Efficacy DataOpen-label results showed nomlabofusp increased skin frataxin to levels comparable to healthy volunteers in all evaluable participants, supporting skin frataxin as a surrogate that suggests potential disease-modifying benefit pending randomized confirmation.
Prescriber Acceptance And UptakeHigh prescriber comfort with nomlabofusp despite anaphylaxis concerns, combined with adoption precedent for therapies with similar risks, supports the prospect of meaningful market uptake if efficacy is confirmed.
Regulatory Pathway And Filing TimelineInclusion in the FDA START program and ongoing agency interactions, together with a planned BLA submission under an accelerated approval pathway in the second quarter of 2026, could strengthen the clarity of the regulatory path.