Nomlabofusp
Nomlabofusp is a frataxin replacement fusion protein developed by Larimar Therapeutics for Friedreich's ataxia. The next event is BLA submission for Nomlabofusp, expected H2 2026 (source).
- Also known as
- CTI-1601
- Company
- Larimar Therapeutics (LRMR) · Developer
- How it works
- Frataxin replacement fusion protein
- Approved
- Not approved in any market we track
Development by disease
| Friedreich's ataxia | Filed |
Upcoming events
Estimated timing · a month, quarter, half-year or year-end the company gave
Expected in H2 2026
- H2 2026Nomlabofusp · BLA submission · Friedreich's ataxia
Expected in H1 2027
- H1 2027Nomlabofusp · FDA decision · Friedreich's ataxia
Past results and milestones
- Jun 29, 2026First module of rolling BLA for accelerated approval of nomlabofusp submitted to FDA on June 29, 2026, following FDA Type B pre-BLA meeting confirmation that existing data package appears sufficient to support submission. Remaining modules expected in H2 2026. Source
- Feb 24, 2026FDA granted Breakthrough Therapy Designation to nomlabofusp for the treatment of adults and children with Friedreich's ataxia, based on preliminary open-label study data showing skin frataxin increases to asymptomatic carrier levels and consistent directional improvements in mFARS, ADL, 9-HPT, and MFIS after 1 year of treatment. Source