• 14 Jul 2025
• Admin
• News Article

FDA turns back Capricor’s Duchenne cell therapy

Overview

After scheduling, then canceling an advisory committee meeting, the agency rejected deramiocel for Duchenne-related cardiomyopathy, arguing Capricor needs more data to prove its effectiveness. The Food and Drug Administration has rejected what was set to be the first treatment for heart-related complications of Duchenne muscular dystrophy, indicating that its developer, Capricor Therapeutics, didn’t prove the treatment’s effectiveness in clinical testing.

FDA Rejects Capricor’s Deramiocel, Requests More Data and Manufacturing Fixes

• Capricor on Friday said the agency turned back the drug, deramiocel, because the company’s application didn’t meet the FDA threshold for “substantial evidence of effectiveness” and needs “additional clinical data.” The regulator’s complete response letter also referenced manufacturing issues, though Capricor claimed it has addressed most of the problems in comments the regulator has not yet reviewed.

• The FDA granted Capricor a meeting to discuss a path forward for deramiocel, but will “restart the review clock” following a resubmission. This process can take between six to 10 months.

Statement from CEO Linda Marban said in a statement

• “We are surprised by this decision by the FDA,” CEO Linda Marban said in a statement. Marban added that Capricor followed the FDA’s guidance throughout the regulatory process and noted how, prior to the rejection, the review had advanced “without major issues.” The company believes results from an ongoing, placebo-controlled Phase 3 study of about 100 patients could resolve the agency’s questions. Summary data from that trial are expected in the third quarter.

• “While this was an unexpected decision by the FDA, we remain committed to the [Duchenne] community to get deramiocel through the approval process,” Marban said.

Company shares fell substantially in pre-market trading Friday.

FDA Setback Stalls Capricor’s Bid for First Duchenne Cardiomyopathy Treatment
The setback is the latest twist in an unusual regulatory journey. Capricor hoped deramiocel would become the first treatment for Duchenne-related cardiomyopathy, a progressive weakening of the heart muscle that’s the leading cause of death in people with the disease.

Deramiocel Shows Promise in Small Trial, But FDA Seeks More Evidence

• Deramiocel is derived from a special type of heart cell known to help preserve function. 

• Phase 2 testing showed treatment improved measures of upper limb function and the heart’s ability to pump blood. 

• But the results came from a tiny trial of 20 participants, only eight of whom received therapy. Capricor also leaned on data from a historical study of heart function in Duchenne patients to support its case.

Regulatory Shake-Up Adds Uncertainty to Capricor’s Deramiocel Rejection

• The combined results made for an ambiguous case at a time when FDA leadership was in flux. 

• Since the agency accepted Capricor’s approval application in March, multiple officials who championed regulatory flexibility for gene and cell therapies were forced out. 

• Vinay Prasad, who has criticized another Duchenne treatment with imperfect data, Sarepta Therapeutics’ Elevidys, now runs the office that regulates such medicines.

The FDA had scheduled an advisory committee meeting to help it review Capricor’s application, but then canceled its request, which Stat News reported was due to Prasad’s negative view of deramiocel.