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Alnylam's Vutrisiran Gets Fast Track Tag for hATTR Amyloidosis

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Alnylam Pharmaceuticals, Inc. ALNY announced that the FDA has granted a Fast Track designation to its investigational RNAi therapeutic vutrisiran for the treatment of polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The Fast Track status will now help Alnylam submit a rolling new drug application (NDA) for vutrisiran.

The fast track tag from the FDA is designed to provide certain benefits and expedite the review time for new drugs/vaccines that treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs.

Shares of Alnylam have increased 4.8% so far this year against the industry’s decline of 6.1%.


We remind investors that in February 2020, Alnylam completed enrollment in the HELIOS-A phase III study on vutrisiran for treating hATTR amyloidosis with polyneuropathy. Top-line data from the same is expected in early 2021. The company also initiated the HELIOS-B phase III study in patients with hereditary and wild-type ATTR (wtATTR) amyloidosis with cardiomyopathy in November 2019.

Per the company, the HELIOS-B study complements the ongoing HELIOS-A phase III study to address patients with hATTR amyloidosis with polyneuropathy. This creates a comprehensive clinical development program evaluating the safety and efficacy of vutrisiran across the entire disease spectrum of ATTR amyloidosis.

The candidate was already granted an Orphan Drug designation for the treatment of ATTR amyloidosis both in the United States and the European Union.

Meanwhile, earlier this month, Alnylam completed the rolling submission of the NDA seeking approval for another RNAi candidate, lumasiran, to treat primary hyperoxaluria type 1 (PH1). The ultra-rare disease causes a progressive decline in kidney function, and can lead to end-stage renal disease.

The company is developing another candidate, fitusiran, in partnership with Sanofi SNY and continues enrollment in the ATLAS phase III program for the treatment of hemophilia A or B with and without inhibitors.

Zacks Rank & Stocks to Consider

Alnylam currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the biotech sector include Blueprint Medicines Corporation (BPMC - Free Report) and Celldex Therapeutics, Inc. CLDX, both sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Blueprint Medicines’ loss per share estimates have been narrowed 1.1% for 2020 and 1.2% for 2021 over the past 60 days.

Celldex’s loss per share estimates have been narrowed 12.5% for 2020 and 12.8% for 2021 over the past 60 days.

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