Alnylam Pharma Says Canada Authorizes OXLUMO For Treatment Of Primary Hyperoxaluria Type 1

Alnylam Pharmaceuticals, Inc. (ALNY) announced Wednesday that Health Canada has issued a Notice of Compliance (NOC) authorizing OXLUMO (lumasiran) injection for subcutaneous use for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in paediatric and adult patients.

OXLUMO is a double-stranded siRNA that reduces the levels of the enzyme glycolate oxidase (GO) responsible for supporting the production of oxalate by targeting the hydroxy acid oxidase 1 (HAO1) messenger ribonucleic acid (mRNA) in major cells of the liver known as hepatocytes through RNA interference.

The authorization is based on two pivotal phase III trials, ILLUMINATE-A and ILLUMINATE-B, that showed significant reduction in urinary oxalate which drives the progression of PH1 disease.

PH1 is an ultra-rare and debilitating genetic disease of the liver characterized by oxalate overproduction. Oxalate is an end-product of metabolism and high levels of it are toxic because it cannot be broken down by the human body.

Until today, there were no authorized pharmaceutical therapies for PH1. The only curative treatment is a liver transplant and if the patient has already progressed to kidney failure, then a dual liver/kidney transplant is required.

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