Larimar Therapeutics receives FDA Breakthrough Therapy Designation for nomlabofusp in Friedreich's ataxia

Larimar Therapeutics, Inc.

FDA granted Breakthrough Therapy Designation for nomlabofusp for adults and children with Friedreich's ataxia.
FDA aligned on using skin FXN as a surrogate endpoint to support BLA seeking accelerated approval.
Planned BLA submission remains on track for June 2026; topline open-label data expected Q2 2026.
Global confirmatory Phase 3 study to initiate Q2 2026; first patient dosing targeted mid-2026.
U.S. launch targeted for first-half 2027, if approved; estimated 5,000 U.S. patients with FA.

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