– ONPATTRO is the First RNAi Therapeutic Approved in Japan –
− Approval Marks the Arrival of an Entirely New Treatment Approach for People Living with hATTR Amyloidosis with Polyneuropathy in Japan –
Alnylam Pharmaceuticals, Inc. (ALNY), the leading RNAi therapeutics company, today announced that the Japanese Ministry of Health, Labour and Welfare (MHLW) has approved ONPATTRO® (patisiran) for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy. ONPATTRO is the first approved RNAi therapeutic in Japan and Alnylam will launch and directly market it in the country.
“hATTR amyloidosis is a genetic disease that can cause a variety of debilitating symptoms and can often be fatal. Historically, treatment options for people living with this condition in Japan, where the V30M mutation of the condition is prevalent, were limited,” said Professor Yukio Ando, President of ISA (International Society of Amyloidosis) and JSA (Japan Society of Amyloidosis), Chairman and Professor/Department of Amyloidosis Research of Nagasaki International University and Professor Emeritus and Visiting Professor of Kumamoto University, Japan. “The approval of ONPATTRO, a treatment that has the potential to mitigate and potentially reverse symptoms of polyneuropathy and to improve quality of life, marks a paradigm shift in the way we approach and treat this serious disease.”
“We are very excited about the approval of ONPATTRO and are proud to be bringing this important new treatment to patients with hATTR amyloidosis with polyneuropathy in Japan,” said Masako Nakamura, Senior Vice President, Head of Asia, Alnylam. “With this approval, we look forward to working with the hATTR amyloidosis community to continue to raise disease awareness, increase diagnosis rates and ultimately provide treatment to patients suffering from this devastating disease. This is a significant milestone in our efforts to bring RNAi therapeutics to people around the world and we look forward to continuing to build our presence in Asia.”
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