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Alnylam to Pursue Full Approval & Rolling NDA for Givosiran

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Shares of Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) , fell 2.4% after the company announced that it plans to pursue a full approval of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP), after consulting with the FDA.

The full approval will be based on the complete results of the ENVISION phase III study on givosiran, rather than filing based on the interim phase III results. This signifies a more extended approval timeline, resulting in a decline in the company’s shares.

So far this year, shares of Alnylam have declined 37% compared with the industry’s fall of 15.4%.

 

The FDA also agreed to a rolling submission of a new drug application (NDA), which will be initiated in 2018, with full clinical sections submitted in mid-2019, assuming positive study results.

In October 2018, Alnylam announced positive early results from the interim analysis of the ENVISION phase III study.  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 a disease biomarker, which is likely to predict clinical benefit.

The study also showed serious adverse events (SAEs) in 22% of givosiran patients and 10% of placebo patients in the interim analysis cohort of 43 patients, with one patient (4%) on givosiran discontinuing treatment due to an increase in liver transaminase that resolved.

We remind investors that enrollment in the ENVISION study was completed ahead of schedule with 94 AHP patients, exceeding the target of about 75 patients due to high patient demand.

The company expects to report top-line full study results of the primary endpoint — the annualized attack rate after six months of treatment — in early 2019. The company plans to file for marketing authorization in all other markets based on the complete results of the ENVISION study, assuming positive results.

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 (RHHBY - Free Report) , Takeda, Merck, The Medicines Company and Sanofi’s specialty care global business unit, Genzyme, among others.

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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