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Alnylam Submits MAA in Europe for RNAi Therapeutic Givosiran

Zacks Equity Research

Alnylam Pharmaceuticals, Inc. ALNY announced that it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1), in development for the treatment of acute hepatic porphyria (AHP).

The MAA was supported by data from the pivotal ENVISION phase III study. In the study, givosiran met the primary efficacy endpoint, with a 74% mean reduction relative to placebo in the annualized rate of composite porphyria attacks. There was a corresponding 90% median reduction in composite annualized attack rate (AAR), with a median AAR of 1.0 in givosiran patients compared with that of 10.7 in placebo patients. In the trial, 50% of givosiran-treated patients were attack free during the six-month treatment period compared to 16.3% of placebo-treated patients.

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 EMA for the same.

In June 2019, the company also completed the rolling submission of a new drug application (NDA) to the FDA for givosiran in AHP.

Shares of Alnylam have declined 1.3% year to date against the industry’s growth of 7.4%.


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

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

Alnylam’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, Roche RHHBY, Takeda, Merck and Sanofi’s specialty care global business unit, Genzyme, among others.

Alnylam Pharmaceuticals, Inc. Price

Alnylam Pharmaceuticals, Inc. Price

Alnylam Pharmaceuticals, Inc. price | Alnylam Pharmaceuticals, Inc. Quote

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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