Update from Larimar Therapeutics
In a press release and webcast today, June 23, 2025, Larimar Therapeutics announced that they received clear expectations on the safety database recommendations from the FDA for their nomlabofusp program in FA and refined their timeline for Biologics License Application (BLA) submission to the second quarter of 2026 to allow for inclusion of the recommended safety data for adults and children. Nomlabofusp is designed to increase frataxin levels in FA with protein replacement.
The FDA recommended that the safety database include a total of at least 30 participants with continuous exposure for 6 months including a subset of at least 10 with 1-year; large majority of the exposure should be on the 50 mg dose. Larimar remarked about being selected for the pilot START program at FDA as an important factor in regular and clear communication that is helping advance the program.
The ongoing OLE Study is evaluating safety and tolerability, PK, and FXN levels in buccal and skin cells, along with exploratory pharmacodynamic markers and clinical outcomes following long-term once daily subcutaneous administration of nomlabofusp. Enrollment is ongoing and all active participants are currently receiving the 50 mg dose. Expansion of the open label study is planned to include patients who have never participated in any of our prior clinical trials and have never been exposed to nomlabofusp.
Larimar Therapeutics reviewed results of completed studies and also shared that activities are ongoing with the identification and qualification of sites in U.S., Europe, U.K., Canada, and Australia for a global Phase 3 study. The Phase 3 study is expected to be underway at the time of BLA submission and is currently intended as the confirmatory study to verify clinical benefit as required by FDA’s accelerated approval pathway.
Click button below to read the full press release or listen to this morning’s webcast.
