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Larimar Therapeutics Announces FDA Breakthrough Therapy Designation for Nomlabofusp in FA and Reiterates Planned BLA Submission in June 2026
Nomlabofusp program granted Breakthrough Therapy Designation for the treatment of adults and children with FA based on FDA’s review of available clinical data
About this update from Larimar Therapeutics, Inc.
[{"type":"text","content":"Nomlabofusp program granted Breakthrough Therapy Designation for the treatment of adults and children with FA based on FDA’s review of available clinical data from open label studyFDA written communications after recent START meeting continue to align with use of skin FXN to support BLA submission seeking accelerated approvalTopline open label study data to support BLA submission expected in Q2 2026 Planned BLA submission seeking accelerated approval on track for June 2026; U.S. launch targeted for first-half 2027, if approved BALA CYNWYD, Pa., Feb. 24, 2026 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to nomlabofusp, a frataxin (FXN) protein replacement therapy with disease modifying potential, for the treatment of adults and children with Friedreich’s ataxia (FA). Additionally, after a recent Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program meeting with FDA, the Company announced continued alignment with the FDA to consider the use of skin FXN as a novel surrogate endpoint reasonably likely to predict clinical benefit to support a planned Biologics License Application (BLA) submission seeking accelerated approval. There was also agreement on the relevant clinical outcomes with consistent directional improvement in all four clinical outcomes assessed and the type of analyses required to support the exposure response relationships for the nomlabofusp program. The FDA stated that the adequacy of the safety database will be a matter of review at the time of BLA submission. The planned BLA submission is targeted for June 2026. Breakthrough Therapy Designation and FDA feedback on BLA submission were based on the FDA’s review of available clinical data from the Company’s ongoing open label (OL) study evaluating nomlabofusp in adult and pediatric patients with FA. Breakthrough Therapy Designation Granted by FDA for Nomlabofusp for the Treatment of FA Breakthrough Therapy Designation is intended to expedite the development and regulatory review of a drug intended to treat a serious condition. A drug is eligible for BTD if preliminary clinical evidence indicates that...