Lumasiran sodium

Lumasiran sodium is a medication specifically designed to treat primary hyperoxaluria type 1 (PH1), a rare genetic disorder. It is marketed under the brand name Oxlumo by Alnylam Pharmaceuticals.

Mechanism of Action

Lumasiran sodium works by inhibiting the production of a protein called glycolate oxidase (GO). This protein is involved in the production of oxalate, a substance that can form crystals in the kidneys and other organs in people with PH1. By reducing the levels of GO, lumasiran sodium decreases the amount of oxalate produced, thereby reducing the risk of crystal formation and kidney damage.

Uses

Lumasiran sodium is used to treat PH1 in pediatric and adult patients. It is administered via subcutaneous injection.

Side Effects

Common side effects of lumasiran sodium include injection site reactions, abdominal pain, and nausea. Serious side effects can include allergic reactions and kidney problems.

Approval

Lumasiran sodium was approved by the U.S. Food and Drug Administration (FDA) in November 2020. It is the first treatment approved by the FDA specifically for PH1.

See Also

• Primary hyperoxaluria
• Alnylam Pharmaceuticals
• Glycolate oxidase
• Oxalate