- Larimar Therapeutics (LRMR, Financial) receives FDA recommendations for safety database in nomlabofusp BLA submission.
- FDA suggests using skin frataxin concentrations as a surrogate endpoint for accelerated approval.
- Larimar aims to submit its Biologics License Application (BLA) by Q2 2026.
Larimar Therapeutics, Inc. (LRMR), a biotechnology company focused on treating rare diseases, has announced that it has received recommendations from the U.S. Food and Drug Administration (FDA) for its Biologics License Application (BLA) for nomlabofusp, targeting Friedreich's Ataxia. The FDA outlined the need for a safety database consisting of at least 30 participants with 6-month continuous exposure and 10 participants with 1-year exposure, predominantly at a 50 mg dosage.
Significantly, the FDA is considering the use of skin frataxin (FXN) concentrations as a reasonably likely surrogate endpoint for accelerated approval. This development marks substantial progress towards an expedited regulatory pathway for the drug, representing a potential first-in-class disease-modifying therapy for Friedreich's Ataxia.
Larimar plans to submit the BLA in the second quarter of 2026. Meanwhile, the company anticipates releasing data from an open-label extension study involving 30-40 participants and adolescent pharmacokinetic (PK) run-in data by September 2025. The global Phase 3 study, which includes sites across the U.S., Europe, the U.K., Canada, and Australia, is also moving forward and will act as a confirmatory study required for the accelerated approval process.
