This month marks ten years of Ultragenyx in Europe, the Middle East and Africa — a milestone that tells the story of our growth as a global company and reaffirms the mission that brought us here.

Ultragenyx was founded with a simple belief: people living with rare diseases do not have time to wait. This urgency has guided us from the beginning – helping us develop first ever treatments for diseases with no approved therapies and invest in innovations like gene therapies. It still drives our work today as we focus on improving the lives of people living with rare diseases around the world.

We believe rare disease innovation can — and should — advance differently: in the EMEA region we work with urgency, scientific ambition and a clear focus on patients and families. It shapes how we approach research, how we engage with regulators and health systems, and how we address the barriers that can stand between science and patient impact.

A Global Mission

Over the past ten years, the EMEA region has been an important part of Ultragenyx’s global mission. Across the region, we have established clinical trial sites and enrolled participants in 14 countries. Today, 39% of Ultragenyx active clinical trial sites are in Europeⁱ . We work with manufacturing partners across the region to supply medicines to patients in the region and globally.

Our EMEA team includes 140 people operating in 15 countries, reaching patients in more than 30 countries with partnersⁱⁱ . We’ve brought to the region four new therapeutic options in four disease areas – MPS VII, HoFH, LC-FAOD and XLH. In the last year alone, we treated over a thousand patients across the regionⁱⁱⁱ .

This region plays an important role in delivering care as well as shaping ideas and partnerships that strengthen the rare disease community worldwide. At Ultragenyx, we think of people living with rare diseases as belonging to “One Rare Nation”.

Serving More Patients Across More Countries in EMEA

What began 10 years ago as a small regional footprint has grown into an important presence, across the region including recent expansions beyond MENA to central and eastern Europe. As our presence has grown, so has our ability to reach more patients and expand our partnership with the rare disease community.

Looking ahead to the next 10 years, our focus is clear: reach more patients in more places, and make sure innovation isn’t limited to the most established markets. Much work remains to improve diagnosis, expand access, strengthen healthcare systems and advance innovation for people living with rare diseases across EMEA.

We are proud to be founding sponsors of the Health Leadership Mission For Rare Diseases in the EU and contribute to the World Health Assembly’s Global Action Plan for Rare Diseases. We also continue to partner closely with patient organizations, including EURORDIS and FH Europe, and 46 regional national patient advocacy groups in metabolic, cardiovascular and neurological diseases.

Ten years is a meaningful milestone. The unmet need that inspired Ultragenyx’s global expansion remains significant. We are proud of what has been built over the past ten years, and more focused than ever on what comes next.