Larimar Therapeutics submits first BLA module for nomlabofusp in Friedreich’s ataxia
- Larimar Therapeutics (NASDAQ:LRMR) submitted the first module of a rolling Biologics License Application to the FDA seeking accelerated approval of nomlabofusp for Friedreich’s ataxia.
- Remaining BLA modules are expected in the second half of 2026.
- Open-label data demonstrated sustained skin FXN increases and directional clinical improvements versus a reference group, with a generally consistent safety profile.
Larimar Therapeutics (NASDAQ:LRMR) announced the submission of the first module of a rolling Biologics License Application to the U.S. Food and Drug Administration seeking accelerated approval of nomlabofusp for the treatment of Friedreich’s ataxia.
The remaining modules of the BLA are expected to be submitted in the second half of 2026.
Open-label data showed sustained increases in skin frataxin levels and directional clinical improvements compared with a FACOMS reference group.
The safety profile of nomlabofusp was generally consistent with previous studies, including reports of anaphylaxis events.
Nomlabofusp is a novel protein replacement therapy designed to increase frataxin levels in patients with Friedreich’s ataxia.
Larimar Therapeutics is advancing nomlabofusp as a potential first disease-modifying treatment for this rare, progressive neuromuscular disorder.
The company continues to work closely with the FDA to complete the rolling BLA submission and bring this therapy to patients.
