Deramiocel

iStock, Olena Koliesnik Capricor Therapeutics’ deramiocel was rejected in July 2025, potentially caught between Nicole Verdun, a former top biologics regulator at the FDA, and Vinay Prasad, director of the Center for Biologics Evaluation and Research. Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy is back on track after the FDA announced that it will once again review the biotech’s application package. The agency has lifted its previous complete response letter on the cell therapy, dubbed deramiocel, Capricor revealed in a news release on Tuesday . The biotech is seeking full approval for deramiocel, for which the FDA’s verdict is now expected on Aug. 22. The agency has yet to identify any potential review issues that deramiocel could encounter, according to Capricor. Capricor’s shares were up 9% to $33.40 as of market close Tuesday after the announcement. Composed of cardiosphere-derived cells—a rare subset of cells from the heart that can modulate the immune response and reduce fibrosis—deramiocel is an allogenic cell therapy that helps preserve the function of cardiac and skeletal muscles. Capricor tested the therapy in the Phase 2 HOPE-2 study, and in May 2020 reported that it elicited meaningful improvements in upper limb function in patients with Duchenne muscular dystrophy (DMD). HOPE-2 became the basis of Capricor’s initial approval package for deramiocel, which the FDA turned down in July 2025 claiming that the submission lacked the “statutory requirement for substantial evidence of effectiveness.” At the time, observers suggested that deramiocel might have been caught in the crossfire between two top figures in the FDA: Nicole Verdun, former top regulator of cell and gene therapies at the Center for Biologics Evaluation and Research (CBER), who wanted to hold an advisory committee meeting for the asset, and Vinay Prasad, current director of the Center for Drug Evaluation and Research (CDER), who unilaterally canceled the meeting. Verdun was ultimately pushed out of the agency in June 2025. Duchenne muscular dystrophy Capricor Heads Back to FDA With Pivotal Results for DMD Cardiomyopathy Therapy Six months after receiving a surprise rejection due to what the FDA called “lack of substantial evidence of effectiveness,” Capricor’s cell therapy deramiocel showed significant benefits in upper-limb function and slowed decline in cardiac function in a Phase III trial. December 3, 2025 · 3 min read · Heather McKenzie Read more Prasad is now once again set to leave the FDA by the end of April, a move that analysts say could be a tailwind for the rare disease space—including Capricor. In December 2025, Capricor resubmitted the drug package for deramiocel, beefing it up with data from the Phase 3 HOPE-3 trial. The study found that patients given the cell therapy saw significant improvements in upper-limb function and cardiac function compared with those on placebo. Deramiocel also slowed skeletal muscle disease progression. If approved, Capricor could receive a priority review voucher under the FDA’s rare pediatric disease program, the biotech said on Tuesday. FDA ‘Unprecedented’ FDA Leaks Sow Confusion for Patients, Sarepta and Capricor Sarepta and Capricor learned of key regulatory decisions from the media and investors, and Duchenne muscular dystrophy families have turned to the news for answers. Meanwhile, the FDA insists it remains committed to notifying companies of any regulatory action before sharing information with the media or public. August 4, 2025 · 7 min read · Heather McKenzie Read more

After receiving a surprise rejection last year, Capricor Therapeutics' Duchenne muscular dystrophy (DMD) cell therapy seems back on track for a potential FDA approval. The biotech said Tuesday that the FDA has lifted its complete response letter (CRL) and is resuming review of its application for deramiocel to treat cardiomyopathy associated with DMD. Deemed a Class 2 resubmission, the cell therapy now has a PDUFA date of Aug. 22.Capricor was one of several cell and gene therapy makers in the last 12 months to have an biologic application rebuffed by the regulator in a decision seemingly inconsistent with earlier feedback (see – Vital Signs: The FDA's 2025 report card).Its BLA for deramiocel was knocked back last July after the FDA said the application lacked "substantial evidence of effectiveness." Following a Type A meeting, the agency agreed that data from the pivotal Phase III HOPE-3 study would be sufficient to address the concerns it raised in the CRL. And proving to be a good decision on Capricor's part, HOPE-3's December readout showed that deramiocel significantly improved both skeletal and cardiac outcomes in patients with DMD (see – KOL Views Q&A: Capricor’s deramiocel has doubts to assuage but DMD cell therapy offers intriguing commercial profile)."We believe the positive HOPE-3 results and broader clinical evidence reinforce deramiocel's potential to become a first-in-class therapy for Duchenne muscular dystrophy, with the opportunity to address both skeletal and cardiac manifestations of the disease," said CEO Linda Marbán in a company release. If the cell therapy is approved, Capricor expects to receive a priority review voucher.While the PDUFA date is a positive sign for the biotech, even if deramiocel is approved, it will come about a year after the candidate was originally expected to receive marketing clearance. Prasad's exitBut with the upcoming departure of Vinay Prasad from the FDA, investors seem more confident that the days of regulatory uncertainty at the agency are behind them. After the announcement that Prasad would step down from his role as director of the FDA's Center for Biologics Evaluation and Research (CBER), several cell and gene therapy developers who had received snubs from the agency during his tenure saw their share price rise, including Capricor. The biotech gained about 10% on the news Monday, and added an additional 11% on Tuesday (see – Vital Signs: PDUFAs to watch during CBER shakedown).Some of the biggest gains went to uniQure and Regenxbio, which added 26% and 20%, respectively, after Prasad's exit was disclosed late last Friday. The former was caught up in a regulatory storm after the FDA reiterated it would need to conduct a sham surgery-controlled trial of its Huntington's disease gene therapy, prompting ethical concerns. Regenxbio received a CRL for its Hunter syndrome gene therapy after the FDA questioned the appropriateness of its selected biomarker as a surrogate endpoint reasonably likely to predict clinical benefit, despite previously agreeing to its use (see – Vital Signs: Is the FDA making gene therapy implausible?).