Capricor Pivots to Skeletal Muscle Indication, Amending Deramiocel BLA After 9-3 FDA Advisory Panel Rejection
Following a major regulatory setback, Capricor Therapeutics has announced a dramatic pivot in its strategy for deramiocel, its investigational cell therapy for Duchenne muscular dystrophy (DMD). On July 29, 2026, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 9 to 3 against the efficacy of deramiocel for treating cardiomyopathy in DMD, citing "fragile" efficacy data and study design issues.
The regulatory case for cardiomyopathy was further undermined by a statistical correction discovered during peer review with The Lancet and the FDA. Reverting to the pre-specified statistical analysis plan version 3.0 shifted the treatment difference for the left ventricular ejection fraction (LVEF) endpoint in all patients from a statistically significant p=0.04 to a non-significant p=0.09.
On August 13, 2026, during its Q2 2026 earnings call, Capricor announced that it is actively working with the FDA to amend its Biologics License Application (BLA). Instead of cardiomyopathy, the amended application will focus on a refined upper limb skeletal muscle indication, which was the primary endpoint of the HOPE-3 study and remains statistically significant. The amendment will incorporate 24-month open-label extension data and additional analyses. The FDA has indicated a willingness to review this amendment, which will formally extend the PDUFA target action date.
To preserve capital during this extended review period, Capricor has placed all non-deramiocel pipeline programs on hold. Additionally, its ongoing contractual dispute with NS Pharma over U.S. pricing has been referred to arbitration, which is scheduled to begin in fall 2026.
Verbatim Quotes
- Linda Marban, CEO, on the statistical model correction:
"Linda Marban, CEO, explained that an issue with the statistical model in the clinical study report was identified during peer review with The Lancet and the FDA. The company reverted to the pre-specified statistical analysis plan version 3.0, which included an interaction term for age and baseline. This change only impacted the left ventricular ejection fraction endpoint in all patients, shifting the treatment difference from 2.4 percentage points (p=0.04) to 1.8 percentage points (p=0.09)."
- Linda Marban, CEO, on the BLA amendment plan:
"They plan to submit an amendment to the BLA including 24-month open-label extension data and additional analyses, with the FDA indicating willingness to review and extend the PDUFA date accordingly."