Welcome to our dedicated page for Ultragenyx Pharm news (Ticker: RARE), a resource for investors and traders seeking the latest updates and insights on Ultragenyx Pharm stock.
Ultragenyx Pharmaceutical Inc. develops and commercializes therapies for serious rare and ultra-rare genetic diseases. Company news commonly covers revenue from approved medicines such as Crysvita, Dojolvi, and Mepsevii, financial guidance, and updates to a portfolio of approved therapies and product candidates for diseases with high unmet medical need and clear biology.
Recurring developments include FDA interactions and clinical data for investigational programs such as UX111 for Sanfilippo syndrome type A, GTX-102 for Angelman syndrome, UX016 for GNE myopathy, and AAV gene therapy candidates for rare metabolic disorders. Updates also include healthcare conference participation, employment inducement equity grants under Nasdaq rules, and corporate restructuring or expense-management actions when disclosed.
Ultragenyx (NASDAQ: RARE) announced that the U.S. FDA has granted accelerated approval for GENGLYCOS (pariglasgene brecaparvovec-opnr), also known as DTX401, for adult and pediatric patients ≥8 years with glycogen storage disease type Ia (GSDIa). GENGLYCOS is the first FDA‑approved treatment designed to address the underlying cause of GSDIa and Ultragenyx’s first approved gene therapy, and it reduces daily cornstarch intake as an adjunct to nutritional management. Approval is based on the 48‑week randomized, double‑blind, placebo‑controlled Phase 3 GlucoGene study in 46 participants, in which DTX401 significantly reduced cornstarch requirements versus placebo (p<0.001). Continued approval may depend on verification of clinical benefit through a post‑marketing program including 2‑year data from 50 commercial patients and 20 controls and long‑term follow‑up via the GSDIa Disease Monitoring Program for up to 10 years. Ultragenyx also received a Priority Review Voucher and will supply GENGLYCOS via Qualified Treatment Centers, with in‑house manufacturing at its Bedford, Massachusetts facility and patient support through the UltraCare program.
Ultragenyx (NASDAQ: RARE) reported second quarter 2026 total revenue of $214 million, up from $167 million a year ago, led by Crysvita revenue of $156 million and Dojolvi revenue of $27 million. Evkeeza contributed $21 million and Mepsevii $10 million.
According to Ultragenyx, net loss narrowed to $92 million, or $0.90 per share, versus a $115 million loss, or $1.17 per share, in Q2 2025. Total operating expenses were $289 million, including $34 million of non‑cash stock-based compensation.
The company reaffirmed 2026 guidance for total revenue of $730–$760 million, Crysvita revenue of $500–$520 million, and Dojolvi revenue of $100–$110 million, and expects combined R&D and SG&A expenses in 2026 to be flat to down low-single digits versus 2025 and to decrease by at least 15% in 2027, supporting its stated path to profitability in 2027.
Ultragenyx highlighted near‑term catalysts including two FDA PDUFA action dates for DTX401 (August 23, 2026) and UX111 (September 19, 2026), and Phase 3 GTX‑102 Aspire data for Angelman syndrome expected in September or October 2026. As of June 30, 2026, cash, cash equivalents, and marketable securities were $436 million, with net cash used in operations of $97 million in the quarter.
Ultragenyx (NASDAQ: RARE) will host a conference call at 5:00 p.m. ET on Tuesday, August 4, 2026, to review its financial results and corporate update for the quarter ended June 30, 2026. A live and replay webcast will be accessible via the company’s investor relations website, with the replay available for three months.
Ultragenyx (NASDAQ: RARE) granted 65,886 restricted stock units (RSUs) of its common stock to 39 newly hired non-executive officers under the Ultragenyx Employment Inducement Plan. The RSUs, granted on July 16, 2026, vest over four years at 25% per year, subject to continued employment.
Ultragenyx (NASDAQ: RARE) released its 2025 Impact Report highlighting progress for rare and ultra-rare disease patients across innovation, patient access, people, communities, planet, and governance.
Key 2025 metrics include 62% of operating expenses invested in R&D, five pivotal clinical programs, and support for 700+ patients in 50 countries.
Ultragenyx (NASDAQ: RARE) granted 44,409 restricted stock units to 15 newly hired non-executive officers under its Employment Inducement Plan. The awards, approved by the compensation committee, were granted on June 16, 2026, as inducements in accordance with Nasdaq Listing Rule 5635(c)(4).
The RSUs vest over four years, with 25% of shares vesting on each anniversary of the grant date, contingent on continued employment.
Ultragenyx (NASDAQ:RARE) announced that president and CEO Emil Kakkis, M.D., Ph.D., will participate in a fireside chat at the Goldman Sachs 47th Annual Global Healthcare Conference on June 9, 2026, at 4:00 PM ET.
A live and archived webcast will be available on Ultragenyx’s investor relations website.
Ultragenyx (NASDAQ: RARE) reported an inducement equity grant under Nasdaq Listing Rule 5635(c)(4).
The company granted 17,567 restricted stock units of common stock to 11 newly hired non-executive officers on May 16, 2026, under the Ultragenyx Employment Inducement Plan. The RSUs vest over four years, in 25% annual installments.
Ultragenyx (NASDAQ: RARE) will participate in Bank of America’s 2026 Healthcare Conference. Company executives Howard Horn, CFO and EVP, and Joshua Higa, Chief of Staff and VP Investor Relations, will appear in a fireside on May 12, 2026 at 2:20 PM PT.
A live and archived webcast will be available on the company’s investor site at https://ir.ultragenyx.com/events-presentations.
Ultragenyx (NASDAQ: RARE) reported Q1 2026 revenue of $136 million with Crysvita $93M, Dojolvi $18M and reaffirmed 2026 guidance of $730–$760M total revenue. The company reaffirmed flat-to-slightly-down 2026 combined R&D and SG&A versus 2025 and a path to profitability in 2027.
Key clinical and regulatory milestones include FDA acceptance and PDUFA dates for DTX401 (Aug 23, 2026) and UX111 (Sep 19, 2026), Phase 3 Aspire data for GTX-102 expected H2 2026, and $534M cash and marketable securities as of March 31, 2026.