FAYUVI is an Ultragenyx Pharmaceutical Inc. program in sanfilippo syndrome type A (MPS IIIA). The FDA approved it on Sep 17, 2026. 1 FDA decision on record below. Every date links its source.
All upcoming FDA decision dates on the 2026 PDUFA calendar
FDA approval tracked on pdufa.bio: sanfilippo syndrome type A (MPS IIIA) (approved Sep 17, 2026). Earlier approvals may not be listed here; the FDA label is the full record. Sponsor: Ultragenyx Pharmaceutical Inc. (RARE). Approval record: Standard full approval on September 17, 2026, two days before the September 19 PDUFA goal date; the first FDA-approved treatment for Sanfilippo syndrome type A and Ultragenyx's second gene therapy approval. A Priority Review Voucher was awarded. UX111 (ABO-102) was licensed from Abeona Therapeutics (ABEO). Market-cap tier at the time we tracked it: Small. We track 1 catalyst event for this program: 0 upcoming and 1 in the record. A PDUFA date is the FDA's target date to complete review of a marketing application; a readout is the sponsor's expected date for clinical trial results. What a PDUFA date is · why we do not compare trial results across drugs.
| Fund | Shares | Value at quarter end | Filing |
|---|---|---|---|
| RTW Investments | 7,796,895 | $260.3 million | filed August 14, 2026 |
13F reports list long positions in common stock at quarter end, are due 45 days later, and show no short positions or later trades. Notes, warrants and other instruments are not counted as shares.
No upcoming catalyst is on our calendar for FAYUVI; 1 FDA decision is on record below.
On Sep 17, 2026 the FDA approved the application. The decision page carries the source document and the measured share-price reaction.
In our records FAYUVI is FDA-approved for sanfilippo syndrome type A (MPS IIIA) (approved Sep 17, 2026). Other approved uses may not be tracked here; the FDA label is the full record.
Ultragenyx Pharmaceutical Inc. (RARE).
Yes. The FDA approved an application for FAYUVI on Sep 17, 2026; the decision page linked above carries the source.
FDA announcements, sponsor press releases and SEC filings; every date on this page links its source.