Longeveron Inc. announced the positive Type C meeting with the FDA, supporting the advancement of Lomecel-B™, a proprietary, scalable, allogeneic, investigational cellular therapy currently being evaluated in a Phase 2b clinical trial (ELPIS II) for hypoplastic left heart syndrome (HLHS).
The Company and the FDA reached foundational alignment on the primary endpoint and secondary endpoints for ELPIS II. The FDA confirmed that, with several conditional requirements to be accomplished, ELPIS II may be deemed pivotal, and, if positive, acceptable for Biological License Application (BLA) submission for full traditional approval. Among other items, the Company will need to submit its prespecified Statistical Analysis Plan (SAP) and Chemistry, Manufacturing and Controls (CMC) readiness plan, including Lomecel-B™ stability and comparability data, to the FDA for prior review.
“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.”
ELPIS II builds on the positive clinical results of ELPIS I, in which children in the trial experienced 100% transplant-free survival up to five years of age after receiving Lomecel-B™ compared to an approximate 20% mortality rate observed from historical control data.
ELPIS II is being conducted in collaboration with the National Heart, Lung, and Blood Institute (NHLBI) through grants from the National Institutes of Health (NIH).
The Lomecel-B™ HLHS program has received three FDA designations: Orphan Drug designation, Fast Track designation and Rare Pediatric Disease designation. Under the Rare Pediatric Disease designation, if Longeveron were to receive FDA marketing approval for Lomecel-B™ for HLHS, the Company could be eligible to receive a Priority Review Voucher.
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