FDA approves first treatment for cerebrotendinous xanthomatosis
The US Food and Drug Administration (FDA) late Friday revealed it has approved Ctexli (chenodiol) for the treatment of cerebrotendinous xanthomatosis (CTX) in adults.
Developed by Mirum Pharmaceuticals (Nasdaq: MIRM), Ctexli is the first FDA-approved drug to treat CTX, a very rare lipid storage disease.
Mirum gained access to the drug through its acquisition in July 2023 of Travere Therapeutics’ (Nasdaq: TVTX) bile acid product portfolio. The drug has already been cleared for decades to treat radiolucent gallstones under the brand name Chenodal.
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