FDA Nixes Capricor Rare Disease Treatment, Stock Plummets
San Diego firm hopes to overturn decision, no comment from CIRM, which backed therapy with millions.
The Food and Drug Administration (FDA) has rejected a San Diego firm’s treatment for a rare muscular disorder that leads to early death, sending the company’s stock price 30 percent lower this morning and delivering a blow to California’s $12 billion stem cell research program.
The FDA said there was insufficient evidence to justify approving the treatment for commercialization. The company, Capricor, Inc., said, “We are surprised by this decision by the FDA.” The state program, the California Institute for Regenerative Medicine (CIRM), did not have an immediate comment.
CIRM backed the proposed therapy for Duchenne muscular dystrophy with $3.4 million. In total, Capricor has received nearly $18 million from the agency, which funds research, including clinical trials, for the development of revolutionary stem cell and genetic therapies. (See here for more on Capricor’s travails with the FDA.)
Most of CIRM’s Capricor funding involves afflictions other than the genetic disorder that “causes progressive muscle weakness due to muscle fiber disarray, death, and replacement with connective tissue or fat.”
Capricor’s treatment, known as Deramiocel, had advanced into a clinical trial. Capricor is seeking approval for its widespread use.
The company said it would appeal today’s action and include information from an ongoing phase three clinical trial.
“Capricor plans to submit data from the Phase 3 HOPE-3 clinical trial to provide additional evidence of effectiveness from an adequate and well-controlled study,” company CEO Linda Marbán said in a statement.
“The HOPE-3 trial is a randomized, double-blind, placebo-controlled clinical trial of 104 patients, with topline results expected in the third quarter of 2025.
“We believe these data, if positive, along with our existing long-term clinical results showing cardiac stabilization, preservation of skeletal muscle function, and a consistent safety profile, could support efforts to resolve the questions raised by the FDA for the treatment of cardiomyopathy associated with DMD (Duchenne).”
CIRM has helped to finance 116 clinical trials during its 20-year history. None has resulted in a stem cell therapy that is available for widespread use.
The California Stem Cell Report plans to carry an update on this story this afternoon with additional comments and information from the company.