FDA Grants Longeveron Orphan Drug Designation for Lomacel-B

Longeveron, a clinical stage biotechnology company, announced on Dec. 6, 201, that FDA granted Orphan Drug Designation (ODD) to Lomecel-B, an allogenic injection designed to treat age-related chronic illnesses. The ODD was granted for the treatment of Hypoplastic Left Heart Syndrome (HLHS), a rare and life-threatening congenital heart defect in infants.

HLHS is a condition wherein the left ventricle is underdeveloped, resulting in impaired ability to pump blood through the body; it is often fatal. According to a company press release, HLHS occurs in approximately 1000 babies in the United States each year. Because the incidence rate is this low, it qualifies for an ODD, which grants exclusive benefits to treatments designed for rare and underserved conditions.

“Adding to the Rare Pediatric Disease 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, CEO at Longeveron, in the press release. “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.”