FDA Grants Longeveron Orphan Drug Designation for Lomecel-B in Infants with Hypoplastic Left Heart Syndrome

Longeveron Inc., a clinical-stage biotechnology company developing cellular therapies for chronic aging-related and certain life-threatening conditions, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for Lomecel-B for the treatment of Hypoplastic Left Heart Syndrome (HLHS), a rare and life-threatening congenital heart defect in infants.

ODD is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases or conditions. ODD positions Longeveron to be able to potentially leverage a range of financial and regulatory benefits, including government grants for conducting clinical trials, waiver of FDA user fees for the potential submission of a marketing application, and certain tax credits. Receiving ODD may also result in the product receiving seven years market exclusivity upon approval for use in the rare disease or condition for which the product was designated if all of the statutory and regulatory requirements are met.

“Adding to the Rare Pediatric Disease (RPD) designation already granted to Lomecel-B for treatment of HLHS, the FDA’s decision to grant ODD to Lomecel-B for this indication indicates the ongoing and unmet need for new therapies to treat infants with HLHS,” said Geoff Green, Chief Executive Officer at Longeveron. “Building on results from our completed Phase 1 safety-focused trial, we believe Lomecel-B has potential to improve outcomes for these severely impacted infants by way of repairing cardiac tissue and improving ventricular function. The combination of both RPD and ODD allows us to potentially move more efficiently through clinical development and regulatory review, and Lomecel-B may be eligible for a period of marketing exclusivity upon approval for this indication.”