Update from Larimar Therapeutics
Larimar Therapeutics, Inc. announced today, February 24, 2026, that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to nomlabofusp, a frataxin (FXN) protein replacement therapy 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. The planned BLA submission is targeted for June 2026.
Breakthrough Therapy Designation is intended to expedite the development and regulatory review of a drug intended to treat a serious condition. Larimar reported that FDA is aligned with plans to have the global confirmatory Phase 3 study underway at the time of BLA submission.
Learn more about Larimar’s nomlabofusp program on our pipeline page.
