Larimar Therapeutics Completes Rolling BLA Submission To FDA Seeking Accelerated Approval Of Nomlabofusp For Treatment Of Patients With Friedreich’s Ataxia

• Completed BLA submission marks a major milestone in advancing nomlabofusp as the first potential disease-modifying therapy to address the underlying cause of FA
   
• Submission follows FDA alignment on key elements of the BLA package for submission, including willingness to consider skin frataxin as a potential novel surrogate endpoint
   
• Previously reported open label data showed sustained increases in skin frataxin with directional clinical improvements observed in multiple outcomes relative to a natural history reference population
   
• Patients currently enrolling in FORWARD-FA, the global confirmatory Phase 3 study for nomlabofusp

BALA CYNWYD, Pa., Nov. 09, 2026 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (NASDAQ:LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced completion of its rolling Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) seeking accelerated approval of nomlabofusp for the treatment of patients with Friedreich’s ataxia (FA). Nomlabofusp is an investigational protein replacement therapy designed to address the underlying cause of FA, frataxin deficiency, by delivering human frataxin to mitochondria.

Completion of the submission follows previously disclosed FDA feedback on key elements of the BLA and represents a significant milestone for Larimar. Larimar is currently enrolling patients in FORWARD-FA, its global confirmatory Phase 3 study, a double-blind, placebo-controlled study that will evaluate daily subcutaneous use of nomlabofusp over 18 months in ambulatory participants ages 2 to 40 who are new to the drug. Approximately two thirds of the participants will be under the age of 21.