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Plozasiran, also known by its developmental name ARO-APOC3, is an investigational RNA interference (RNAi) therapeutic drug being developed by Arrowhead Pharmaceuticals. It is designed to treat conditions characterized by dangerously high levels of triglycerides in the blood, such as familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG).
Plozasiran works by targeting and reducing the production of apolipoprotein C-III (APOC3), a protein that plays a key role in regulating triglyceride metabolism. APOC3 inhibits the breakdown of triglyceride-rich lipoproteins (TRLs). By silencing the gene that produces APOC3, plozasiran allows the body to more effectively clear TRLs from the bloodstream, leading to a significant reduction in triglyceride levels. It is administered by subcutaneous injection (under the skin), and in clinical trials, it has shown potential for infrequent dosing, such as once every three months.
Plozasiran has shown promising results in several clinical trials:
Based on these positive results, Arrowhead Pharmaceuticals has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the treatment of FCS. The drug has also received several regulatory designations from the FDA, including Breakthrough Therapy Designation, Orphan Drug Designation, and Fast Track Designation.
In clinical trials, plozasiran has generally been well-tolerated. The risk of adverse events has been similar to that of a placebo. The most common reported side effects include abdominal pain, nasopharyngitis (inflammation of the nose and pharynx), headache, and nausea. Hyperglycemia was more frequent in patients with pre-existing diabetes or prediabetes.