Commercial Rollout And Patient DemandActivation of multiple qualified treatment centers and the treatment of the first commercial patient demonstrate rising patient demand and provide a scalable launch pathway that can drive sustained commercial uptake and improved cash flow.
Regulatory ApprovalRegulatory resubmission for UX111 includes substantial long-term clinical and biomarker evidence with FDA acknowledgement of robust neurodevelopmental outcomes, and approval would establish the first therapy for Sanfilippo type A while creating meaningful upside to analyst valuations.
Reimbursement And CodingPermanent CMS coding together with published coverage policies from major commercial payers should streamline insurance processing for ZEVASKYN and reduce access barriers, supporting faster patient adoption.