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.
Theodore Alan Huizenga, SVP and Chief Accounting Officer and director of Ultragenyx Pharmaceutical Inc. (RARE), reported a sale of 64 shares of common stock on 09/18/2025 at a price of $29.17 per share. The filing states the shares were sold to pay required tax withholdings related to the vesting of restricted stock units (RSUs). After the transaction, the reporting person beneficially owned 50,242 shares. The Form 4 was signed by an attorney-in-fact on 09/19/2025.
Theodore Alan Huizenga, SVP and Chief Accounting Officer and director of Ultragenyx Pharmaceutical Inc. (RARE), reported a sale of 64 shares of common stock on 09/18/2025 at a price of $29.17 per share. The filing states the shares were sold to pay required tax withholdings related to the vesting of restricted stock units (RSUs). After the transaction, the reporting person beneficially owned 50,242 shares. The Form 4 was signed by an attorney-in-fact on 09/19/2025.
Ultragenyx Pharmaceutical Inc. reported positive longer-term Phase 3 results for its DTX401 gene therapy in glycogen storage disease type Ia. Patients treated with DTX401 showed a 41% mean reduction in daily cornstarch intake at Week 48 versus 10% with placebo, while maintaining glycemic control. By Week 96, both the original DTX401 group and a crossover group achieved a 61% mean reduction in daily cornstarch, with nighttime cornstarch reduced by 70% and 75% respectively and two-thirds of participants eliminating at least one nighttime dose. Quality-of-life measures improved, with 83% and 95% of patients in the two groups reporting lower disease burden, and safety remained acceptable without observed AAV8 class effects such as dorsal root ganglion toxicity, malignancy or thrombotic microangiopathy.
Theodore A. Huizenga, SVP and Chief Accounting Officer of Ultragenyx Pharmaceutical Inc. (RARE), reported a sale of company common stock on 09/05/2025. The Form 4 shows a sale (Code S) of 84 shares at $31.39 per share. After the reported transaction the reporting person beneficially owned 50,306 shares directly. The filing explains the sale represents shares sold to pay required tax withholdings upon vesting of restricted stock units, and also notes the reporting person acquired 266 shares under the company’s Employee Stock Purchase Plan on April 30, 2025. Previously reported RSU-related shares subject to vesting are included in the total.
Ultragenyx Pharmaceutical Inc. reported that it has begun a rolling submission of a Biologics License Application to the U.S. FDA for DTX401, an AAV gene therapy for Glycogen Storage Disease Type Ia. The company has submitted the non-clinical and clinical modules and plans to complete the full application, including the chemistry, manufacturing and controls module, in the fourth quarter of 2025.
The BLA will include 96-week data from a Phase 3 trial showing patients had greater reductions in total daily cornstarch from baseline at their last visit, with a -60% reduction in the ongoing DTX401 group and -64% in the Crossover Placebo to DTX401 group compared to the 48-week data. Ultragenyx also plans to incorporate updates in the CMC section to proactively address FDA observations from the UX111 complete response letter and at its gene therapy manufacturing facilities.
Ultragenyx (Nasdaq: RARE) filed an 8-K disclosing receipt of FDA Breakthrough Therapy Designation for GTX-102 (apazunersen) to treat Angelman syndrome.
The decision is supported by positive Phase 1/2 data in 74 children (ages 4-17) showing rapid, sustained and continuing developmental gains over up to three years.
Breakthrough status grants intensive FDA guidance, rolling review and potential priority review, which could materially shorten development timelines and lower regulatory risk.
The filing includes customary forward-looking statements covering clinical, regulatory and manufacturing uncertainties but announces no new financial terms.
Form 4 filing reveals insider trading activity at Ultragenyx Pharmaceutical (RARE) by Director Corazon (Corsee) D. Sanders. On June 20, 2025, Sanders executed a significant sale of company shares:
- Disposed of 2,405 shares at a price of $37.39 per share
- Transaction value: approximately $89,873
- Retained ownership: 15,344 shares held directly
This insider sale represents a notable reduction in Sanders' direct holdings in Ultragenyx. The transaction was executed as a standard sale (transaction code 'S') and was reported within the required SEC filing deadline. No derivative securities transactions were reported in this filing.