CAPR - Capricor Therapeutics Announces for the Treatment of Duchenne Muscular Dystrophy | Benzinga
SAN DIEGO, June 25, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ:CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has scheduled a Pre-BLA (Biologics License Application) meeting with the Company in the third quarter of 2024 for deramiocel (CAP-1002), for the treatment of Duchenne Muscular Dystrophy (DMD). Capricor's goal for this meeting will be to finalize its BLA filing plans based on all currently available data as well as to work with the FDA to outline the rolling BLA submission timeline.
"We continue to move rapidly towards potential approval of deramiocel for the treatment of DMD," said Linda Marbán, Ph.D., Capricor's chief executive officer. "We now have formally scheduled our Pre-BLA meeting with the FDA, which will finalize our BLA filing plans and discuss available options to expedite the filing of our BLA. We recognize the FDA's willingness to bring impactful therapies to market as quickly as possible due to the enormous unmet needs of patients with DMD. While we are delighted with the approval of gene therapy for DMD, we believe that it will take multiple therapies to combat DMD effectively. Based on the need to address the secondary consequences of DMD, we believe deramiocel can serve as a potential anchor therapy for DMD patients."
Additionally, Capricor will present its latest update of the HOPE-2 open label extension (OLE) 36-month data, both skeletal and cardiac, at the upcoming Parent Project Muscular Dystrophy (PPMD) 30th Annual Conference being held June 27-29, 2024.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory ...