Bala Cynwyd, Pennsylvania-based Larimar Therapeutics (Nasdaq: LRMR) has submitted the first module of a rolling Biologics License Application (BLA) to the US FDA seeking accelerated approval of nomlabofusp for the treatment of adolescent and adult patients with Friedreich's ataxia (FA), a rare progressive neurological disease. The submission represents the first BLA filing for nomlabofusp and the first attempt to seek accelerated approval in FA based on tissue frataxin levels as a novel surrogate endpoint. The company said the filing was made following receipt of FDA meeting minutes from a Type B multidisciplinary pre-BLA meeting, in which the agency confirmed the existing data package appears sufficient to support submission and review.
The rolling BLA, for which the FDA has agreed to review modules as they are submitted, covers daily subcutaneous administration of nomlabofusp at 50 mg in adolescent and adult FA patients. Remaining modules, including the chemistry, manufacturing, and controls section, are expected to be submitted in the second half of 2026. The filing seeks accelerated approval using skin frataxin (FXN) concentration as a reasonably likely surrogate endpoint. Larimar said it is targeting a mid-2027 commercial launch if approved. No PDUFA target action date has been disclosed. The company noted that the FDA confirmed approval will be a matter of review, and that the adequacy of the safety database was reviewed as part of the pre-BLA package.
The clinical evidence supporting the BLA derives primarily from an ongoing long-term open-label study evaluating nomlabofusp in adolescent and adult FA patients. As of a March 2026 data cutoff, 43 participants had received at least one dose, with 13 completing one year of treatment at the 50 mg dose. All nine evaluable participants at 12 months achieved skin FXN levels exceeding 50% of the mean concentration observed in healthy volunteers, a threshold comparable to levels in asymptomatic heterozygous carriers. Directional improvements were reported across multiple clinical outcome measures — including the modified Friedreich Ataxia Rating Scale (mFARS), FARS-Activities of Daily Living, 9-hole peg test, and Modified Fatigue Impact Scale — relative to a matched reference population from the FACOMS natural history study, in which scores worsened over the same period. At one year, a mean 1.0-point mFARS improvement was observed with nomlabofusp compared to a mean 1.6-point worsening in the FACOMS reference group. Anaphylaxis occurred in 10 of 43 participants, with nine of those having prior nomlabofusp exposure; all recovered without sequelae. Larimar also received Breakthrough Therapy Designation for nomlabofusp in February 2026, and dosing of the first patient in a global confirmatory Phase III study is expected in Q3 2026.