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Mirum Submits New Drug Application to FDA for Chenodiol for the Treatment of CTX
Submission based on the positive Phase 3 RESTORE study Mirum holds orphan designation for chenodiol in CTX Potential to have first and only therapy indicated
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":"\n\nSubmission based on the positive Phase 3 RESTORE study\n\n\n\nMirum holds orphan designation for chenodiol in CTX\n\n\n\nPotential to have first and only therapy indicated for CTX in the US\n\n\n\n FOSTER CITY, Calif.--(BUSINESS WIRE)--\nMirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the submission of a new drug application (NDA) for chenodiol for the treatment of patients in the U.S. with cerebrotendinous xanthomatosis (CTX).\n\n\nCTX is a rare autosomal genetic progressive disorder of cholesterol metabolism that affects many parts of the body. In CTX, a deficiency of the bile acid CDCA leads to a buildup of bile alcohols which precedes a toxic accumulation of cholestanol. Cholestanol is the key driver of symptomatic burden and disease progression, including irreversible neurologic dysfunction. If not treated, patients with CTX can experience symptoms that disrupt their lives and can progress over time, including chronic diarrhea, juvenile bilateral cataracts, tendon xanthomas, and neurologic deterioration.\n\n\nThe submission of the NDA is based on the positive results of the Phase 3 RESTORE study which evaluated chenodiol in adult patients with CTX. The study met its primary endpoint of reduction in bile alcohols with high statistical significance (p","length":1500,"tagName":"div"}]