Feb. 24, 2025 – The FDA has approved a new pill for adults with cerebrotendinous xanthomatosis (CTX), a rare genetic disease that makes it hard for the body to handle cholesterol properly.

CTX is caused by mutations in the CYP27A1 gene, which under healthy circumstances encode for an enzyme that breaks down cholesterol into bile acids. Ctexli, an orally available drug, is Mirum’s answer ...

Caused by a mutation in the cytochrome P450 family 27 subfamily A member 1 (CYP27A1) gene, CTX leads to a deficiency of an enzyme that is crucial for fat breakdown. This results in abnormal ...

CTX is a genetic metabolic disorder caused by a mutation in a gene called CYP27A1, resulting in a deficiency of the enzyme that is important to the body's ability to break down fats. Ctexli ...

In CTX, the major bile acid synthesis pathways are disrupted due to partial or total deficiency in sterol 27-hydroxylase encoded by the CYP27A1 gene, resulting in the body’s inability to break ...

CTX is a genetic metabolic disorder caused by a mutation in a gene called CYP27A1 resulting in a deficiency of the enzyme that is important in the body's ability to break down fats. Due to reduced ...

Today’s approval provides a safe and effective treatment option for CTX.” CTX is a genetic metabolic disorder caused by a mutation in a gene called CYP27A1 resulting in a deficiency of the enzyme that ...