Capricor has again come up with the pivotal results for DMD cardiomyopathy therapy after the rejection. After six months of receiving the shocking rejection for Capricor’s cell therapy deramiocel, which showed a steady decline in cardiac function and a noteworthy benefit in upper-limb function in a Phase 3 trial.
The FDA stated that it lacked exponential evidence of effectiveness. Despite this rejection and failure, the company has prepared robust data. McDonald stated that the HOPE-3 study is the first Phase 3 trial in a vast non-ambulatory group with DMD to achieve the initial endpoint.
Capricor is waiting for the pivotal data that is intended to support it as a response to the FDA’s previously sent complete response letter. The CEO of Capricor, Linda Marban, said, “This new day marks our 20 years of hardship. With all the years of learning and understanding the mechanism of action and seeking the ideal way to deploy the strength and potential value of deramiocel, we are now excited and pleased to present this data.”
“This new data will confirm that the HOPE-3 trial has shown measurably noteworthy improvement in cardiomyopathy and skeletal muscle.”
Linda added, “We trust that these pivotal study results, attached with the proof paper from the HOPE-2 and HOPE-2 (open label expansion) studies, will give us the power to identify the clinical hurdles in the Complete Response Letter received this year. The FDA’s claim continued with the important FDA guidance that the HOPE-3 results should be strong enough to support the regulatory approval.”
This was a long journey for Capricor and deramiocel. In July, CRL, FDA also stated that Capricor’s data package does not meet the statutory requirements and is short enough to prove substantial evidence of effectiveness. This was shocking for Linda. Recently the Capricor and deramiocel have been all covered up in the FDA’s ongoing communication and personnel melodrama this year.
An infinite amount of R&D dollars and attention has been spent by the company to establish the appropriate treatment for the muscular impact of Duchenne muscular dystrophy. Hopefully, Capricor’s data will be convincing for the FDA. The year ending is bringing new hope to the company’s hardship after their earlier faced rejection. These consistent efforts are counted in the excellence list for this year, which might bring development and milestones into Capricor’s operation.
Mansi Kadam is a market research writer with over 3 years of experience analyzing trends in the healthcare industry. At Towards Healthcare, she covers innovations in medical sector, sustainability initiatives, and the evolving regulatory landscape.
