Welcome to our dedicated page for Ultragenyx Pharm SEC filings (Ticker: RARE), a comprehensive resource for investors and traders seeking official regulatory documents including 10-K annual reports, 10-Q quarterly earnings, 8-K material events, and insider trading forms.
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.
RARE files a Form 144 notice reporting proposed and recent resales of common stock by a selling holder. The filing lists proposed restricted stock to be sold by the issuer dated 10/09/2024 (1744 shares) and 10/09/2025 (1375 shares). It also records three reported sales by Howard Horn on 12/10/2025 (3081 shares, $112,086.78), 01/02/2026 (3150 shares, $71,946.00), and 02/02/2026 (3061 shares, $72,117.16).
Ultragenyx Pharmaceutical Inc. reported that the U.S. Food and Drug Administration has accepted for review its Biologics License Application for DTX401 AAV gene therapy (pariglasgene brecaparvovec) to treat Glycogen Storage Disease Type Ia. The FDA granted the application Priority Review and set a Prescription Drug User Fee Act action date of August 23, 2026. This step marks an important U.S. regulatory milestone for DTX401, though the company highlights that approval is not assured and development and regulatory risks remain.
Ultragenyx Pharmaceutical Inc. reported that the U.S. Food and Drug Administration has accepted for review its Biologics License Application for DTX401 AAV gene therapy (pariglasgene brecaparvovec) to treat Glycogen Storage Disease Type Ia. The FDA granted the application Priority Review and set a Prescription Drug User Fee Act action date of August 23, 2026. This step marks an important U.S. regulatory milestone for DTX401, though the company highlights that approval is not assured and development and regulatory risks remain.
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.