Clinical Efficacy And Surrogate BiomarkerNomlabofusp increased skin frataxin to about half of healthy levels in all evaluable participants by six months, supporting skin frataxin as a surrogate endpoint and indicating potential disease-modifying effect pending randomized confirmation.
Physician Acceptance And Adoption PotentialHigh prescriber comfort with nomlabofusp's risk–benefit profile despite anaphylaxis suggests clinicians may adopt the therapy where unmet need is high, supporting commercial uptake prospects.
Regulatory Pathway And TimingInclusion in the FDA START program and ongoing agency interactions, along with a planned BLA submission under an accelerated approval pathway in Q2 2026, provides regulatory clarity that could boost investor confidence.