• Patisiran is approved in the US for hATTR
• It has orphan drug status in the US and Japan
• Patisiran is elegible for priority review in Japan

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) announced on 9/28/18 that it has submitted a New Drug Application (NDA) to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for approval of patisiran for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis. Patisiran has orphan drug designation from the Ministry of Health, Labor and Welfare (MHLW), which makes it eligible for priority review as well as 10 years of market exclusivity, if approved. Based on the priority review timeline, Alnylam expects a decision from the MHLW and PMDA in mid-2019.

Patisiran – which will be commercialized under the brand name Onpattro is Alnylam’s first drug submitted for regulatory review in Japan, and if approved, will be the first product to be launched and marketed by Alnylam in the country. The Company established operations in Japan this summer.
“The NDA submission highlights Alnylam’s continued commitment to bring RNAi therapeutics to patients in need with the potential to make a meaningful impact on serious rare diseases that have devastating effects on the lives of patients and their families,” said Masako Nakamura, Senior Vice President, Head of Asia, Alnylam. “Hereditary ATTR amyloidosis is endemic in Japan, with V30M being the most common mutation. We look forward to working with the PMDA during the review process so that we can make patisiran available to patients as expeditiously as possible.”

Patisiran is an intravenously administered RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR amyloidosis. It is designed to target and silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. Patisiran blocks the production of transthyretin 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 (FDA) approval for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, having been reviewed by the FDA under Priority Review and previously granted Breakthrough Therapy and Orphan Drug Designations. Also, in August 2018, patisiran received European Commission marketing authorization for the treatment of hATTR amyloidosis in adults with stage 1 or stage 2 polyneuropathy. The European Medicines Agency (EMA) reviewed patisiran under the accelerated assessment procedure that is granted to medicines judged to be of major interest for public health and therapeutic innovation. In Japan, the non-proprietary adopted name (JAN) for patisiran is patisiran sodium.

Hereditary transthyretin (TTR)-mediated amyloidosis (hATTR) is an inherited, progressively debilitating, and often fatal disease caused by mutations in the TTR gene. TTR protein is primarily produced in the liver and is normally a carrier of vitamin A. Mutations in the TTR gene cause abnormal amyloid proteins to accumulate and damage body organs and tissue, such as the peripheral nerves and heart, resulting in intractable peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations. hATTR amyloidosis represents a major unmet medical need with significant morbidity and mortality, affecting approximately 50,000 people worldwide. The median survival is 4.7 years following diagnosis, with a reduced survival (3.4 years) for patients presenting with cardiomyopathy.