FDA Extends Review of Capricor Cell Therapy by 3 Months, Final Decision Due in November

The US Food and Drug Administration (FDA) has extended its review of Capricor Therapeutics’ Duchenne muscular dystrophy (DMD) cell therapy deramiocel from Aug. 22 to Nov. 22, a 3-month extension. The review period was extended after Capricor narrowed the indication and submitted a substantially revised application, which the FDA classified as a “Major Amendment.” Depending on the decision on Nov. 22, deramiocel could become the first allogeneic cell therapy approved to preserve upper-limb function in nonambulatory patients with DMD, drawing industry attention.

Deramiocel is a cell therapy based on allogeneic cardiosphere-derived cells (CDCs). The therapy is designed to preserve muscle and cardiac function by inducing immunomodulatory and antifibrotic effects through exosomes secreted by the cells. DMD is a rare genetic disease that primarily affects boys, causing progressive loss of muscle throughout the body and eventually leading to death from cardiac complications such as cardiomyopathy. It occurs in approximately 1 in 3,500 to 5,000 boys worldwide.

■ Cardiac indication rejected 9-3; company pivots to upper-limb function

Capricor initially submitted a biologics license application (BLA) emphasizing data on improvements in cardiomyopathy. However, in July 2026, an FDA advisory committee voted 9-3 against the cardiac indication, citing issues including changes to the statistical analysis plan. After the committee responded more favorably to the upper-limb function data, Capricor narrowed the indication to “preservation of upper-limb function,” the primary endpoint of the phase 3 HOPE-3 trial, and submitted a revised application that included 24-month open-label extension (OLE) data.

The phase 3 HOPE-3 trial enrolled 106 patients with advanced DMD. At 12 months, the deramiocel group met the primary endpoint by slowing the rate of decline in upper-limb function, as measured by the PUL 2.0 scale, by 54% compared with the placebo group. Long-term follow-up data at 36 months also showed that the effect of preserving upper-limb function was maintained. Craig McDonald, MD, of UC Davis Health, the principal investigator of the HOPE-3 trial, said, “Preserving arm and hand function is critically important for maintaining independence and quality of life, including enabling patients to feed themselves and carry out activities of daily living.”

■ Potential to become first allogeneic cell therapy; market could reach 25 trillion won

Capricor CEO Linda Marban said, “With an additional year of follow-up from the HOPE-3 trial, we have obtained the most extensive clinical data evaluating upper-limb function in patients with DMD,” expressing confidence that the additional submitted data would strongly support the indication for preserving upper-limb function. If the FDA approves deramiocel on Nov. 22, it would become the first allogeneic cell therapy approved to preserve upper-limb function in patients with advanced DMD.

The global DMD therapeutics market was estimated at approximately $4 billion to $4.6 billion (about 5.3 trillion to 6.1 trillion won) in 2025. In a market reshaped primarily by exon-skipping drugs and gene therapies, deramiocel presents a new treatment modality: an “exosome-mediated cell therapy” that suppresses inflammation and preserves muscle regardless of the specific genetic mutation. Driven by advances in gene and cell therapies, the market is projected to grow at an annual rate of 11% to 17% and reach as much as $19.1 billion (about 25 trillion won) by 2034.

Industry experts believe the FDA’s review process will establish an important regulatory precedent for South Korean biotech companies. The FDA’s rejection of the cardiomyopathy indication based on strict standards for post hoc data, along with its acceptance of a strategy focused on the primary endpoint, provides a direct reference for South Korean companies developing similar cell and gene therapies as they design clinical trials and develop regulatory strategies. Deramiocel’s commercialization rights are currently structured through partnerships focused on the US and Japanese markets, including Nippon Shinyaku, while a direct partner in South Korea has not yet been disclosed.