(Reuters) -The U.S. Food and Drug Administration approved Arrowhead Pharmaceuticals’ drug for a type of genetic disorder that causes extremely high levels of fat in the bloodstream, the health regulator’s website showed on Tuesday.
The approval marks Arrowhead’s first product on the market and the second U.S.-authorized treatment for a hereditary condition known as familial chylomicronemia syndrome.
In a late-stage study with 75 patients, the drug branded as Redemplo reduced triglycerides, a key hallmark of the disease, by 80% and cut the risk of pancreatitis by 83% versus placebo.
FCS is a rare inherited disorder caused by defects in lipoprotein lipase, an enzyme responsible for breaking down fat particles, leading to very high triglyceride levels in the blood.
The disorder causes symptoms such as abdominal pain and, in severe cases, acute pancreatitis. It is estimated to affect 3,000 to 5,000 patients globally, according to National Institutes of Health data.
Shares of the company were halted in morning trading.
(Reporting by Padmanabhan Ananthan and Christy Santhosh in Bengaluru; Editing by Tasim Zahid)
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