Longeveron Inc. (NASDAQ: LGVN) has announced a positive Type C meeting with the U.S. Food and Drug Administration (FDA) regarding the development pathway for Lomecel-B™, a cellular therapy being investigated for Hypoplastic Left Heart Syndrome (HLHS). The FDA's feedback supports the potential for Lomecel-B™ to advance towards a Biological License Application (BLA) submission, contingent on the outcomes of the ongoing Phase 2b ELPIS II clinical trial. This alignment could significantly expedite the availability of a new treatment option for this life-threatening condition affecting infants.
Regulatory Alignment on ELPIS II Trial
Longeveron and the FDA have reached an agreement on the primary and secondary endpoints for the ELPIS II trial. The FDA has indicated that, subject to certain conditions, a positive ELPIS II trial could be considered pivotal and, if successful, acceptable for BLA submission, potentially leading to full traditional approval of Lomecel-B™ for HLHS. These conditions include submitting a pre-specified Statistical Analysis Plan (SAP) and a Chemistry, Manufacturing, and Controls (CMC) readiness plan, including Lomecel-B™ stability and comparability data, for prior review.
Lomecel-B™ and HLHS
Lomecel-B™ is an allogeneic, investigational cellular therapy derived from medicinal signaling cells (MSCs) isolated from the bone marrow of healthy adult donors. These MSCs are believed to promote tissue repair and regeneration through anti-inflammatory and pro-vascular mechanisms. HLHS is a severe congenital heart defect where the left ventricle is underdeveloped, affecting approximately 1,000 infants annually in the U.S. Current treatment involves a complex three-stage surgical process, but even with this intervention, only 50% to 60% of infants survive to adolescence due to right ventricular failure, highlighting a significant unmet medical need.
Clinical Data and FDA Designations
The ELPIS II trial builds on the encouraging results from the ELPIS I trial, where children treated with Lomecel-B™ experienced 100% transplant-free survival up to five years of age, compared to an approximate 20% mortality rate observed in historical control data. Lomecel-B™ has been granted Orphan Drug, Fast Track, and Rare Pediatric Disease designations by the FDA for the HLHS program. If Longeveron receives FDA marketing approval for Lomecel-B™ for HLHS, the company may be eligible for a Priority Review Voucher, further incentivizing the development of this therapy.
Executive Perspective
"We are pleased to have alignment with FDA on the development pathway for our Lomecel-B™ development program in HLHS, which has a devastating impact on patients and their families," said Wa’el Hashad, Chief Executive Officer of Longeveron. "While we have a lot of work yet to do, the potential for ELPIS II to serve as the foundation for a BLA submission significantly reduces the time to reach submission and potential approval of Lomecel-B™ as an HLHS adjunct therapy."
