US FDA approves Mirum Pharma’s genetic disorder drug
The U.S. Food and Drug Administration (FDA) on Friday approved Mirum Pharmaceuticals’ Ctexli to treat a genetic disorder, making it the first drug for a rare condition that interferes with the body’s ability to break down cholesterol.
Patients with the disorder cerebral cholesterolosis, or CTX, are unable to break down cholesterol properly, a condition that causes toxins to build up in the body over time.
Ctexli, a form of quinodeoxycholic acid — a bile acid naturally found in the liver — was previously approved to treat people with a type of stone called radiolucent gallstones.
The drug is expected to add $150 million to $200 million to the pharmaceutical company’s revenue by 2030.
