Larimar Therapeutics is a clinical-stage biotech with a single drug in development: nomlabofusp, a treatment for Friedreich's ataxia (FA), a rare and fatal genetic disease affecting roughly 20,000 people globally. FA is caused by a genetic defect that prevents cells from producing sufficient frataxin (FXN), a protein essential to mitochondrial function, leading to progressive neurological and cardiac deterioration and death typically between age 30 and 50. Nomlabofusp is a recombinant fusion protein that delivers FXN directly into mitochondria via a cell-penetrating peptide, administered by daily subcutaneous injection. It is the only program designed to directly raise FXN levels, and Larimar argues it could be the first disease-modifying therapy for FA. The only approved FA therapy, Biogen's omaveloxolone, does not address the underlying FXN deficiency. Larimar has no approved products and no revenue, funding operations entirely through equity raises. The company is targeting a BLA submission for accelerated approval in June 2026, using skin FXN concentration as a surrogate endpoint, after the FDA granted Breakthrough Therapy Designation in February 2026. A confirmatory Phase 3 trial is planned to start in mid-2026. Manufacturing is fully outsourced. If approved, Larimar's commercial approach is undetermined, though a targeted rare disease model with a high-priced therapy is the most likely path given the small patient population. A key risk is anaphylaxis, observed in 7 of 39 participants in the open-label study, which Larimar is managing through a modified dosing regimen agreed upon with the FDA.