The U.S. Food and Drug Administration approved Ctexli (chenodiol) for the treatment of cerebrotendinous xanthomatosis (CTX) in adults, according to an announcement. Ctexli is the first FDA-approved drug to treat CTX, a very rare lipid storage disease.
The efficacy of Ctexli for the treatment of patients with CTX was evaluated in a double-blind, placebo controlled, randomized crossover withdrawal trial. The 24-week trial demonstrated that treatment with Ctexli, 250 milligrams three times per day, resulted in significant reduction in plasma cholestanol and urine 23S-pentol (cholesterol metabolites that are markedly increased in CTX patients) compared to placebo treatment.
The prescribing information for Ctexli includes a warning for liver toxicity in all patients with increased risk for liver damage in patients with pre-existing liver disease or bile duct abnormalities. Patients should obtain liver blood tests before starting treatment, annually while on treatment and as clinically indicated. If signs of liver toxicity (e.g., stomach pain, nausea, fatigue, dark urine, bruising, yellowing of the eyes and skin, itching) occur, patients are advised to see their doctor and discontinue Ctexli.
The most common side effects of Ctexli are diarrhea, headache, abdominal pain, constipation, hypertension, muscular weakness and upper respiratory tract infection.
The recommended dosage is 250 milligrams, taken orally three times a day.
The FDA granted Ctexli Priority Review, Fast Track and Orphan Drug designations for this application.
