Mirum’s CTEXLI™ (chenodiol) Tablets Receives FDA Approval for Treatment of Cerebrotendinous Xanthomatosis (CTX)
Mirum Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has approved Ctexli (chenodiol) tablets as the first treatment for cerebrotendinous xanthomatosis (CTX), a rare and progressive genetic disorder that affects cholesterol metabolism.
CTX leads to harmful cholesterol buildup in the body, resulting in neurological and systemic complications. Ctexli, an oral therapy, works by restoring bile acid balance and reducing disease-related damage. The FDA approval was based on clinical data demonstrating significant reductions in cholestanol levels, a key marker of CTX.
Mirum Pharmaceuticals is committed to ensuring access to Ctexli and supporting the CTX community. This approval marks a major step forward in addressing an unmet medical need for patients affected by this rare disease.
For more details, read the full announcement here.
*The United Leukodystrophy Foundation is not liable for any inaccuracies or gaps in these overviews , which are offered “as is” without assurances on completeness, accuracy, or timeliness. For verification or detailed information, please consult the original articles directly.
As a leukodystrophy related nonprofit, the ULF provides awareness to scientific literature. Inclusion on our website does not imply endorsement of, or agreement with, the contents by the ULF.