Ultragenyx Pharmaceutical Inc. filings document a rare-disease biopharmaceutical business with approved medicines, investigational genetic-disease therapies, and a Nasdaq-listed common stock capital structure. Form 8-K reports include operating results, financial guidance, FDA communications, clinical-study data, and risk language tied to drug development, regulatory review, manufacturing, funding needs, competition, and commercialization.
Proxy materials describe annual-meeting matters, board and compensation governance, equity compensation, and stockholder voting. Recent current reports also record restructuring and cost disclosures, employment-related equity plans, and material updates for programs including UX111, UX016, DTX301, DTX401, and GTX-102.
Ultragenyx Pharmaceutical Inc. reported third-quarter revenue of $173 million, up 8% from $160 million a year earlier, while net loss narrowed to $118 million from $180 million. Cash, cash equivalents and marketable securities totaled $401 million as of September 30, 2026; operating activities used $337 million in cash during the first nine months.
FDA granted accelerated approval to GENGLYCOS in August and standard approval to FAYUVI in September. The GENGLYCOS priority review voucher sale closed for $210 million on October 30; the company expects $164 million in net proceeds after a $42 million NIH payment and related fees. The $203 million FAYUVI voucher sale is expected to close by year-end 2026, subject to customary closing conditions, with $185 million in expected net proceeds after a $16 million payment to a third-party licensor.
An October restructuring plan calls for an approximately 20% workforce reduction and estimated charges of approximately $25 million. The Phase 3 GTX-102 study missed its primary and key secondary endpoints, and the program is being wound down. Ultragenyx plans to terminate UX701 after it did not meet the criteria for advancement to pivotal Phase 3.
Ultragenyx Pharmaceutical Inc. (RARE) reported third-quarter 2026 revenue of $173 million, compared with $160 million a year earlier, and a net loss of $118 million, versus $180 million. CRYSVITA revenue was $109 million and DOJOLVI revenue was $30 million. The company reaffirmed 2026 total revenue guidance of $730 million to $760 million, excluding revenue from new product launches. Cash, cash equivalents and marketable securities were $401 million as of September 30, 2026.
Ultragenyx completed the GENGLYCOS priority review voucher sale for $210 million in gross proceeds; 20% of the gross proceeds will be paid to NIH. Agreements for both vouchers provide a combined sale price of $413 million; the $203 million FAYUVI sale is expected to close later in November, and net proceeds from both sales are expected to be approximately $350 million. The company expects a workforce reduction of approximately 20%, approximately $25 million in restructuring charges, and substantial completion by year-end 2026. It expects 2027 R&D expenses below $350 million and SG&A expenses below $375 million, and reaffirmed full-year GAAP profitability in 2027.
Ultragenyx Pharmaceutical Inc. (RARE) common stock was reported as beneficially owned by Vanguard Portfolio Management in an amount of 8,481,266 shares, or 8.6% of the class, as of September 30, 2026. The ownership note says the reported position reflects securities beneficially owned or deemed beneficially owned by Vanguard Portfolio Management LLC and certain affiliates, including Vanguard Fiduciary Trust Company and Vanguard Global Advisers, LLC. Vanguard reported sole dispositive power over 8,481,266 shares and sole voting power over 62,161 shares; shared voting and dispositive power were both zero. Vanguard and managed accounts were identified as having the right to receive or direct dividends or sale proceeds, and no other person's interest in the reported securities exceeded 5%.
Ultragenyx Pharmaceutical Inc. (RARE) shares were reported as beneficially owned by Madison Avenue International LP, which directly owned 6,307,798 shares as of October 20, 2026. Madison Avenue Partners, LP, EMAI Management, LLC, Madison Avenue GP, LLC, Caraway Jackson Investments LLC and Eli Samaha may also be deemed beneficial owners of those same shares through their stated relationships with Madison Avenue International LP.
The six reporting persons reported shared voting and dispositive power over 6,307,798 shares. The position represented approximately 6.4% of the common stock, based on 98,588,873 shares outstanding as of June 30, 2026.
Ultragenyx Pharmaceutical Inc. (RARE) Chief Financial Officer Howard Horn reported a direct sale of 7,066 shares on October 15, 2026, at $15.27 per share. The shares were sold to pay required tax withholdings due to RSU vesting. Horn reported 89,229 shares after the transaction; no Rule 10b5-1 plan is reported.
Ultragenyx Pharmaceutical Inc. (RARE) and Baylor Research Institute entered into a settlement agreement with Navinta LLC resolving patent litigation over DOJOLVI (triheptanoin) oral liquid. Ultragenyx will grant Navinta a non-exclusive, royalty-free license to market a generic version in the United States beginning January 1, 2033; if pediatric exclusivity is granted for DOJOLVI, the start date would extend to July 1, 2033, subject to customary conditions and exceptions. Similar patent litigation brought by Ultragenyx against other ANDA filers remains pending in the U.S. District Court for the District of New Jersey. The settlement agreement will be submitted to the U.S. Federal Trade Commission and the U.S. Department of Justice as required by law.
On October 8, 2026, Ultragenyx Pharmaceutical Inc. entered into an asset purchase agreement to sell a Rare Pediatric Disease Priority Review Voucher for $203 million in cash, payable upon closing. The FDA awarded the voucher on September 17, 2026, upon approval of UX111 (rebisufligene etisparvovec-hopf) for pediatric patients with MPS IIIA, or Sanfilippo syndrome Type A. The transaction remains subject to customary closing conditions, including expiration or termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976.
Ultragenyx Pharmaceutical Inc. (RARE) entered into an agreement on October 6, 2026, to sell a Rare Pediatric Disease Priority Review Voucher (PRV). The buyer agreed to pay $210 million in cash upon closing. Under a patent license agreement dated December 10, 2018, Ultragenyx will pay NIH 20% of the gross proceeds from the PRV sale after closing.
The transaction remains subject to customary closing conditions, including expiration or termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976. FDA awarded Ultragenyx the PRV on August 19, 2026, upon approval of GENGLYCOS (pariglasgene brecaparvovec-opnr), also known as DTX401, in adult and pediatric patients eight years and older with glycogen storage disease type Ia. The agreement includes customary representations, warranties, covenants and indemnification provisions, subject to certain limitations.
Ultragenyx Pharmaceutical Inc. (RARE) announced that the U.S. Food and Drug Administration granted standard full approval of FAYUVI™ (rebisufligene etisparvovec-hopf), also known as UX111, for treating pediatric patients with mucopolysaccharidosis type IIIA (Sanfilippo syndrome Type A).
FAYUVI is described as the first FDA-approved treatment for this progressive and fatal neurodegenerative disease and the second gene therapy approval for Ultragenyx. The company also received a Priority Review Voucher in connection with this approval.
Ultragenyx Pharmaceutical Inc. (RARE) senior vice president and chief accounting officer Theodore Alan Huizenga reported selling 1,283 shares of common stock on September 15, 2026, at $13.57 per share in an open-market or private transaction pursuant to a Rule 10b5-1 trading plan. Following this sale, he reported holding 67,863 shares directly, including shares underlying restricted stock units that remain subject to vesting conditions.