Larimar Therapeutics, Inc. (LRMR) is trading sharply higher today, up over 30%, on news of the FDA granting Breakthrough Therapy Designation to Nomlabofusp for the treatment of adults and children with Friedreich's ataxia.
Friedreich's ataxia is a rare, progressive, and debilitating genetic disease caused by frataxin (FXN) deficiency, leading to impaired muscle coordination, mobility loss, fatigue, and multisystem complications. An estimated 5,000 individuals in the U.S. live with FA, and treatment options remain limited.
Nomlabofusp is a frataxin protein replacement therapy designed to address the root cause of FA by increasing FXN levels in affected tissues. The FDA's Breakthrough Therapy Designation is based on preliminary clinical data from Larimar's ongoing open-label study, which shows increases in skin FXN to levels expected in asymptomatic carriers and consistent directional improvement across four key clinical outcomes: mFARS scores, Activities of Daily Living (ADL), the 9-Hole Peg Test, and the Modified Fatigue Impact Scale.
The designation follows a recent meeting under the FDA's START pilot program, where the agency confirmed alignment with Larimar on using skin FXN as a novel surrogate endpoint reasonably likely to predict clinical benefit. The FDA also agrees with the company's proposed exposure-response analyses and the selection of a matched reference population from the FACOMS natural history study for comparison.
Larimar reiterates that it remains on track to submit a BLA seeking accelerated approval in June 2026, supported by topline open-label study data expected in the second quarter of 2026. If approved, Nomlabofusp could become the first disease-modifying therapy for FA, with a potential U.S. launch targeted for the first half of 2027.
The company also plans to initiate screening for its global confirmatory Phase 3 study in the second quarter of 2026, with first patient dosing expected by mid-2026. Regulatory submissions for the trial are already under review in France and Canada, with additional filings planned in the U.K.
LRMR has traded between $1.61 and $5.37 over the past year. The stock is currently trading at $3.97, up 42%.
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