regulatory
confidence high
sentiment positive
materiality 0.80
FDA removes partial clinical hold on Larimar's nomlabofusp for Friedreich's Ataxia
Larimar Therapeutics, Inc.
- FDA cleared Phase 2 data review; nomlabofusp generally well-tolerated with dose-dependent frataxin increases.
- OLE study continues at 25 mg; dose escalation to 50 mg planned after further frataxin PD characterization.
- Interim OLE data expected Q4 2024; BLA submission targeted for 2H 2025.
- In 50 mg cohort, all evaluable patients achieved skin frataxin >33% of healthy volunteer average at Day 14.