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.
The Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) SEC filings page provides access to the company’s official disclosures filed with the U.S. Securities and Exchange Commission. These documents include current reports on Form 8-K, quarterly and annual reports, and other filings that describe material agreements, financial results, and key developments in Ultragenyx’s rare disease business.
Form 8-K filings for Ultragenyx often cover topics such as royalty purchase agreements, clinical and regulatory milestones, and financial updates. For example, the company has reported a Royalty Purchase Agreement with an OMERS investment vehicle involving a portion of future Crysvita royalty payments in the United States and Canada, including details on purchase price, royalty percentages, and caps based on multiples of the purchase price. Other 8-Ks summarize press releases announcing quarterly financial results, revenue by product, operating expenses, and net loss.
Ultragenyx also uses Form 8-K to report clinical and regulatory events, including initiation and progress of rolling Biologics License Application submissions for DTX401 in glycogen storage disease type Ia, longer-term Phase 3 data for DTX401, Breakthrough Therapy Designation for GTX-102 in Angelman syndrome, enrollment status of the Phase 3 Aspire study, and FDA communications such as a Complete Response Letter for UX111 in Sanfilippo syndrome type A. These filings outline requested chemistry, manufacturing, and controls information and the company’s plans to address regulatory feedback.
Through Stock Titan, users can review Ultragenyx’s SEC filings and benefit from AI-powered summaries that explain the significance of complex documents, including 10-K and 10-Q reports when available, as well as Form 4 insider transaction reports. Real-time updates from EDGAR combined with AI-generated insights help readers understand how material agreements, clinical data, and regulatory interactions influence Ultragenyx’s operations and capital structure in the rare and ultra-rare genetic disease space.
Howard Horn reported Form 144 sales of RARE securities. The filing lists proposed sales of 674 performance shares and 961 restricted shares dated
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
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 LLCUltragenyx 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
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.