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Alnylam Starts Rolling NDA Submission for RNAi Candidate

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Alnylam Pharmaceuticals, Inc. ALNY announced that it has initiated submission of a rolling New Drug Application (NDA) to the FDA for lead candidate patisiran. The candidate is an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR (hATTR) amyloidosis.  The rolling submission allows completed portions of an NDA to be reviewed by the FDA on an ongoing basis.

Alnylam ‍has also requested priority review of application for patisiran, if granted, it might result in a six-month review process.

Notably, the NDA filing is based on promising results from the APOLLO phase III study that met its primary as well as all secondary endpoints. The patients who were administered patisiran experienced significant improvement in quality of life compared to placebo. In fact, APOLLO was the largest randomized study ever completed in this disease. The study also demonstrated the first-ever positive results for an RNAi therapeutic.

So far this year, shares of Alnylam have skyrocketed 237.5% compared with the industry’s gain of 1.6%.

 

 

Recently, Alnylam also announced that the Committee for Medicinal Products for Human Use (“CHMP”) of the European Medicines Agency (“EMA”) has granted an accelerated assessment for patisiran. With the accelerated assessment, the company can expect the review timeline to be reduced from the typical 210 days to 150 once the marketing authorization application (MAA) is filed and validated in the EU.

Alnylam and its partner Sanofi SNY plan to submit Marketing Authorisation Application to the European Medicines Agency around the year-end.

Meanwhile, Sanofi is preparing for regulatory filings for patisiran in Japan, Brazil and other countries , beginning in the first half of 2018. Once it gets the required regulatory approvals, Alnylam will commercialize patisiran in the United States, Canada and Western Europe with Sanofi commercializing the product in the rest of the world.

Going forward, the potential approval of patisiran is likely to be a huge boost for the people suffering from this often fatal disease.

Zacks Rank & Stocks to Consider

Alnylam carries a Zacks Rank #3 (Hold). Some better-ranked health care stocks in the same space include Ligand Pharmaceuticals Incorporated LGND and Corcept Therapeutics Incorporated CORT holding a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Ligand’s earnings per share estimates have moved up $3.68 to $3.70 for 2018 over the last 60 days. The company pulled off positive earnings surprises in two of the trailing four quarters, with an average beat of 8.22%. The share price of the company has increased 39.4% year to date.

Corcept’searnings per share estimates have moved up from 45 cents to 47 cents for 2017 and from 77 cents to 88 cents for 2018 over the last 60 days. The company delivered positive earnings surprises in two of the trailing four quarters, with an average beat of 14.32%. The share price of the company has increased 162.4% year to date.

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