Ultragenyx Releases Impact Report Emphasizing Commitment to Rare Disease Patients, Innovation, and Global Impact

Ultragenyx Pharmaceutical Inc. released its 2025 Impact Report, detailing the company's work on behalf of patients and families living with rare and ultra-rare diseases. In 2025, Ultragenyx advanced five investigational therapies in pivotal clinical programs with the potential to reach tens of thousands of patients around the world — while continuing to build on years of work to support patients who cannot yet access approved treatments, which have now reached individuals in 50 countries.

“The rare disease community has waited long enough — every program we advance, every trial site we open, and every patient we support through access to approved medicines is a reflection of that belief," said Emil Kakkis, M.D., Ph.D., founder, president, and chief executive officer of Ultragenyx. "Our 2025 Impact Report reflects the progress we are making today to deliver urgently needed, first-ever therapies, as well as our commitment to shaping a more accessible future for rare diseases.”

The report outlines progress across six key pillars — Innovation, Patients, People, Communities, Planet, and Governance — as well as Sustainability Accounting Standards Board (SASB), Global Reporting Initiative (GRI), and Task Force on Climate Related Disclosures (TCFD) indices. Updates reflect Ultragenyx’s ongoing focus on delivering meaningful, long-term impact for patients and families impacted by rare and ultra-rare diseases and cover activities from January 1 through December 31, 2025. For the full report and more on Ultragenyx’s Corporate Responsibility efforts, visit here.

Highlights from 2025 include:
Innovation
Ultragenyx continued to expand access to its four approved therapies across five indications and advanced its pipeline with a singular focus on addressing significant unmet medical needs for rare disease patients:

• Invested approximately 62% of operating expenses in research and development in 2025
• Drove progress on five clinical-stage investigational therapies with the potential to reach tens of thousands of patients
• Applied its Dynamic Development Model (DDM) — centered around direct patient and caregiver input — to inform clinical trial design and accelerate clinical development taking place across 160+ clinical trial sites in 19 countries

Patients
Ultragenyx remained committed to improving access, advancing advocacy, and partnering with the rare disease community.

• Hosted two Rare Bootcamps, including the first held on the U.S. East Coast, to support patient-led drug development initiatives
• Supported more than 700 patients in 50 countries through expanded access and patient assistance programs since 2013
• Participated in over 45 global patient advocacy events and engagements in 2025
• Launched Act for Ultra-Rare, initiating policy discussions to advocate for legislation that supports the development of treatments for ultra-rare diseases to help ensure that these patient communities are not left behind

People
Ultragenyx invested in its global workforce through engagement, inclusion, and professional development.

• Maintained strong employee engagement, with an overall engagement score of 86%
• Delivered more than 80 employee learning and development workshops in 2025
• Achieved 92% participation in the annual employee engagement survey

Communities
Ultragenyx expanded its philanthropic impact and community engagement efforts globally.

• Supported ~180 organizations across ~230 grant programs focused on education, awareness, and patient advocacy
• Contributed more than 2,800 employee volunteer hours supporting local and global initiatives and hosted the third annual Global Days of Service, encompassing nearly 40 volunteer projects worldwide
• Approved approximately $3.3 million in charitable donations, medical education, and health-related grants across more than 20 countries

Planet
Ultragenyx advanced environmental sustainability initiatives across its operations.

• Purchased ~100% renewable electricity for its Novato, CA headquarters campus and Bedford, MA Gene Therapy Manufacturing Facility, and diverted tens of thousands of pounds of waste through recycling and sustainable lab practices
• Initiated its second My Green Lab certification in Somerville, MA, building on a ‘Green’ level certification in Novato, CA
• Conducted its inaugural Climate Risk Assessment to evaluate physical and transition risks across operations and supply chain

Governance
Ultragenyx focused on strong corporate governance, compliance, and ethical business practices.

• Continued to integrate corporate responsibility oversight through board-level governance structures
• Maintained a comprehensive compliance program aligned with global regulatory standards
• Reported no material data privacy breaches or cybersecurity incidents in 2025

Recognition
Ultragenyx’s achievements in 2025 were recognized through multiple awards and honors.

• Named a Top Place to Work in the USA and recognized by The Boston Globe and San Francisco Chronicle
• Received multiple Top Workplaces Culture Excellence Awards, including recognition for compensation, well-being, innovation, leadership, and professional development
• Honored for excellence in patient engagement, including recognition from the rare disease community

About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the company's website here.
Press Release via ultragenyx.com.