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. (NASDAQ: RARE) delivers innovative therapies for rare genetic disorders through targeted research and biomarker-driven clinical programs. This page consolidates official announcements and verified updates about the company's therapeutic advancements, regulatory progress, and strategic initiatives.
Investors and medical professionals will find timely updates on clinical trial outcomes, FDA designations, and partnership developments. The curated news collection includes earnings reports, research milestones, and corporate updates relevant to rare disease treatment innovation.
Key content focuses on therapy approvals, orphan drug status achievements, and collaborations with patient advocacy networks. All materials adhere to regulatory disclosure standards while maintaining accessibility for both clinical experts and general investors.
Bookmark this page for centralized access to Ultragenyx's latest developments in addressing unmet medical needs through precision biopharmaceutical solutions.
Ultragenyx Pharmaceutical (NASDAQ: RARE) has announced the grant of 36,831 restricted stock units to 21 newly hired non-executive officers. The awards were approved by the company's compensation committee and issued under the Ultragenyx Employment Inducement Plan on April 16, 2025.
The stock units feature a four-year vesting schedule, with 25% of shares vesting annually on the grant date anniversary, contingent on continuous employment. This grant was made in accordance with Nasdaq Listing Rule 5635(c)(4) as an employment inducement for new hires.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the grant of 21,806 restricted stock units to 16 newly hired non-executive officers. The awards, approved by the compensation committee and granted under the Ultragenyx Employment Inducement Plan on March 16, 2025, serve as an employment inducement in compliance with Nasdaq Listing Rule 5635(c)(4). The restricted stock units feature a four-year vesting schedule, with 25% of shares vesting annually on the grant date anniversary, contingent on continuous employment.
Ultragenyx Pharmaceutical (NASDAQ: RARE), a biopharmaceutical company specializing in novel therapies for rare and ultrarare genetic diseases, has announced its participation in three major investor conferences in March 2025.
The company's Chief Medical Officer, Dr. Eric Crombez, will represent Ultragenyx at:
- The 45th Annual Cowen Healthcare Conference in Boston on March 3
- The Barclays 27th Annual Global Healthcare Conference in Miami on March 11
- The Leerink Partners Global Biopharma Conference in Miami on March 12
At each event, Dr. Crombez will participate in a fireside chat and conduct one-on-one meetings. Investors can access live and archived webcasts of the fireside chats through Ultragenyx's investor relations website.
Ultragenyx Pharmaceutical (NASDAQ: RARE) has announced the grant of 23,760 restricted stock units to 10 newly hired non-executive officers. The compensation committee approved these awards under the Ultragenyx Employment Inducement Plan, with a grant date of February 16, 2025. The grants, made in accordance with Nasdaq Listing Rule 5635(c)(4), serve as employment inducements for the new hires.
The restricted stock units have a four-year vesting schedule, with 25% of the shares vesting annually on the grant date anniversary, contingent on continuous employment.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced FDA acceptance of their Biologics License Application (BLA) for UX111, an AAV gene therapy targeting Sanfilippo syndrome type A (MPS IIIA). The FDA granted Priority Review with a PDUFA date of August 18, 2025, and indicated no current plans for an advisory committee meeting.
The BLA submission is supported by data from the pivotal Transpher A and long-term follow-up studies presented at WORLDSymposium™ 2025. Results showed rapid and sustained decreases in heparan sulfate levels in cerebrospinal fluid, with statistically significant improvements in Bayley-III scores for cognition and communication compared to untreated patients. Treatment-related adverse events were primarily mild to moderate liver enzyme elevations that resolved.
Ultragenyx Pharmaceutical (NASDAQ: RARE) reported strong financial results for Q4 and full year 2024, with total revenue of $560 million, representing 29% growth compared to 2023. Key revenue drivers included Crysvita at $410 million (+25% YoY) and Dojolvi at $88 million (+25% YoY).
The company provided 2025 guidance projecting total revenue between $640-670 million, with Crysvita revenue of $460-480 million and Dojolvi revenue of $90-100 million. Despite revenue growth, Ultragenyx reported a net loss of $569 million ($6.29 per share) for 2024, though improved from $607 million loss in 2023.
The company ended 2024 with $745 million in cash and equivalents, with net cash used in operations of $414 million for the year. Multiple clinical milestones are expected in 2025, including a PDUFA decision for Sanfilippo syndrome gene therapy and Phase 3 results in osteogenesis imperfecta.
Ultragenyx Pharmaceutical (NASDAQ: RARE), a biopharmaceutical company specializing in novel products for rare and ultrarare genetic diseases, has announced a conference call scheduled for February 13, 2025, at 5:00 p.m. ET. The call will discuss the company's financial results and provide a corporate update for both the fourth quarter and full year ending December 31, 2024. Investors and interested parties can access the live webcast and replay through the company's investor relations website. The replay will remain available for three months following the call.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced significant clinical improvements in children treated with UX111 AAV gene therapy for Sanfilippo Syndrome Type A. The modified intent-to-treat group showed a +22.7 point treatment effect in Bayley-III cognitive raw scores compared to untreated patients, with a p-value <0.0001.
The treatment demonstrated substantial reductions in heparan sulfate levels in cerebrospinal fluid, with a median reduction of 65% across all patients (N=27) and 66% in the modified intention to treat group (N=17). The mean follow-up duration was 34 months for all patients, with the longest being 77 months.
In older patients with advanced disease, 9 out of 10 children retained abilities in ambulation and self-feeding, while all 10 maintained communication skills. The therapy was generally well-tolerated, with mostly mild to moderate liver enzyme elevations that resolved. A BLA submission was filed with the FDA in December 2024, with a PDUFA decision expected in H2 2025.
Ultragenyx Pharmaceutical (NASDAQ: RARE), a biopharmaceutical company specializing in rare and ultrarare disease therapies, has announced the grant of 7,750 restricted stock units to eight newly hired non-executive officers. The compensation committee approved these awards under the Ultragenyx Employment Inducement Plan on January 16, 2025. The grants serve as employment inducements in compliance with Nasdaq Listing Rule 5635(c)(4). The restricted stock units feature a four-year vesting schedule, with 25% of shares vesting annually on the grant date anniversary, contingent on continuous employment.
Ultragenyx Pharmaceutical (NASDAQ: RARE) reported preliminary 2024 revenue of $555-560 million, exceeding guidance, with Crysvita revenue of $405-410 million and Dojolvi revenue of $87-89 million. The company projects 2025 total revenue of $640-670 million.
Key pipeline updates include: UX143 Phase 3 Orbit study for osteogenesis imperfecta progressing to second interim analysis in mid-2025; GTX-102 Phase 3 Aspire study for Angelman syndrome expected to complete enrollment in second half of 2025; BLA submission for UX111 gene therapy for Sanfilippo syndrome with PDUFA decision expected in second half of 2025; and DTX401 BLA filing for Glycogen Storage Disease Type Ia planned for mid-2025.
The company ended 2024 with approximately $745 million in cash and investments, expecting 14-20% revenue growth in 2025 with decreased net cash usage in operations.
