Larimar Therapeutics Reports Fourth Quarter and Full Year 2025 Financial Results

Breakthrough Therapy Designation granted to nomlabofusp for the treatment of adults and children with FA

Mar. 19, 2026 at 11:20am

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Larimar Therapeutics, a clinical-stage biotechnology company, reported its fourth quarter and full year 2025 operating and financial results. The company highlighted receiving Breakthrough Therapy Designation from the FDA for its lead compound, nomlabofusp, as a potential treatment for Friedreich's ataxia (FA). Larimar also announced continued alignment with the FDA on its planned Biologics License Application (BLA) submission for nomlabofusp seeking accelerated approval, expected in June 2026, and plans to initiate screening for a global confirmatory Phase 3 study in the second quarter of 2026.

Why it matters

The Breakthrough Therapy Designation for nomlabofusp underscores the significant unmet medical need in FA and the potential of the compound to address the underlying frataxin (FXN) deficiency that causes the debilitating symptoms experienced by people with FA. Larimar's progress in aligning with the FDA on its registrational path for nomlabofusp, including the use of FXN as a novel surrogate endpoint, brings the company closer to potentially delivering the first disease-modifying therapy for this rare and devastating disease.

The details

Larimar announced that the FDA granted Breakthrough Therapy Designation to nomlabofusp based on the review of available clinical data from the company's ongoing open-label study. The company also reported continued alignment with the FDA on the content of its planned BLA submission, including the use of FXN as a novel surrogate endpoint and the proposed statistical plan for natural history comparisons. Larimar plans to report topline data from the open-label study in the second quarter of 2026 and initiate screening for a global confirmatory Phase 3 study in the same timeframe, with dosing of the first patient expected mid-2026. The company's BLA submission seeking accelerated approval is on track for June 2026, with a targeted U.S. launch in the first-half of 2027 if approved.

  • In February, the FDA granted Breakthrough Therapy Designation to nomlabofusp.
  • In February, Larimar announced continued alignment with the FDA on BLA content following a recent START pilot program meeting.
  • Larimar plans to report topline data from the open-label study in the second quarter of 2026.
  • Larimar plans to initiate screening in the global confirmatory Phase 3 study in the second quarter of 2026, with dosing of the first patient expected mid-2026.
  • Larimar's planned BLA submission seeking accelerated approval is scheduled for June 2026.

The players

Larimar Therapeutics, Inc.

A clinical-stage biotechnology company focused on developing treatments for complex rare diseases, including Friedreich's ataxia.

Carole Ben-Maimon, MD

President and Chief Executive Officer of Larimar Therapeutics.

nomlabofusp

Larimar's lead compound being developed as a potential treatment for Friedreich's ataxia.

Food and Drug Administration (FDA)

The regulatory agency that granted Breakthrough Therapy Designation to nomlabofusp and is working with Larimar on the registrational path for the compound.

Friedreich's Ataxia Clinical Outcomes Measure Study (FACOMS)

A database that Larimar plans to use to select a reference population for natural history comparisons in its BLA submission.

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What they’re saying

“This is an exciting and pivotal time for Larimar as we continue advancing nomlabofusp toward registration. Receiving Breakthrough Therapy Designation from the Food and Drug Administration (FDA) highlights both the significant unmet needs in Friedreich's ataxia (FA) and the potential of nomlabofusp to address the underlying frataxin (FXN) deficiency that causes the disabilities experienced by people with FA.”

— Carole Ben-Maimon, MD, President and Chief Executive Officer of Larimar Therapeutics

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