Larimar Therapeutics announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to its lead candidate, nomlabofusp, for the treatment of Friedreich’s ataxia. The designation, awarded on February 24 2026, signals that the FDA believes the drug has the potential to offer substantial therapeutic benefit over existing options and will provide the company with an accelerated development and review pathway.
The designation is based on preliminary data from Larimar’s ongoing open‑label study. The study showed that skin frataxin (FXN) levels rose to levels comparable to asymptomatic carriers and that patients experienced consistent directional improvements across four key clinical outcome measures. The FDA has aligned with Larimar on using skin FXN as a novel surrogate endpoint that is reasonably likely to predict clinical benefit, a critical step toward a Biologics License Application (BLA) that could be submitted in June 2026.
Financially, Larimar has strengthened its balance sheet, with cash increasing from $21 million at the end of 2024 to $90 million by the end of September 2025. The company’s current ratio of 4.06 reflects a solid liquidity position, although it continues to burn cash at a rate typical for a clinical‑stage biopharma advancing toward commercialization.
The company’s development roadmap now includes a BLA submission slated for June 2026, topline data from the open‑label study expected in Q2 2026, and the initiation of a global Phase 3 confirmatory study in Q2 2026. First patient dosing in the Phase 3 trial is projected for mid‑2026, with a potential U.S. launch in the first half of 2027 if regulatory approval is obtained.
In the broader therapeutic landscape, Skyclarys (omaveloxolone) was approved in February 2023 as the first treatment for Friedreich’s ataxia, but it addresses symptoms rather than the underlying frataxin deficiency. Nomlabofusp aims to be the first disease‑modifying therapy for the condition, positioning Larimar as a potential first‑mover in a market with no approved disease‑modifying options.
"We were pleased to recently share exciting long‑term data from our open‑label study showing consistent directional improvement across four key clinical outcome measures relative to a Friedrich’s Ataxia Clinical Outcomes Measure Study (FACOMS) reference population, and increased skin frataxin (FXN) levels similar to asymptomatic carriers," said Larimar Therapeutics.
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