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 focuses on therapies for serious rare and ultra-rare genetic diseases and reports a broad mix of approved drugs and late-stage programs. Approved products include Crysvita for X-linked hypophosphatemia and tumor-induced osteomalacia, Mepsevii for MPS VII, Dojolvi for long-chain fatty acid oxidation disorders, and Evkeeza for homozygous familial hypercholesterolemia in partnered territories.
The company highlights an advanced pipeline across four modalities: biologics, small molecules, AAV gene therapy, and nucleic acids. Key programs include UX111 for Sanfilippo syndrome type A, where a resubmitted BLA received an Incomplete Response Letter focused on CMC documentation, and DTX401 for glycogen storage disease type Ia, supported by 96-week Phase 3 data and a completed rolling BLA with an expected PDUFA action date in the third quarter of 2026.
Ultragenyx is also running pivotal studies for Angelman syndrome (GTX-102), OTC deficiency (DTX301), Wilson disease (UX701), and osteogenesis imperfecta (UX143). The company notes that Phase 3 Orbit and Cosmic studies for UX143 did not meet their primary fracture endpoints, and additional analyses are underway to assess a potential path forward. As of June 30, 2025, non-affiliate equity market value was approximately $3.4 billion, and 96,629,788 shares of common stock were outstanding as of February 13, 2026.
Ultragenyx Pharmaceutical reported strong 2025 revenue growth but continued heavy losses while launching a major cost-cutting plan. Total revenue reached $673 million, up 20% year over year, led by Crysvita at $481 million and Dojolvi at $96 million. Fourth-quarter revenue was $207 million, up 25% from 2024. Despite this, the company posted a 2025 net loss of $575 million, or $5.83 per share, and operating expenses of $1.2 billion, including $153 million of stock-based compensation.
Ultragenyx initiated a strategic restructuring with a 10% workforce reduction, affecting about 130 employees, and expects about $50 million in restructuring and related charges, mainly from severance and terminating UX143 manufacturing agreements. Cash, cash equivalents and marketable securities were $737 million at year-end 2025. For 2026, the company guides total revenue from current products of $730–$760 million, Crysvita revenue of $500–$520 million, and Dojolvi revenue of $100–$110 million, and reiterates a path to profitability in 2027 with a planned 38% reduction in R&D spending versus 2025 and at least a 15% combined R&D and SG&A reduction.
FMR LLC and Abigail P. Johnson filed an amended Schedule 13G reporting a passive ownership stake in Ultragenyx Pharma Inc.3.5% of the outstanding common stock as of the event date of 12/31/2025.
FMR LLC has sole voting power over 3,279,798 shares and sole dispositive power over 3,347,890.21 shares. Abigail P. Johnson has sole dispositive power over the same 3,347,890.21 shares but no voting power. The filing states the shares are held in the ordinary course of business and not for the purpose of changing or influencing control of Ultragenyx, with control-related activities limited to those connected to a nomination under Rule 14a-11.
Ultragenyx Pharmaceutical Inc.'s Chief Financial Officer, Howard Horn, reported a stock sale. On 02/02/2026, he sold 3,061 shares of Ultragenyx common stock at a price of $23.56 per share.
After this transaction, he beneficially owns 88,935 shares of common stock. This figure includes previously reported shares underlying restricted stock units (RSUs) that were granted to him and are still subject to vesting conditions.
Ultragenyx Pharmaceutical reported new long‑term clinical data for UX111, its investigational AAV9 gene therapy for Sanfilippo syndrome type A, and has resubmitted a Biologics License Application seeking accelerated FDA approval with a PDUFA date expected in the third quarter of 2026.
In younger or earlier‑stage children (n=17), treatment produced a +23.2‑point improvement in Bayley‑III cognitive raw scores versus natural history, with additional gains in receptive (8.1 points), expressive (11.1 points), fine motor (9.0 points), and gross motor (3.9 points) function. Eight children reached a 36‑month cognitive developmental age, which natural‑history patients did not achieve.
In later‑stage patients (n=10), most retained communication, independent walking, and ability to eat by mouth beyond typical decline ages. Across the overall efficacy set (N=27), cerebrospinal fluid heparan sulfate fell rapidly, with a median 63.98% reduction and most children achieving at least a 50% decrease. UX111 was generally well tolerated in 33 treated patients over a median 4.8‑year follow‑up, with mainly mild to moderate, reversible liver enzyme elevations and no reported treatment‑associated hypersensitivity, thrombotic microangiopathy, myocarditis, dorsal root ganglion toxicity, or malignancy.
Howard Horn filed a notice to sell 3,061 common shares through Morgan Stanley Smith Barney, with an aggregate market value of 72,117.16. The shares relate to restricted stock acquired from the issuer on 10/09/2024 and 10/09/2025. The planned sale is expected around 02/02/2026 on the NASDAQ, where 96,477,569 shares of this class were outstanding. In the prior three months, Howard Horn sold 3,081 shares for gross proceeds of 112,086.78 and 3,150 shares for 71,946.00.
Ultragenyx Pharmaceutical Inc. has resubmitted its Biologics License Application seeking accelerated approval for UX111, an AAV9 gene therapy for Sanfilippo syndrome type A, to the U.S. FDA. The filing adds longer-term neurologic, biomarker, and safety data, and addresses prior CMC-related observations from a July 2025 Complete Response Letter.
The FDA previously granted Priority Review to the UX111 application, and a new Prescription Drug User Fee Act (PDUFA) action date is expected to be assigned within about a month, with the company anticipating a review period of up to six months and a PDUFA date in the third quarter of 2026. If approved, UX111 would become the first approved therapy for Sanfilippo syndrome type A.
Ultragenyx Pharmaceutical Inc. filed a current report to note that it has issued a press release with preliminary unaudited fiscal year 2025 results. The release covers total revenue for the year, as well as revenue contributions from its key products Crysvita® and Dojolvi®, plus the company’s cash and investment balances at the end of the 2025 fiscal year. Ultragenyx plans to report full audited results for the 2025 fiscal year in February 2026. The company specifies that this preliminary financial information and the accompanying press release are being furnished rather than filed under securities laws.
Ultragenyx Pharmaceutical (RARE) reported higher Q3 revenue and a wider loss. Total revenues were $159.9 million, up from $139.5 million a year ago, driven by product sales of $95.0 million and Crysvita royalties of $64.9 million. Crysvita revenue across product sales and royalties reached $111.9 million.
The company posted a net loss of $180.4 million (basic and diluted loss per share $1.81) versus a loss of $133.5 million last year, reflecting increased R&D and SG&A. For the first nine months, revenue was $465.7 million and net loss was $446.4 million.
Cash and cash equivalents were $202.5 million, with $244.8 million of marketable debt securities (current and non‑current). Total assets were $1.19 billion against total liabilities of $1.17 billion, leaving stockholders’ equity at $9.2 million. Liabilities for sales of future royalties totaled $829.3 million. As of October 30, 2025, 96,477,569 shares of common stock were outstanding.
Ultragenyx (RARE) entered a Royalty Purchase Agreement with an OMERS affiliate, receiving $400 million in cash in exchange for a defined share of future Crysvita royalties from Kyowa Kirin in the U.S. and Canada. OMERS will receive an additional 25% of royalties starting January 1, 2028, and 30% after the prior Royalty Cap is met.
The arrangement ends when OMERS has received 1.55 times the purchase price ($620 million) or when royalties under the license end. Ultragenyx also obtained a two‑year option to repurchase the interest for 1.35 times the purchase price ($540 million). The agreement includes customary terms. Separately, the company furnished a press release announcing results for the quarter ended September 30, 2025.