{"id":7030912,"date":"2026-08-06T22:14:37","date_gmt":"2026-08-06T22:14:37","guid":{"rendered":"https:\/\/peraltafinancing.com\/business\/legal\/part-1-lessons-from-the-meeting-on-capricors-deramiocel\/"},"modified":"2026-08-06T22:14:37","modified_gmt":"2026-08-06T22:14:37","slug":"part-1-lessons-from-the-meeting-on-capricors-deramiocel","status":"publish","type":"post","link":"https:\/\/fivemor.com\/?p=7030912","title":{"rendered":"Part 1; Lessons from the Meeting on Capricor\u2019s Deramiocel"},"content":{"rendered":"


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After more than a year with nary an adcomm (just 2 drug approval-related meetings in the Makary era), CBER\u2019s Cellular, Tissue, and Gene Therapies Advisory Committee meetings came back with a vengeance last week with back-to-back meetings to discuss two seemingly controversial cell and gene therapies that received complete response letters (CRLs) and are now under review following BLA resubmissions: Capricor\u2019s Deramiocel for DMD and Replimune\u2019s RP1 for advanced melanoma.<\/p>\n

Covered in Part 1 of this FDA Law Blog series is the July 29, 2026 meeting on Capricor Inc.\u2019s Deramiocel<\/a> for the treatment of cardiomyopathy in patients in Duchenne muscular dystrophy (DMD).\u00a0 Capricor previously submitted a BLA on December 31, 2024, based on results from a Phase 2 randomized trial (HOPE-2) and open label extension (HOPE-2-OLE). \u00a0FDA granted priority review but ultimately issued a CRL on July 9, 2025, finding a lack of substantial evidence of effectiveness. \u00a0At issue in the recent adcomm was Capricor\u2019s BLA resubmission, and whether the data from its Phase 3 randomized, double-blind, placebo-controlled HOPE-3 study provided substantial evidence of effectiveness. A 9 to 3 vote by the advisory committee opined that it did not, at least for the cardiomyopathy indication (versus the broader impact on skeletal muscle which was not asked directly).\u00a0 The PDUFA date for Capricor\u2019s BLA resubmission is set for August 22, 2026.<\/p>\n

The FDA\u2019s review principally focused on data analysis and the statistical analysis plan (SAP).\u00a0 They highlighted that decisions made on imputation, exclusion of certain patient data, and changing the primary endpoint from an absolute change to percent change, with key changes in primary endpoint and methodology made after the last subject visit.\u00a0 FDA further noted the potential for functional unblinding from the AE profile and cross-over into the open-label extension.<\/p>\n

Because of these concerns, in its briefing book and presentation, FDA relied on version 1.1 of the SAP, prior to when substantial data collection began (i.e., first subject, first visit), to analyze the data and found a lack of statistical significance.\u00a0 While shifting to version 1.1 of the SAP seems to be a draconian and overly conservative analysis of the data, particularly in a rare and devastating disease like DMD where unmet needs are substantial and regulatory flexibility is warranted, the advisory committee members appeared concerned by these issues.\u00a0 In their discussion, the committee members noted that whether statistical significance was reached appeared to hinge on the inclusion or exclusion of certain data or imputations from just a few patients \u2013 leading to what the committee members dubbed as data \u201cfragility.\u201d<\/p>\n

Without opining on the merits of FDA\u2019s assertions about Capricor\u2019s program, the critiques the agency raised provide important insights for sponsors:<\/p>\n