US FDA approves Mirum Pharma's genetic disorder drug (updated)

Mirum Pharmaceuticals

21/02/2025 17:07

(Adds background on the company and drug in paragraphs 2 to 4)
       Feb 21 (Reuters) - The U.S. Food and Drug Administration
said on Friday it has approved Mirum Pharmaceuticals'  MIRM.O 
drug to treat a type of rare disorder, which affects the body's
ability to metabolize fats known as cholesterol.
    Patients with the disorder known as cerebrotendinous
xanthomatosis are unable to break down cholesterol properly,
causing toxins to build up throughout the body over time.
    The drug is a form of chenodeoxycholic acid, a naturally
occurring bile acid that was originally approved for the
treatment of people with a type of stone, called radiolucent
stones in the gallbladder.
    Mirum gained access to the drug, sold under the brand name
Ctexli, through its acquisition of Travere Therapeutics'
 TVTX.O  bile acid product portfolio.

 (Reporting by Sriparna Roy and Christy Santhosh in Bengaluru;
Editing by Shilpi Majumdar)
 ((Sriparna.Roy@thomsonreuters.com))