STAT+: Ahead of hearing, FDA questions the efficacy of Capricor’s Duchenne drug

STAT PlusBreaking News Ahead of hearing, FDA questions the efficacy of Capricor’s Duchenne drug Capricor had previously said its drug, called deramiocel, hit its targets in a Phase 3 study Manage alerts for this article Email this article Share this article By Jason MastJuly 27, 2026 General Assignment Reporter Jason Mast[email protected]Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77. The Food and Drug Administration said Monday that Capricor Therapeutics’ stem cell treatment for Duchenne muscular dystrophy did not meet the objectives of Phase 3 trial — contrary to the company’s claims last year. Capricor said in December that the drug, known as deramiocel, met both the primary and secondary endpoints in a large, randomized study. It was a striking result in a fatal, childhood disease that has proven stubbornly difficult to treat, despite immense advances in genetic medicine. Advertisement The data were also notable for coming primarily in teenagers and young men who have already lost the ability to walk, a population with few options. The drug appeared to both preserve their upper-arm function and stave off the heart failure most patients eventually experience, Capricor had said. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts. Already have an account? Log in Monthly $39 Totals $468 per year $39/month Get Started Totals $468 per year Starter $30 for 3 months, then $399/year $30 for 3 months Get Started Then $399/year Annual $399 Save 15% $399/year Get Started Save 15% 11+ Users Custom Savings start at 25%! Request A Quote Request A Quote Savings start at 25%! 2-10 Users $300 Annually per user $300/year Get Started $300 Annually per user View All Plans To read the rest of this story subscribe to STAT+. Subscribe Log In biotechnology, drug development, rare disease, STAT+ Submit a correction requestReprints Jason Mast General Assignment Reporter Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77. Newsletter Understand how science, health policy, and medicine shape the world every day Recommended Biotech July 27, 2026 STAT Plus: RA Capital backs upstart rival to Ultragenyx and Ionis in rare brain disease Biotech July 27, 2026 STAT Plus: Argenx to buy Forte Biosciences for $2.2B Advertisement Health July 24, 2026 FDA advisory panel narrowly rejects compounding of one peptide, backs two others The Readout July 24, 2026 STAT Plus: Fatal gene-editing trial in China raises ethics and oversight questions Biotech July 23, 2026 STAT Plus: In win for RFK Jr., FDA advisory panel narrowly votes to allow compounding of unapproved peptides Subscriber Picks