Larimar submits first module of rolling BLA for nomlabofusp in FA

Larimar has initiated a rolling BLA submission for accelerated approval of nomlabofusp in Friedreich’s ataxia after FDA confirmed the existing data package appears sufficient. The first module was submitted; remaining modules are expected in 2H 2026. FDA reaffirmed willingness to consider skin FXN as a novel surrogate endpoint.

Key facts

Filed: Jun 29, 2026, 10:30 AM
Event: Application submitted
Direction: Positive
Company: LRMRLarimar
Asset: nomlabofusp
Source: Company press release
Confidence: Sourced
anaphylaxis: 10/43 (9 prior exposure)
skin fxn 1yr: 100% (9/9) >50% healthy mean
mFARS 1yr diff: 2.6-point
mFARS 18mo diff: 4.6-point

Source excerpt

Larimar Therapeutics Reports Positive Open Label Data and Submission of First Module of Rolling BLA for Accelerated Approval of Nomlabofusp for Friedreich’s Ataxia FDA alignment on submission of BLA data package in multi-disciplinary Type B pre-BLA meeting minutes; first module of rolling BLA submitted with remaining modules expected 2H 2026 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 Continued directional improvement across mFARS, FARS-ADL, 9-HPT, MFIS observed at 1 year of nomlabofusp treatment (n = 13) relative to a worsening in a FACOMS natural history study reference population One of six non-ambulatory participants at baseline became ambulatory after 1 year of dosing; none of the seven ambulatory participants progressed to non-ambulatory at 1 year Well-characterized safety profile; long-term dosing continues to be generally well-tolerated; anaphylaxis occurred in 10/43 patients with 9 of the 10 having exposure to nomlabofusp in a prior study 11 participants had no exposure to nomlabofusp in a prior study; 1 experienced anaphylaxis Dosing of first patient in global confirmatory Phase 3 study expected Q3 2026 Company management to host webcast and conference call today at 7:45 a.m. EDT BALA CYNWYD, Pa., June 29, 2026 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (

Company press release