First Treatment for Cerebrotendinous Xanthomatosis Approved

By SPC Staff

The FDA approved chenodiol (Ctexli, Mirum Pharmaceuticals) for the lipid storage disease cerebrotendinous xanthomatosis (CTX) in adults.

Chenodiol is the first treatment for CTX, an extremely rare disease caused by a mutation in the CYP27A1 gene that leads to reduced bile acid production in the liver and impaired ability to break down cholesterol. The resulting atypical cholesterol metabolites are deposited in, and damaging to, tissues and organs. Chenodiol replaces deficient levels