Capricor Meeting with FDA on BLA Pathway for CAP-1002 in Duchenne

 Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment and prevention of rare diseases, announced today that the Company has been granted an in-person Type-B meeting with the U.S. Food & Drug Administration (FDA) scheduled for late March. In the upcoming meeting with the FDA, the Company intends to discuss its proposed chemistry, manufacturing and controls (CMC) plans for commercial launch, if approved which may enable opportunities to expedite the pathway to a Biologics License Application (BLA) submission. The Company has previously been granted Regenerative Medicine Advanced Therapy (RMAT) and orphan drug designations for the use of CAP-1002 in Duchenne muscular dystrophy (DMD) and these designations enable sponsors to work closely with the FDA and receive its guidance on potential expedited pathways for approval.

“At this time, with our pivotal Phase 3, HOPE-3 trial fully enrolled in Cohort A and top-line data expected in the fourth quarter of this year, we believe that discussing our CMC plans for potential commercialization with the FDA will allow us the opportunity to bring CAP-1002 to patients in the most expeditious manner possible for patients in need,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “At Capricor, we remain focused on getting to approval as quickly as possible and we look forward to providing more details after this meeting.”

https://www.biospace.com/article/releases/capricor-therapeutics-announces-upcoming-type-b-meeting-with-the-fda-to-discuss-commercial-manufacturing-planning-with-an-aim-to-expedite-bla-pathway-for-cap-1002-in-duchenne-muscular-dystrophy/