Trial Profile

A Phase 3 Study to Evaluate the Effects of Chenodeoxycholic Acid in Adult and Pediatric Patients With Cerebrotendinous Xanthomatosis

Status: Completed

Phase of Trial: Phase III

Latest Information Update: 03 Mar 2025

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At a glance

Drugs Chenodeoxycholic acid (Primary)
Indications Cerebrotendinous xanthomatosis
Focus Registrational; Therapeutic Use
Acronyms RESTORE
Sponsors Mirum Pharmaceuticals

Most Recent Events

03 Mar 2025 New trial record
24 Feb 2025 According to Mirum Pharmaceuticals media release, company announced that the U.S. Food and Drug Administration (FDA) has approved CTEXLI™ (chenodiol) tablets, a bile acid, for the treatment of adults with cerebrotendinous xanthomatosis (CTX).
13 Jan 2025 According to a Mirum Pharmaceuticals media release, FDA Prescription Drug User Fee Act (PDUFA) date for chenodiol in cerebrotendinous xanthomatosis (CTX) is March 28, 2025.

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