The U.S. Food and Drug Administration (FDA) has approved Redemplo (plozasiran) for the treatment of adult patients with familial chylomicronemia syndrome (FCS).
FCS is an inherited condition that disrupts the normal breakdown of fats in the body, causing large amounts of fat to build up in the blood. This condition is characterized by inflammation of the pancreas, abdominal pain, enlargement of the liver and spleen, and small yellow skin lesions called eruptive xanthomas. Familial chylomicronemia syndrome is caused by genetic changes in the LPL gene.
Plozasiran is a small interfering RNA therapy designed to suppress the production of apolipoprotein C-III protein, decreasing triglyceride levels. It is the first and only treatment approved as supplemental therapy to a diet used to reduce triglyceride levels.
The approval is supported by data from the PALISADE clinical trial (NCT05089084), a phase 3, randomized, double-blind, placebo-controlled study evaluating plozasiran for treatment in adults with FCS. The study illustrated that 25 mg of plozasiran achieved deep and durable reductions in triglycerides, with a median change from baseline of -80% versus -17% in the placebo group and lower incidence of acute pancreatitis compared with placebo.
The most common adverse events with plozasiran were hyperglycemia, headache, nausea, and injection site reaction.
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To learn more about FCS and other rare metabolic disorders, visit https://checkrare.com/diseases/metabolic-disorders/
