Larimar Therapeutics IncFDA pre-BLA meeting and rolling BLA submission for nomlabofusp support accelerated approval pathway.

Larimar Therapeutics has submitted the first module of its rolling Biologics License Application seeking accelerated approval of nomlabofusp for Friedreich’s ataxia, with completion expected in the second half of 2026. The submission follows a successful Type B pre-BLA meeting with the FDA, which confirmed the existing data package appears capable of supporting review and agreed to consider frataxin as a novel surrogate endpoint. Longer-term open-label study data showed sustained increases in skin frataxin levels, with 100% of participants reaching levels above asymptomatic carriers at one year, and a 2.6-point benefit on the mFARS scale at one year relative to a natural history population. The company reported $156.3 million in cash, cash equivalents and marketable securities as of June 30, 2026, providing a projected cash runway into the third quarter of 2027. Dosing of the first patient in a global confirmatory Phase 3 study is expected in the third quarter of 2026, with a potential launch targeted for mid-2027 if approved.
Larimar Therapeutics IncFDA pre-BLA meeting and rolling BLA submission for nomlabofusp support accelerated approval pathway.