Alnylam’s Onpattro Approved in Japan for Treatment of hATTR

June 18, 2019

Alnylam Pharmaceuticals announced that the Japanese Ministry of Health, Labour and Welfare has approved Onpattro (patisiran) for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy, the first approved RNAi therapeutic in Japan.

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. “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.”

Alnylam submitted a New Drug Application to Japan’s Pharmaceuticals and Medical Devices Agency (MHLW) on September 27, 2018 based on the results from the APOLLO phase 3 study, which evaluated the efficacy and safety of patisiran in hATTR amyloidosis patients with polyneuropathy. Results from the study were published in the July 5, 2018, issue of the New England Journal of Medicine. Patisiran had previously received orphan drug designation from the MHLW, which made it eligible for priority review as well as 10 years of market exclusivity in Japan.

“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,” said Masako Nakamura, senior vice president, head of Asia, at Alnylam.

RNAi, or RNA interference, is a natural cellular process of gene silencing. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made.

Onpattro (patisiran), is an intravenously administered RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR amyloidosis. It is designed to target and silence TTR messenger RNA, thereby blocking the production of TTR protein before it is made. Patisiran blocks the production of TTR in the liver, reducing its accumulation in the body’s tissues in order to halt or slow down the progression of the disease.

In August 2018, Patisiran received U.S. Food and Drug Administration (FDA) approval for the treatment of the polyneuropathy of hATTR amyloidosis in adults, as well as European Medicines Agency marketing authorization for the treatment of hATTR amyloidosis in adults with stage 1 or Stage 2 polyneuropathy.

Author: Rare Daily Staff

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