Longeveron Inc. (NASDAQ: LGVN) has announced that the FDA has granted its Lomecel-B drug for Hypoplastic Left Heart Syndrome treatment an Orphan Drug Designation. HLHS is a life-threatening and rare congenital heart defect that affects infants.
ODD grants market exclusivity
The goal of ODD is to help and inspire companies to find safe and effective treatments for rare diseases and ailments. Longeveron will be able to take advantage of a variety of financial and regulatory benefits as a result of ODD, including government subsidies for clinical trials, FDA user fee waivers for the future filing of a marketing application, and certain tax credits. In addition, if all of the statutory and regulatory requirements are completed, gaining ODD may result in the product receiving market exclusivity for seven years following approval for use in the rare disease or condition for which it was designated.
CEO Geof Green said, “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. 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.”
Lomecel was granted rare disease and orphan drug designation.
Principal investigator Sunjay Kaushal said, “We are gratified to receive both rare pediatric disease and orphan drug designations from FDA for this clinical trial program. This is a severe condition and a significant unmet medical need, and these important designations will be vital to facilitating a potentially more rapid development program. My colleagues around the country and I are extremely excited about the progress of the current trial, and we look forward to seeing this program move through the regulatory and clinical pathway.”
