The FDA plans to hold an advisory committee meeting to discuss Capricor Therapeutics’ application for deramiocel, which the agency rejected last July. The news surprised CEO Linda Marbán, who told BioSpace the FDA has not communicated any issues of concern with the company’s resubmitted application.
Linda Marbán was “surprised” when the FDA informed Capricor Therapeutics it would be holding an advisory committee meeting for its Duchenne muscular dystrophy cardiomyopathy cell therapy prior to its August target action date.
“We really thought that it’s a very clean data set,” the CEO told BioSpace from Orlando, where Capricor is presenting five-year data from the ongoing open-label extension study of deramiocel at the Parent Project Muscular Dystrophy (PPMD) conference. “We have not gotten any issues that they want to discuss.”
Marbán did offer a couple of possible theories for the adcomm—which comes after deramiocel aced a pivotal Phase 3 trial last December.
“We did hear that [acting FDA Commissioner] Kyle Diamantas . . . said he wanted more adcomms, and so we’re kind of wondering if this is sort of in response to that,” she said.
Indeed, Capricor was not the only recently rebuffed biotech on Friday to announce an advisory committee meeting for its new submission. Replimune revealed that the FDA plans to schedule an adcomm for its twice-rejected advanced melanoma therapy RP1 prior to an Aug. 2 decision date.
Another possible reason, Marbán posited, is the recent regulatory friction in the Duchenne muscular dystrophy (DMD) space more broadly.
Patients and families were thrown for a loop last year when the FDA temporarily requested that Sarepta Therapeutics halt shipments of its gene therapy Elevidys following the deaths of two nonambulatory patients with DMD. Sarepta initially stood its ground, refusing to stop all shipments of Elevidys, but ultimately complied. Just a week later, however, the FDA recommended that the voluntary hold be lifted for patients with DMD who are able to walk.
“There’s been so many issues around Duchenne and treatment for Duchenne and Sarepta, maybe they’re being extra careful,” Marbán said of the FDA.
Capricor would be forgiven having a sense of déjà vu.
Deramiocel was rejected by the regulator in July 2025 after then–Center for Biologics Evaluation and Research (CBER) director Vinay Prasad canceled a scheduled adcomm—unilaterally, according to reporting by STAT News.
In its complete response letter (CRL), the FDA said the data supporting deramiocel fell short of the “statutory requirement for substantial evidence of effectiveness.”
Marbán was blindsided by the adcomm’s cancelation, which came in June 2025. The meeting, which had been scheduled for July 30, was listed on the Federal Register—and then, ten minutes later, it wasn’t. “Investors watch these boards all day, every day, so my phone started blowing up with ‘Your adcomm has been canceled. It’s been taken down. Why?’ I had no answers to give,” Marbán told BioSpace last August. “I couldn’t make an official statement.”
In December 2025, Capricor’s fortunes began to look up when deramiocel hit both the primary and secondary endpoints in a pivotal trial. In the Phase 3 HOPE-3 trial, the cell therapy showed statistically significant benefits in upper-limb function, the study’s primary endpoint. It also slowed decline in cardiac function as measured by left ventricular ejection fraction, satisfying the key secondary endpoint.
In March, the FDA accepted a resubmission for deramiocel and set a target action date of Aug. 22.
A return to FDA normalcy?
With the successful Phase 3 results, Capricor may not be a perfect fit with the FDA’s recent trend of providing second chances to rare disease drug applications previously rejected or refused by the agency. But that’s not to say deramiocel won’t benefit from the FDA’s seemingly adjusted stance toward external controls.
In the past year, uniQure and REGENXBIO have had their gene therapy applications either discouraged or rejected, due in part to the use of externally controlled studies. Recently, however, the FDA has cleared uniQure to file for approval of AMT-130 for Huntington’s disease, and REGENXBIO to resubmit its application for its Hunter syndrome therapy, based on these very data.
The use of an externally controlled study was “one of the major issues” raised in Capricor’s July 2025 CRL, Marbán said during a BioSpace webinar earlier this year.
At the BIO International Convention in San Diego this week, former regulators cited the agency’s new investigational new drug pilot program as a sign that the FDA is returning to a state of normalcy. A return to adcomms could be another sign of this phenomenon.
On April 30, the agency ended a 9-month advisory committee drought when the Oncologic Drugs Advisory Committee met to discuss two cancer drug applications from AstraZeneca. Then, last week, Moderna’s mRNA-based flu vaccine—which drew considerable industry skepticism when the FDA refused in February to even review the company’s application—won unanimous support from the agency’s advisory panel for vaccines.
While these and other recent regulatory reversals following the May departure of former Commissioner Marty Makary have hinted at significant change at the FDA, Marbán is not convinced.
“I think everybody thought that we had Prasad leave and Makary leave, that it was going to be a clean house at FDA, and as a result of a clean house, everything was going to get better again,” she said. “I personally am not convinced of that at all.”
At CBER at least, Marbán said she believes the reviewers were “really clouded . . . by the Prasad etiology.”
But the CEO was clear that she is not concerned about deramiocel’s chances of approval.
While Capricor does not have a list of questions the FDA plans to discuss at the July adcomm, “We have the best key opinion leaders in the space that are going to be lined up to be presenting with us,” Marbán said, in addition to quality-of-life specialists and statisticians from around the globe.
“We have a randomized double blind placebo control trial. We hit the primary endpoint, we hit the secondary endpoints,” Marbán said. “I don’t know what their issues would be.”
