Mirum Pharmaceuticals' (MIRM) treatment for cerebrotendinous xanthomatosis, a lipid storage disease, has received approval from the US Food and Drug Administration, the regulator said Friday.
Chenodiol, with the brand name Ctexli, is the first FDA-approved drug to treat the genetic metabolic disorder in adults. It lowers abnormal deposits of cholesterol metabolites believed to be responsible for clinical abnormalities in the disease, the FDA said.
The effectiveness of Ctexli was tested in a 24-week clinical trial where patients either received the drug or a placebo. Patients who took 250 milligrams of Ctexli three times a day had a significant drop in certain cholesterol byproducts that are unusually high in people with cerebrotendinous xanthomatosis, compared to those who received a placebo, the regulator added.
Shares of Mirum Pharmaceuticals were down more than 1% in recent trading.
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