As part of the FDA’s ongoing review, the Company has been informed that an Advisory Committee meeting is not indicated at this time. The BLA remains under Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of August 31, 2025.
- FDA has indicated that an Advisory Committee meeting is not required at this time.
- In-person late-cycle review meeting scheduled for mid-July.
- Biologics License Application (BLA) remains under Priority Review with PDUFA target action date of August 31, 2025.
- Four-year data presented at PPMD 2025 Conference demonstrate sustained cardiac function.
Capricor recently presented four-year data from its HOPE-2 Open-Label Extension (OLE) study at the 2025 Parent Project Muscular Dystrophy (PPMD) Conference, representing one of the longest-running treatment datasets in DMD, including measures of both cardiac and skeletal muscle function. The data demonstrated continued preservation of cardiac function, along with sustained clinical benefit from long-term Deramiocel treatment.
Why Capricor shares are falling?
Capricor Therapeutics shares fell on Monday. The FDA said an Advisory Committee meeting is not currently required for the company’s cell therapy for Duchenne muscular dystrophy.
In June, the company announced that the FDA had inspected its manufacturing facility and found no significant issues. However, recent developments have raised doubts about the treatment’s effectiveness.
Capricor shares have fallen about 30% since Friday on the news.
An advocacy organization that works in the field of cell and gene therapies has emphasized the importance of regulatory decisions and clinical trials in the field.
Capricor shares are trading at $7.30, down 11.6% on Monday.
