Larimar submits first BLA module for nomlabofusp in Friedreich's ataxia, reports open-label data

Larimar Therapeutics announced on June 29, 2026 that it submitted the first module of its rolling Biologics License Application to the FDA for accelerated approval of nomlabofusp, with the remaining modules expected in the second half of 2026¹. The submission followed FDA meeting minutes from a Type B multidisciplinary pre-BLA meeting¹.

The company also reported data from its long-term open-label study of daily subcutaneous nomlabofusp in adolescent and adult Friedreich's ataxia patients. As of June 2026, 43 adolescent and adult participants in the OL study had received at least one dose of nomlabofusp, and 22 remained in the study, with maximum treatment duration exceeding 800 days.¹

On safety, 10 of 41 participants in the OL study experienced anaphylaxis, with 9 having had prior exposure to nomlabofusp and one having no prior exposure¹. All participants returned to their usual state of health after standard treatment with no further sequelae, and 3 experienced generalized urticaria with no new occurrences after starting antihistamine therapy.¹

On efficacy, FDA reviewed the open-label data and confirmed the existing data package appears capable of supporting submission and review of a BLA seeking accelerated approval, with approval to be a matter of review¹. FDA reaffirmed willingness to consider FXN as a novel surrogate endpoint and confirmed Larimar's exposure-response analysis linking nomlabofusp exposures to clinical outcomes is the type that could support the BLA submission.¹

Looking ahead, Larimar said dosing of the first patient in its global confirmatory Phase 3 study is planned for Q3 2026, with a targeted mid-2027 launch if approved¹.