Larimar submits BLA module for nomlabofusp accelerated approval; positive open-label data in Friedreich's ataxia

Larimar Therapeutics, Inc.

• Submitted first module of rolling BLA to FDA for accelerated approval of nomlabofusp; FDA confirmed existing data appears sufficient.
• Open-label study: 100% (9/9) of participants achieved skin FXN levels >50% of healthy volunteer mean at 1 year.
• Clinical outcomes improved vs natural history: mean 1.0-point mFARS improvement vs 1.6-point worsening at 1 year.
• Safety: anaphylaxis in 10/43 patients; 9 had prior nomlabofusp exposure, 1 no prior; all recovered.
• First patient in confirmatory Phase 3 expected Q3 2026; targeting mid-2027 launch if approved.