Larimar Therapeutics announces FDA granted BTD to nomlabofusp

Larimar Therapeutics (LRMR) announced the U.S. Food and Drug Administration, FDA, has granted Breakthrough Therapy Designation, BTD, to nomlabofusp, a frataxin protein replacement therapy with disease modifying potential, for the treatment of adults and children with Friedreich’s ataxia. Additionally, after a recent Support for Clinical Trials Advancing Rare Disease Therapeutics 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 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.