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Alnylam Completes Rolling NDA Submission to FDA for Givosiran

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Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) announced that it has completed the rolling submission of a new drug application (NDA) to the FDA for givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP).

Shares of Alnylam have declined 3.3% year to date against the industry’s growth of 1.1%.


The filing is supported by positive top-line data from the phase III ENVISION study. The study showed that givosiran substantially reduced the annualized rate of composite porphyria attacks, compared to placebo, with an overall safety and tolerability profile. If approved, givosiran will be the first potential treatment demonstrating substantial reduction in the frequency of porphyria attacks.

Givosiran previously received Breakthrough Therapy designation and Orphan Drug status from the FDA for AHP. The drug has also been granted Priority Medicines (PRIME) designation and Orphan Drug status by the European Medicines Agency (EMA) for the same indication. Alnylam expects to file a Marketing Authorization Application (MAA) in mid-2019.

We remind investors that the company’s only approved drug, Onpattro, is a first-of-its-kind RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The drug is the first and only FDA-approved treatment for this indication. 

Alnylam also has other candidates in its pipeline. The company along with partner The Medicine Company (MDCO - Free Report) is evaluating inclisiran in phase III ORION studies for hypercholesterolemia.

The company’s expertise in RNAi therapeutics and broad intellectual property estate have allowed it to enter collaborations with leading pharmaceutical and life sciences companies, including Ionis Pharmaceuticals, Novartis (NVS - Free Report) , Roche, Takeda, Merck and Sanofi’s specialty care global business unit, Genzyme, among others.

Alnylam and Regeneron Pharmaceuticals (REGN - Free Report) extended their collaboration agreement. Both the companies will work together to discover, develop and commercialize new RNAi therapeutics for a broad range of diseases by addressing disease targets expressed in the eye and central nervous system (CNS), in addition to a select number of targets expressed in the liver.


Zacks Rank

Alnylam currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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