Shots:
- The FDA has granted full approval to Fayuvi (rebisufligene etisparvovec-hopf) for pediatric pts with Sanfilippo syndrome Type A, plus a Priority Review Voucher; commercial product will be available for shipment to QTCs within 30-60 days
- Approval was supported by pivotal Transpher A & long-term follow-up data, where Fayuvi-treated pts from the mITT population (n=17) showed a 23.5-point higher cognitive score vs untreated pts from a comparable external natural history cohort (n=27)
- Trial showed clinical benefit vs natural-history decline, plus durable treatment effects across clinical assessments & multiple biomarkers, with clinical data including up to nearly 8 years of follow-up
Ref: Globenewswire | Image: Ultragenyx | Press Release
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