FDA Grants Fast Track Designation for Longeveron’s Treatment for Hypoplastic Left Heart Syndrome in Infants

Longeveron Inc. recently announced the US FDA has granted Fast Track Designation to Lomecel-B for the treatment of Hypoplastic Left Heart Syndrome (HLHS), a rare and life-threatening congenital heart defect affecting approximately 1,000 infants per year. Lomecel-B, an investigational allogeneic, bone marrow-derived medicinal signaling cell (MSC) product, is currently in a Phase 2a trial for HLHS.

Fast Track Designation is intended to facilitate development and expedite the review of drugs that treat serious conditions and fill an unmet medical need so a product can potentially be approved and reach patients more quickly. Fast Track Designation enables the company to have more frequent interactions with the FDA throughout the drug development process and allows for eligibility for priority review and accelerated approval if certain criteria are met, as well as a rolling review. The Fast Track Designation must continue to be met or FDA can withdraw the designation. The FDA previously granted Longeveron’s Lomecel-B Orphan Drug and Rare Pediatric Disease designations in November of 2021 for HLHS.

“Fast Track Designation represents a significant milestone in our efforts to develop Lomecel-B as a treatment for infants with HLHS,” said Chris Min, MD, PhD, Longeveron’s Interim Chief Executive Officer and Chief Medical Officer. “Fast Track Designation underscores the urgent need in HLHS, and we look forward to continuing to work closely with the FDA to bring this potential new therapy to infants as expeditiously as possible.”

HLHS is a rare congenital heart defect that affects approximately 1,000 babies per year in the US. Infants born with HLHS have an underdeveloped or absent left ventricle, which impairs the heart’s ability to pump adequate amounts of blood throughout the body. Patients require three reconstructive heart surgeries within the first five years of life, and many require a heart transplant. Without treatment, the condition is always fatal. Even with reconstructive surgical interventions, HLHS is still associated with high mortality. Overall survival from birth to adolescence is estimated to be 50% to 60%.

Longeveron is currently evaluating Lomecel-B in ELPIS II, an ongoing 38-patient, randomized (1:1), blinded, controlled Phase 2a clinical trial intended to evaluate the safety and efficacy of intramyocardial (directly into the heart) injection of the Lomecel-B product in infants with HLHS who are undergoing Stage II reconstructive cardiac surgery. In ELPIS, a Phase 1 open-label trial in ten HLHS patients, Lomecel-B was found to be well tolerated with no major adverse cardiac events one-year post-surgery, and/or treatment-related infections.

One hundred percent of the infants enrolled in the ELPIS Phase 1 trial (n=10) were alive and had not required a transplant between 2-3.5 years post-surgery. Normally, approximately 20% of patients require heart transplants to survive within one year of Stage II reconstructive surgery. Longeveron recently announced the release of a manuscript titled Intramyocardial cell-based therapy during bidirectional cavopulmonary anastomosis for hypoplastic left heart syndrome: The ELPIS Phase I trial on MedRxiv, a preprint server that posts papers before they are peer reviewed. The paper will now undergo peer review prior to publication of the final study report.

Longeveron is a clinical stage biotechnology company developing cellular therapies for specific aging-related and life-threatening conditions. The company’s lead investigational product is the Lomecel-B cell-based therapy product, which is derived from culture-expanded medicinal signaling cells (MSCs) that are sourced from bone marrow of young, healthy adult donors. Longeveron believes that by using the same cells that promote tissue repair, organ maintenance, and immune system function, it can develop safe and effective therapies for some of the most difficult disorders associated with the aging process and other medical disorders. Longeveron is currently sponsoring Phase 1 and 2 clinical trials in the following indications: Alzheimer’s disease, hypoplastic left heart syndrome (HLHS), Aging Frailty, and Acute Respiratory Distress Syndrome (ARDS). For more information, visit www.longeveron.com.