The U.S. Food and Drug Administration (FDA) has declined to approve Capricor Therapeutics’ cell therapy, Deramiocel, for treating cardiomyopathy in patients with Duchenne muscular dystrophy (DMD). The rejection, delivered through a “complete response letter,” stated that the evidence submitted did not meet the agency’s efficacy standards.
As a result, Capricor’s shares plummeted by 30% in early trading, reflecting investor disappointment. The company was aiming to treat the heart muscle disease often associated with DMD, a genetic disorder that causes progressive muscle degeneration.
Regulatory Shake-Up and Early FDA Decision Challenge Capricor’s Approval Timeline Plans
The FDA’s decision came unexpectedly, more than a month ahead of the scheduled priority review date of August 31. Capricor now plans to resubmit its application in the third quarter of the year, incorporating data from an ongoing late-stage trial.
This move suggests the company remains committed to pursuing approval despite the regulatory hurdle. The early decision underscores heightened scrutiny under the current leadership at the FDA’s Center for Biologics Evaluation and Research (CBER), which oversees cell therapy approvals.
FDA Rejects Capricor’s Duchenne Therapy Deramiocel, Citing Efficacy Concerns and Sparking Stock PlungeAnalysts have pointed to broader changes within the regulatory environment as a contributing factor to the rejection. JonesTrading analyst Catherine Novack had previously suggested that an August approval was unlikely, citing the cancellation of an advisory committee meeting and internal leadership changes at CBER.
The recent appointment of Vinay Prasad as CBER chief has raised concerns among industry observers, as he has expressed strong opposition to single-arm trials and the FDA’s accelerated approval pathway—both common practices in cell therapy development.
FDA Leaves Pathway Open as Capricor Plans Strategic Response to Rejection
Capricor’s stock has been particularly sensitive to regulatory developments, with Friday’s sharp decline highlighting investor wariness. Analysts at B. Riley Securities noted that while the FDA raised technical concerns with the current data, it has left the door open for approval by permitting a major amendment submission rather than a full biologics license application (BLA) restart.
This distinction provides a pathway forward and suggests the agency is open to further evidence rather than issuing a complete denial.
In response to the rejection, Capricor expressed surprise and announced plans to seek a meeting with the FDA to clarify next steps. CEO Linda Marban emphasized that the company does not believe a new BLA will be necessary and remains optimistic about incorporating updated clinical trial results.
The company’s ongoing engagement with regulators and its plan to leverage additional data reflect a determination to address the agency’s concerns and continue pursuing market entry for Deramiocel.
