BriefIQ Markets · 8-K
Ultragenyx Pharmaceutical Inc.
FDA fully approves FAYUVI, adding first MPS IIIA treatment and a second approved gene therapy
Key Facts
- On September 17, 2026, Ultragenyx announced that the FDA granted standard full approval of FAYUVI (rebisufligene etisparvovec-hopf), also known as UX111, for treating pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome Type A).
- FAYUVI is the first FDA-approved treatment for Sanfilippo syndrome Type A and Ultragenyx’s second gene therapy approval.
- Ultragenyx received a Priority Review Voucher in connection with the approval.
Source
Filing details
- Form
- 8-K
- Symbol
- RARE
- Company
- Ultragenyx Pharmaceutical Inc.
- CIK
- 0001515673
- Accession number
- 0001515673-26-000006
- SEC filing date
- 2026-09-17
- SEC acceptance time
- 2026-09-17T19:55:37+00:00
- RSS publication time
- 2026-09-17T19:55:37+00:00
- BriefIQ published
- 2026-09-17T20:04:38.692712+00:00
- Reporting period
- 2026-09-17