FDA Announces Advisory Committee for Capricor's DMD Therapy, Signaling Scrutiny Under New Commissioner

Capricor Therapeutics faced a significant setback as the U.S. Food and Drug Administration (FDA) announced plans for an advisory committee meeting to discuss the company's investigational cell therapy, deramiocel, for Duchenne muscular dystrophy (DMD). This development, likely to be the first such committee under newly appointed FDA Commissioner Marty Makary, M.D., sent Capricor's shares tumbling 13% by the end of trading on Monday.

Regulatory Hurdles for Deramiocel

The FDA's decision to convene an advisory committee (adcomm) for deramiocel comes ahead of the previously announced Prescription Drug User Fee Act (PDUFA) date of August 31. While no specific date has been set for the adcomm, it must occur before the PDUFA deadline. This move has raised concerns among investors, as FDA requests for advisory committees can sometimes be interpreted as a sign of heightened regulatory scrutiny.

Capricor CEO Linda Marbán, Ph.D., attempted to frame the development positively, stating, "The successful completion of our mid-cycle review meeting along with the upcoming advisory committee meeting represents major milestones on the path towards approval of deramiocel." She emphasized the company's preparedness for the meeting and their intention to highlight the "transformative potential of deramiocel in treating DMD-cardiomyopathy."

Clinical Data and Safety Concerns

Deramiocel, a next-generation cell therapy for DMD, is being evaluated for full approval in patients with DMD cardiomyopathy. The therapy's data comes from Capricor's phase 2 HOPE-2 and HOPE-2 open-label extension (OLE) trials. Last fall, the OLE trial demonstrated improvements in left ventricular ejection fraction and upper limb function in DMD patients after three years of treatment.

However, safety concerns have emerged from the trial data. All 13 patients in the OLE study experienced mild to moderate adverse events, with five patients experiencing severe or life-threatening events. Nine of these events were reportedly related to the treatment, according to Capricor's presentation in October.

Implications for the DMD Treatment Landscape

The FDA's decision to hold an adcomm for deramiocel is not entirely unexpected, given the agency's history with DMD therapies. The controversial approval of Sarepta's Exondys 51 in 2016 and the subsequent approval of Vyondys 53 in 2019 have made DMD a contentious area for the FDA. These past decisions have led to internal disagreements within the agency and divergent opinions between U.S. and European regulators.

As the first drug expected to face an FDA advisory committee under the new commissioner, deramiocel's review may set the tone for future evaluations of novel therapies in rare diseases. The outcome of this process could have far-reaching implications for both Capricor and the broader landscape of DMD treatment development.