Alnylam Pharmaceuticals, Inc. ALNY announced that the FDA has granted priority review to its new drug application (“NDA”) seeking approval for its RNAi candidate, lumasiran, as a treatment for 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 had completed the rolling submission of the NDA in April. A decision from the FDA is expected by Dec 3, 2020.
The candidate also enjoys Orphan Drug, Breakthrough Therapy and Pediatric Rare Disease designations for treating PH1.
The company’s marketing authorization application ("MAA") to the European Medicines Agency, seeking approval for lumasiran in patients with PH1, is under review. A decision is expected later in the year.
Regulatory applications for lumasiran included positive data from the pivotal ILLUMINATE-A study. Data from the study showed that PH1 patients treated with the candidate met the primary efficacy endpoint of percent change from baseline, relative to placebo. The study also achieved statistically significant results for all six tested secondary endpoints including the proportion of patients achieving a near-normalization or normalization of urinary oxalate.
The company is also evaluating lumasiran in two phase III studies — ILLUMINATE-B and ILLUMINATE-C — in PH1 patients less than six years of age with preserved renal function and advanced PH1 in patients of all ages with advanced renal disease, respectively.
Shares of Alnylam have gained 18.5% year to date compared with the industry’s increase of 7.9%.
Meanwhile, the company is also evaluating several other candidates. A few significant ones include vutrisiran, inclisiran and fitusiran. The HELIOS-A phase III study is evaluating vutrisiran for the treatment of hATTR amyloidosis with polyneuropathy. Another phase III study, HELIOS-B, is evaluating the candidate hereditary and wild-type ATTR amyloidosis with cardiomyopathy. The company is developing fitusiran in partnership with Sanofi SNY in the ATLAS phase III program for the treatment of hemophilia A or B with and without inhibitors. The company and partner Novartis’ NVS inclisiran is under review in the United States and Europe for treating heterozygous familial hypercholesterolemia and atherosclerotic cardiovascular disease.
Alnylam has a collaboration with Regeneron Pharmaceuticals REGN to discover, develop and commercialize new RNAi therapeutics for a broad range of diseases by addressing disease targets expressed in the liver, eye and central nervous system.
Alnylam Pharmaceuticals, Inc. Price
Alnylam Pharmaceuticals, Inc. price | Alnylam Pharmaceuticals, Inc. Quote
Alnylam currently carries a Zacks Rank #3 (Hold).
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