Photo: the Ultragenyx Japan team on the day of filing for the approval of our first rare disease drug in Japan 

Ultragenyx recently established its first headquarters in Asia with the opening of its Japan offices. Our team has been building our operations and strengthening our relationships with health care professionals, policymakers, industry partners, and the rare disease community throughout the country. At the same time, we’ve achieved meaningful progress toward our goals and laid the foundation for a thriving future here.  

We’ve just celebrated our first commercial drug launch in Japan for the treatment of patients with Mucopolysaccharidosis Type VII (MPS VII or Sly Syndrome), and we’re pleased to ensure broad access to this therapy for patients moving forward. We work diligently to ensure that anyone who may benefit from our therapies has access – this is core to the Ultragenyx vision of leading the future of rare disease medicine. This launch is the result of a long-term collaboration between the Ultragenyx global team and the team at Amicus Therapeutics.  

The term is derived from the word Nanbyo, which translates to ‘difficult + illness,’ reflecting the heavy burden on those affected and their families quotation mark

We’ve also brought on a talented team of professionals who are dedicated to improving the lives of individuals with rare diseases and who are focused on ensuring all Ultragenyx’s commercialized therapies are approved and made available to rare disease patients here in Japan. This process involves coordinating with Japan’s regulatory authorities, the Ministry of Health, Labor and Welfare, and the Pharmaceuticals and Medical Devices Agency, and responding to unique requests throughout the drug approval process.  

In Japan, rare diseases are referred to as intractable diseases.The term is derived from the word Nanbyo, which translates to difficult + illness,” reflecting the heavy burden on those affected and their families. Intractable diseases are treated differently in Japan than in the United States and other countries, with most medical costs covered for patients. In addition, Japan implemented a drug price standards list, which sets the price for each approved medication. To date, 788 diseases for children and 331 diseases for adults are designated as intractable diseases in Japan, and all the rare and ultrarare diseases on which Ultragenyx focuses are on this designated list of intractable conditions. 

Throughout my career, my work has always focused on rare diseases, and I remain passionate about addressing the needs of the people living with these conditions – the Nanbyo community. I’m honored to lead our new and growing team and I am excited about the future for Ultragenyx Japan as we grow to support more disease areas. We will continue building relationships with the rare disease community here in Japan, as our colleagues are doing throughout the world, to enable Ultragenyx to advance our shared goal of treating as many rare disease patients as possible. 

Tadashi Kiriya is General Manager at Ultragenyx Japan 

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