US FDA Approves Ctexli – BiotechReality – Information Intelligence System for Biopharma

Ctexli (chenodiol) has been approved by the U.S. Food and Drug Administration to treat adult patients with cerebrotendinous xanthomatosis (CTX). The first medication approved by the FDA to treat CTX, a rare lipid storage condition, is Ctexli. The FDA is dedicated to supporting new drug development for rare diseases including very rare metabolic diseases like cerebrotendinous xanthomatosis, CTX is a progressive multisystemic disorder that significantly impacts patients and previously lacked approved treatments. Today’s approval provides a safe and effective treatment option for CTX. Janet Maynard, M.D., M.H.S. What is CTX? A genetic metabolic condition known as CTX is brought on by a mutation in the CYP27A1 gene, which results in a lack of the enzyme necessary for the body to break down lipids. Patients with CTX are unable to break down cholesterol normally because their livers produce less bile acid. As a result, atypical cholesterol metabolites—substances that are produced when cholesterol is broken down—deposit in different parts of the body, such as the liver, brain, skin, and tendons, causing damage to those organs and tissues. Ctexli reduces the aberrant deposits of cholesterol metabolites believed to be the cause of clinical problems in CTX by replenishing inadequate levels of one of the bile acids. Efficacy of Ctexli The effectiveness of Ctexli in treating CTX patients was assessed in a randomized, double-blind, placebo-controlled crossover withdrawal study. The 24-week study showed that, in comparison to placebo, treatment with 250 mg of Ctexli three times a day significantly decreased plasma cholestanol and urine 23S-pentol, two cholesterol metabolites that are noticeably elevated in CTX patients. Also Read: Intriguing New Class of Antimalarial Drugs Discovered Suspecting Side Effects A warning for liver toxicity is included in the prescribing material for Ctexli for all patients who have a higher risk of liver damage due to pre-existing liver illness or abnormalities of the bile duct. Before beginning treatment, once a year during treatment, and as clinically required, patients should have liver blood tests performed. Patients are recommended to consult their physician and stop using Ctexli if they have any symptoms of liver toxicity, such as nausea, lethargy, dark urine, bruises, itching, or yellowing of the eyes and skin. Diarrhea, headaches, abdominal pain, constipation, hypertension, muscle weakness, and upper respiratory tract infections are the most frequent adverse effects of Ctexli. Source: US FDA Press Release Last Modified:

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US FDA Approves Ctexli, First Treatment for Cerebrotendinous Xanthomatosis of Mirum Pharma

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