Peptide News Digest

#Fayuvi

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Industry · View digest

Ultragenyx FAYUVI Analyst Upgrades After Thursday Sanfilippo Type A Approval: Cantor Fitzgerald and Canaccord Genuity Move to $39 Price Targets, Citi to $32, Morgan Stanley to $20; Cantor Sees $400M Peak Commercial Opportunity

Ultragenyx Pharmaceutical (NASDAQ: RARE) received a broad wave of Friday September 18-19 sell-side analyst updates following Thursday's FDA standard full approval of FAYUVI (rebisufligene etisparvovec-hopf) as the first-ever treatment for pediatric Sanfilippo Syndrome Type A (MPS IIIA). Cantor Fitzgerald called the approval 'a much-needed win' and raised price target to $39, modeling roughly $400 million in peak commercial opportunity. Canaccord Genuity moved to $39. Citi moved to $32. Morgan Stanley maintained Equal Weight at $20. William Blair rated Market Perform, citing the approval as an incremental positive but flagging a lack of major value-inflection catalysts over the next 12 months. Analyst Sami Corwin's model at Cantor projected $325 million peak Fayuvi sales. FAYUVI shipments are expected to commence within 30-60 days. Ultragenyx confirmed retention of its Priority Review Voucher (historically worth $150-350 million on the secondary market) plus mid-single-digit to 8% royalties owed to Abeona Therapeutics (NASDAQ: ABEO) on Fayuvi net sales.

Regulatory · View digest

FDA Grants Standard Full Approval of Ultragenyx FAYUVI (Rebisufligene Etisparvovec-Hopf) as First-Ever Treatment for Pediatric Sanfilippo Syndrome Type A / MPS IIIA; $3.95M WAC Price and Priority Review Voucher Awarded

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced Thursday September 17, 2026 that the FDA granted standard full approval to FAYUVI (rebisufligene etisparvovec-hopf, previously UX111) — a one-time intravenous AAV9-delivered gene therapy carrying a functional copy of the SGSH (N-sulfoglucosamine sulfohydrolase) gene — for the treatment of pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome Type A). MPS IIIA is a progressive, ultimately fatal autosomal-recessive lysosomal storage disorder that causes rapid neurodegeneration in early childhood; before Thursday, no disease-modifying therapy existed. The approval landed two days ahead of the September 19 PDUFA action date, followed the July 2025 complete response letter that had cited chemistry-manufacturing-controls issues plus manufacturing-site observations. The clinical package supporting standard full approval (not accelerated): up to 8 years of follow-up in treated children, sustained cerebrospinal fluid heparan sulfate reduction (the accumulating substrate), and a 23.5-point cognitive-score advantage over natural-history controls. Ultragenyx received a Rare Pediatric Disease Priority Review Voucher — historically valued $150-350 million on the secondary market — alongside the approval, plus U.S. per-patient wholesale acquisition cost set at $3.95 million. RARE shares closed the day up 13% and extended gains after hours. FAYUVI is manufactured domestically at Andelyn Biosciences in Columbus, Ohio and Ultragenyx's own Bedford, Massachusetts facility. Second gene therapy approval for the company.