- Larimar Therapeutics (LRMR, Financial) publishes nonclinical data supporting the therapeutic potential of nomlabofusp for Friedreich's ataxia.
- FDA considers the use of skin FXN concentrations as a surrogate endpoint for accelerated approval.
- Larimar plans to submit a Biologics License Application (BLA) by Q2 2026.
Larimar Therapeutics, Inc. (NASDAQ:LRMR) has unveiled two peer-reviewed articles that highlight promising nonclinical data on nomlabofusp, a potential treatment for Friedreich’s ataxia (FA). The studies underline the drug's mechanism of action and its capacity to elevate frataxin (FXN) protein levels in essential tissues, such as the dorsal root ganglia, heart, and skeletal muscle.
This comprehensive research has been incorporated into the FDA's briefing package, providing support for the utilization of skin FXN concentrations as a reasonably likely surrogate endpoint for accelerated approval. This development is pivotal in expediting the regulatory pathway for nomlabofusp, especially crucial for rare diseases like FA.
Nomlabofusp, a fusion protein designed to deliver functional frataxin into mitochondria, addresses the fundamental cause of FA—insufficient FXN levels leading to mitochondrial dysfunction. Larimar aims to submit a Biologics License Application (BLA) by the second quarter of 2026, positioning nomlabofusp as the first potential disease-modifying therapy for FA patients.
The condition, which affects about 1 in 50,000 people, results in progressive neurological and cardiac issues. With no approved disease-modifying treatments currently available, Larimar's progress represents a significant step forward in potentially altering the course of this debilitating disease.
