The US Food and Drug Administration (FDA) stated on Monday that Capricor Therapeutics' stem cell treatment deramiocel did not meet the objectives of a Phase 3 trial for Duchenne muscular dystrophy.
The FDA's assessment contrasts with Capricor's own characterization in December, when the company claimed that deramiocel met both the primary and secondary endpoints in a large randomized study.
The trial results were notable for coming primarily in teenagers and young men who have already lost the ability to walk, a population with few treatment options. Capricor had previously said the drug appeared to preserve upper-arm function and stave off heart failure in patients.