The stock of Capricor Therapeutics, Inc. (NASDAQ: CAPR) is rising significantly on the charts right now. Following the company’s announcement of plans to submit a Biologics License Application (BLA), shares of CAPR were up 39.03% to $8.30 as of the most recent market check. This action represents a significant step toward the approval of its main medication, deramiocel, for the management of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).
Capricor Plans for Biologics License Application
Capricor disclosed its intent to submit a BLA based on promising cardiac data and natural history studies. After many meetings with the Food and Drug Administration (FDA) in the United States, the business plans to submit a BLA by October 2024, and by the end of the year, the complete application is anticipated.
CAPR Expanding the Treatment Scope
Apart from pursuing approval for managing cardiomyopathy associated with DMD, Capricor intends to conduct post-approval investigations to broaden the drug’s indication to include skeletal muscle myopathy. Data from Cohorts A and B of the company’s current Phase 3 HOPE-3 study will be used. Notably, Capricor has chosen to forgo unblinding Cohort A, which was initially planned for the fourth quarter of 2024, in order to maintain the integrity of the trial for upcoming analysis.
Addressing an Unmet Medical Need
As of right now, DMD cardiomyopathy—the primary cause of mortality for those with Duchenne muscular dystrophy—has no FDA-approved treatments. The FDA’s dedication to developing treatments for uncommon diseases and the compelling clinical evidence demonstrating deramiocel’s efficacy support the company’s application for approval. There is optimism for a much-needed therapy since the medicine has shown in many studies to be able to decrease heart degeneration.
Regulatory Support and Path Forward
Deramiocel holds FDA Orphan Drug Designation and is further supported by the RMAT (Regenerative Medicine Advanced Therapy) designation. If approved, Capricor could also secure a Priority Review Voucher (PRV), owing to its rare pediatric disease designation. These developments position the company to expedite deramiocel’s path to market, potentially offering a groundbreaking treatment to DMD patients.
