropriate next steps.

Capricor’s BLA for Deramiocel was granted Priority Review in March 2025, supported by data from the HOPE-2 trial, its open-label extension (OLE), and natural history comparisons from FDA-funded datasets.

“We are surprised by this decision by the FDA. We have followed their guidance throughout the process. Prior to the CRL, the review had advanced without major issues, including a successful pre-licensure inspection and completion of the mid-cycle review,” said Linda Marbán, CEO of Capricor. “Capricor plans to submit data from the Phase 3 HOPE-3 clinical trial to provide additional evidence of effectiveness from an adequate and well-controlled study. The HOPE-3 trial is a randomized, double-blind, placebo-controlled clinical trial of 104 patients, with topline results expected in the third quarter of 2025.”

Capricor Therapeutics provided regulatory updates on its Biologics License Application (BLA) for Deramiocel in June.

The company has been informed that an Advisory Committee meeting has not yet been scheduled.

Price Action: CAPR stock is down 37.4% at $7.14 during the premarket session at the last check on Friday.

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