Larimar aims to start a rolling Biologics License Application for nomlabofusp in June 2026, submitting nonclinical and clinical modules first and completing the chemistry, manufacturing, and controls module in the second half of the year. A Type B meeting is scheduled prior to submission to secure additional FDA feedback and alignment on data requirements.

Cross-species studies published in April showed nomlabofusp significantly raises frataxin levels across heart, brain, dorsal root ganglia, and skeletal muscle, supporting skin frataxin concentration as a reasonably likely surrogate endpoint. These findings underpin the Accelerated Approval strategy by correlating peripheral tissue FXN changes with target organ levels.

As of March 31, 2026, Larimar held $200.4 million in cash, cash equivalents, and marketable securities, extending its cash runway into Q2 2027. In February, the company completed a $115 million underwritten public offering, securing $107.6 million in net proceeds to fund clinical development.

Topline data from the open-label study is expected in the second quarter of 2026 to support the BLA submission, and the first patient dosing in the global confirmatory Phase 3 trial is planned for mid-2026. These milestones could drive significant regulatory and clinical progress for nomlabofusp in Friedreich’s ataxia.