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) 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.
Ultragenyx (NASDAQ: RARE) will host a conference call on Tuesday, May 5, 2026 at 5:00 p.m. ET to discuss first quarter 2026 financial results and provide a corporate update. The live webcast and a replay (available for three months) will be on the company's investor website.
Ultragenyx (NASDAQ: RARE) reported an inducement grant under Nasdaq Listing Rule 5635(c)(4) consisting of 36,447 restricted stock units awarded to 12 newly hired non-executive officers with a grant date of April 16, 2026.
The RSUs vest over four years with 25% vesting on each anniversary, conditional on continued employment.
Ultragenyx (NASDAQ: RARE) announced the U.S. FDA has accepted the resubmitted BLA for UX111 (rebisufligene etisparvovec) AAV9 gene therapy for Sanfilippo syndrome Type A (MPS IIIA). The FDA set a PDUFA action date of September 19, 2026 and previously granted Priority Review in February 2025.
Updated long-term clinical data in the filing show up to 8 years of follow-up with durable clinical improvements versus natural history and supportive biomarker evidence, and the submission reports an acceptable safety profile. If approved, UX111 would be the first therapy for MPS IIIA and be manufactured in the U.S.
Ultragenyx (NASDAQ: RARE) announced FDA clearance of an Investigational New Drug application for UX016, a sialic acid prodrug for treatment of GNE myopathy. The externally funded program plans a first-in-human Phase 1/2 study starting in the second half of 2026 to enroll ~24 adults.
UX016 is designed to improve muscle delivery of sialic acid via a fatty-acid prodrug tail; the study will assess safety, pharmacokinetics, and muscle delivery, plus efficacy measures through Week 48.
Summary not available.
Ultragenyx (NASDAQ: RARE) reported an inducement grant of 10,839 restricted stock units to two newly hired non-executive officers under the Ultragenyx Employment Inducement Plan, with a grant date of March 16, 2026.
The awards were approved by the compensation committee and vest over four years, with 25% of underlying shares vesting on each anniversary, subject to continued employment, in accordance with Nasdaq Listing Rule 5635(c)(4).
Ultragenyx (NASDAQ: RARE) reported positive 36-week Phase 3 results for DTX301 in OTC deficiency, showing a statistically significant 18% reduction in 24-hour plasma ammonia AUC0-24 versus placebo (p=0.018) and maintenance of average ammonia in the normal range through Week 36.
Patients reduced ammonia scavenger drugs by a mean 27% and increased protein intake ~13% while on DTX301; safety was acceptable with one treatment-related acute hepatitis that resolved. Additional 64-week treatment-burden data are expected in H1 2027.