Regulatory DesignationsNomlabofusp previously received Rare Pediatric Disease Designation, Fast Track Designation, and Orphan Drug Designation from the FDA.
Safety And EfficacyLonger-term safety data begin to confirm that nomla is safe and tolerable for chronic dosing, which has been a key focus of the investment thesis.
Therapeutic PotentialNomlabofusp targets the root cause of Friedreich's Ataxia, addressing a high unmet medical need in this genetic neurodegenerative disease.