Every 8-K that Ultragenyx Pharmaceutical Inc. (RARE) has filed with the SEC in the last 24 months is listed below, newest first, and each one links through to the document itself with the summary and the scores our analysis gives it.
A 8-K covers material events a company has to report between its quarterly reports, so if you follow RARE and want that one kind of document rather than the whole filing history, this is the page to keep. The company's other filings, of every form, are on the full RARE filings page.
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) reported that its Phase 3 Aspire study of apazunersen (GTX-102) in Angelman syndrome did not meet the primary endpoint of change from Baseline in Bayley-4 cognitive raw score or the key secondary endpoint of net response in the Multidomain Responder Index. The safety profile in Aspire was consistent with the earlier Phase 1/2 study, and randomized groups were comparable at baseline. Ultragenyx plans to evaluate the future of the apazunersen program in light of these results and to assess its planned operations to define and implement significant expense reductions while continuing to support its growing commercial business.
Ultragenyx Pharmaceutical Inc. (RARE) reported that the U.S. Food and Drug Administration granted accelerated approval for GENGLYCOS™ (pariglasgene brecaparvovec-opnr, DTX401) to treat adult and pediatric patients eight years and older with glycogen storage disease type Ia (GSDIa). Approval is based on a 48-week randomized, double-blind, placebo-controlled Phase 3 study in 46 participants, which showed a statistically significant reduction in cornstarch requirements for DTX401-treated patients at a 1.0 x 10^13 GC/kg dose (p<0.001). Ultragenyx agreed to a post-marketing program that will follow 50 commercially treated patients and 20 control patients for at least 2 years, with broader disease monitoring over 10 years to further characterize safety, fasting tolerance, and other clinical outcomes.
Ultragenyx Pharmaceutical Inc. reports that it and Baylor Research Institute entered into a settlement agreement with Esjay Pharma regarding patent litigation over DOJOLVI® (triheptanoin) oral liquid. Esjay had filed an Abbreviated New Drug Application seeking approval to market a generic DOJOLVI in the United States.
Under the settlement, Ultragenyx will grant Esjay a non-exclusive, royalty-free license to market its generic version of DOJOLVI starting January 1, 2033, extendable to July 1, 2033 if pediatric exclusivity is granted, subject to customary conditions and exceptions. Similar litigation with other ANDA filers remains pending, and the agreement will be submitted to the FTC and Department of Justice.
Ultragenyx Pharmaceutical Inc. reported second quarter 2026 results, with total revenue of $214 million, up from $167 million a year earlier and the highest quarterly revenue in its history. Key contributors were Crysvita at $156 million, Dojolvi at $27 million, Evkeeza at $21 million and Mepsevii at $10 million.
The company recorded a net loss of $92 million, or $0.90 per share, compared with a net loss of $115 million, or $1.17 per share, in second quarter 2025. Cash, cash equivalents and marketable securities were $436 million as of June 30, 2026, with $97 million of net cash used in operations in the quarter.
Ultragenyx reaffirmed 2026 guidance for total revenue of $730 million to $760 million, Crysvita revenue of $500 million to $520 million, and Dojolvi revenue of $100 million to $110 million, and continues to target profitability in 2027. Upcoming catalysts include FDA PDUFA action dates for DTX401 on August 23, 2026 and UX111 on September 19, 2026, plus a pivotal Phase 3 GTX-102 data readout in Angelman syndrome.
Ultragenyx Pharmaceutical Inc. reported results of its Annual Meeting of Stockholders held on May 14, 2026. As of the record date of March 23, 2026, 98,317,221 shares of common stock were outstanding and entitled to vote, and 84,786,388 shares were represented in person or by proxy.
Stockholders elected Class I directors Emil D. Kakkis, M.D., Ph.D., Shehnaaz Suliman, M.D., and Daniel G. Welch to serve until the 2029 annual meeting. They also approved the Third Amended and Restated 2023 Incentive Plan, with 50,659,150 votes for and 22,888,600 against.
