BALA CYNWYD, PA — Larimar Therapeutics, Inc. (Nasdaq: LRMR) said it recently published data supporting the use of skin-based frataxin measurements as a surrogate endpoint for its experimental therapy nomlabofusp, a step that could enable an accelerated U.S. approval pathway for Friedreich’s ataxia.
The company said the findings, published in Clinical and Translational Science (https://ascpt.onlinelibrary.wiley.com/doi/10.1111/cts.70565), show that increases in frataxin levels in accessible peripheral tissues, including skin and buccal cells, correlate with levels in clinically relevant organs across animal models and patients.
Larimar said the data were included in materials reviewed by the U.S. Food and Drug Administration to support skin frataxin concentrations as a reasonably likely surrogate endpoint for its registrational program.
Use of a surrogate endpoint can allow regulators to grant accelerated approval based on biomarker data rather than long-term clinical outcomes, potentially shortening development timelines for therapies targeting serious or rare diseases.
The company said it plans to submit a Biologics License Application in June 2026 under the accelerated approval pathway.
Nomlabofusp is being developed to treat Friedreich’s ataxia, a rare genetic disease characterized by reduced frataxin levels that leads to progressive neurological and cardiac impairment.
Chief Executive Officer Carole Ben-Maimon said the consistency of frataxin increases across tissues supports the therapy’s mechanism and its potential use in measuring treatment effect.
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