Press Release: Larimar Therapeutics Reports Positive Open Label Data and Submission of First Module of Rolling BLA for Accelerated Approval of Nomlabofusp for Friedreich’s Ataxia

In a press release and investor call today, June 29, 2026, Larimar Therapeutics announced the first module of a rolling Biologics License Application (BLA) has been submitted to the FDA and they provided an update on safety, tolerability, and efficacy in the Open Label (OL) study.

As of June 2026, 43 adolescent and adult participants in the OL study had received at least one dose of nomlabofusp and 22 participants remain in the study with a maximum treatment duration of more than 800 days.

Daily nomlabofusp increased and sustained skin FXN levels at 1 year and 18 months; 100% (9/9) of participants achieved and maintained levels over 50% of mean levels in healthy volunteers (comparable to asymptomatic heterozygous carriers) at 1-year.

Additionally, Larimar reported continued directional improvement across mFARS, FARS-ADL, 9-HPT, MFIS observed at 1 year of nomlabofusp treatment (n = 13).

Dosing of first patient in global confirmatory Phase 3 study expected Q3 2026.