The US Food and Drug Administration (FDA) has rejected Capricor Therapeutics’ cell therapy deramiocel for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD). The decision comes after an advisory committee meeting was canceled by CBER Director Vinay Prasad, citing insufficient evidence.
Capricor’s shares fell 40% on Friday after the FDA’s complete response letter (CRL) stated that deramiocel’s data package did not meet the “statutory requirement for substantial evidence of effectiveness.” The company plans to resubmit its application, backed by upcoming additional findings from the Phase III HOPE-3 trial.
Deramiocel is composed of allogeneic cardiosphere-derived cells (CDC), which have been shown to reduce fibrosis and modulate the immune response. Capricor claims that these cells can help preserve cardiac and skeletal muscle in patients with DMD. However, the FDA cited deficiencies in deramiocel’s Chemistry, Manufacturing, and Controls.
Capricor had scheduled an advisory committee meeting for deramiocel but canceled it due to internal conflicts within the FDA. The company also reported a second patient death linked to its approved DMD therapy Elevidys, which is attributed to acute liver failure.
Source: https://www.biospace.com/fda/capricor-plunges-on-fda-rejection-of-dmd-cell-therapy