Shareholders ratified Ernst & Young LLP as independent registered public accounting firm for the fiscal year ending December 31, 2026. In an advisory, non-binding vote, stockholders approved executive compensation, with 70,945,277 votes for and 2,594,517 against.
Ultragenyx Pharmaceutical reported first quarter 2026 revenue of $136 million, slightly below $139 million a year earlier, with Crysvita contributing $93 million, Dojolvi $18 million, Evkeeza $18 million and Mepsevii $7 million. Product sales were $89 million and royalty revenue $47 million.
The company posted a net loss of $185 million, or $1.84 per share, compared with a $151 million loss, or $1.57 per share, in 2025 as total operating expenses rose to $305 million. Cash, cash equivalents and marketable securities were $534 million as of March 31, 2026, down from $737 million at year-end, with $197 million of net cash used in operations.
Ultragenyx reaffirmed its 2026 guidance, targeting total revenue of $730–$760 million and combined R&D and SG&A expenses flat to slightly down versus 2025, and continues to aim for profitability in 2027. The company highlighted two gene therapy BLAs under FDA Priority Review for DTX401 and UX111, with PDUFA action dates on August 23, 2026 and September 19, 2026, respectively, and positive longer-term data from the GTX-102 Angelman syndrome program ahead of a Phase 3 readout in the second half of 2026.
Ultragenyx Pharmaceutical Inc. reported that the U.S. FDA has accepted for review its resubmitted Biologics License Application seeking accelerated approval of UX111 (rebisufligene etisparvovec), an AAV9 gene therapy for Sanfilippo syndrome Type A (MPS IIIA). The FDA set a Prescription Drug User Fee Act (PDUFA) action date of September 19, 2026 for this application.
Ultragenyx Pharmaceutical Inc. announced that the FDA has cleared its Investigational New Drug application for UX016, a small molecule prodrug of sialic acid being developed as a substrate replacement therapy for GNE myopathy, a rare inherited neuromuscular disorder.
The UX016 program is externally funded by a patient group through clinical proof-of-concept, including a planned first-in-human Phase 1/2 study in the United States. This trial, expected to start in the second half of 2026, will enroll about 24 adults with GNE myopathy to evaluate safety, efficacy, pharmacokinetics, and muscle delivery over up to 48 weeks of treatment.
Ultragenyx Pharmaceutical Inc. reported positive Phase 3 Enh3ance results for DTX301, its investigational AAV8 gene therapy for ornithine transcarbamylase (OTC) deficiency. At Week 36, DTX301-treated patients showed an 18% reduction in 24-hour plasma ammonia AUC0-24 versus placebo, with ammonia levels generally maintained in the normal range.
Treated patients reduced ammonia scavenger medications by a mean 27% and increased protein intake by about 13%, while still keeping ammonia controlled. Patient-reported outcomes at Week 24 showed notably higher rates of symptom improvement with DTX301 compared to placebo. DTX301 was described as well tolerated, with most adverse events being mild to moderate hepatic reactions managed with steroids.
The trial continues to a second primary endpoint assessing reduction in treatment burden through 64 weeks, with additional data expected in the first half of 2027. Ultragenyx states that the program fits within its 2026 spending guidance and its goal of achieving profitability in 2027.
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 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.
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.
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 (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.
Ultragenyx reported that the first patient has been dosed in the global Aurora study (NCT07157254) evaluating GTX-102 (apazunersen), an investigational antisense oligonucleotide for Angelman syndrome.
Aurora plans to enroll approximately 60 participants ages one to less than 65 across all genotypes in four cohorts. Cohorts A–C are single-arm; Cohort D is randomized 2:1 to GTX-102 or No Treatment, with a 48-week primary efficacy period and crossover at Week 24. Participants may continue in a long-term extension after study completion.
The Phase 3 Aspire study (NCT06617429) is fully enrolled, with 129 participants ages four to 17 with full maternal UBE3A gene deletion randomized across 28 global sites. Data from Aspire are expected in the second half of 2026.
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.
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.