Healthcare
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 2026Daily 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 obs
About this update from Larimar Therapeutics, Inc.
BALA CYNWYD, Pa., June 29, 2026 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced it has submitted the first module of its rolling Biologics License Application (BLA) submission to the Food and Drug Administration (FDA) for accelerated approval of nomlabofusp; the remaining modules are expected to be submitted in the second half of 2026. The submission was made after obtaining FDA meeting minutes of a Type B multidisciplinary pre-BLA meeting. The Company also announced positive data from the ongoing long-term open label (OL) study evaluating daily subcutaneous injections of nomlabofusp in adolescent and adult patients with Friedreich's ataxia (FA). FA is a rare, progressive, and fatal neurological disease with no approved disease modifying therapies that address the root cause of the disease. "Today's data represent a pivotal milestone for Larimar and, most importantly, for the FA community," said Carole Ben-Maimon, MD, President and Chief Executive Officer of Larimar. "In the Type B multi-disciplinary pre-BLA meeting minutes, FDA confirmed that our existing data package appears to be sufficient to support a BLA submission seeking accelerated approval based on skin frataxin as a potential novel surrogate endpoint and approval will be a matter of review. We are very excited to share some unique and important initial observations in our publicly available program update deck and on our conference call later this morning. These data together reinforce the strong clinical and biomarker evidence we have built to date for nomlabofusp. With compelling and consistent OL study data in hand, rolling BLA submission initiated, and dosing of the first patient in our global confirmatory Phase 3 study approaching, we are executing on all fronts to bring what could be the first disease-modifying therapy to pediatric and adult patients living with FA."
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