Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) has been in the news in the past week.
Last week, the company announced that it has submitted a new drug application (NDA) to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), seeking approval of Onpattro (patisiran) for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis. Onpattro 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. The company expects a decision in mid-2019. The NDA is based on the results from the APOLLO study that met its primary as well as all secondary endpoints.
The same week, Alnylamalso announced that Health Canada granted Priority Review status to Onpattro for the same indication.
We remind investors that Onpattro was approved by the FDA in the United States in August. The drug is the first and only FDA approved therapy for the treatment of the polyneuropathy of hATTR amyloidosis in adults. Onpattro, which is the first approved drug in Alnylam’s portfolio, is expected to drive revenues for the company.
Alnylam had a partnership with Sanofi (SNY - Free Report) for Onpattro. The agreement was amended in January 2018. Per the agreement, Alnylam obtained all development and commercialization rights for Onpattro.
Alnylam also announced positive early results from the interim analysis of the ENVISION phase III study of its investigational RNA interference (RNAi) therapeutic — givosiran. The candidate is targeting aminolevulinic acid synthase 1 or ALAS1 for the treatment of acute hepatic porphyrias (AHP). The results showed that treatment with givosiran led to a statistically significant reduction in urinary aminolevulinic acid (ALA) levels in acute intermittent porphyria (AIP) patients compared with placebo. ALA is adisease biomarker which is reasonably likely to predict clinical benefit.
Alnylam plans to discuss these data and the regulatory path ahead with the FDA. Depending on the outcome of these discussions, the company intends to file an NDA at or around 2018 year-end. The company expects to report top-line results from the study in early 2019.
We remind investors, that the company also has other candidates in its pipeline. Alnylam along with partner The Medicine Company (MDCO - Free Report) is evaluating inclisiran in phase III ORION studies for hypercholesterolemia.
Alnylam’s expertise in RNAi therapeutics and broad intellectual property estate have allowed the company to enter into collaborations with leading pharmaceutical and life sciences companies, including Ionis Pharmaceuticals, Novartis (NVS - Free Report) , Roche, Takeda, Merck, The Medicines Company and Sanofi’s specialty care global business unit, Genzyme, among others.
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