Overview
- On Friday, Capricor said it will submit an amended Biologics License Application that leans on 24‑month open‑label data for the HOPE‑3 trial’s primary upper‑limb muscle function endpoint.
- The FDA has told the company it will review the amendment and will extend its PDUFA action date once the new submission is received.
- The move follows a July regulatory review in which FDA staff flagged post‑hoc changes to Capricor’s statistical analysis and an advisory committee on July 29 voted 9–3 against recommending approval for Duchenne cardiomyopathy.
- Investors pushed Capricor shares more than 100% higher in premarket trading after the announcement, while a securities‑fraud class action accuses the company of hiding analysis changes and making misleading statements.
- The amended pathway would seek approval for improved arm and hand function rather than disputed cardiac benefit, a shift that would change which patients get access and how clinicians view the drug’s potential; regulators’ response to the new 24‑month data and the pending litigation are now the key next milestones.