FDA approves first treatment for a condition associated with early-onset pediatric cataracts

The US Food and Drug Administration (FDA) approved Ctexli™ (chenodiol) for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. This is the first FDA-approved treatment for CTX, which is a rare lipid storage disease that can be associated with pediatric bilateral cataracts.

The approval was based on results from a 24-week, double-blind, placebo-controlled, randomized, crossover study that assessed chenodiol 250 mg administered three times per day. Compared with placebo, chenodiol significantly reduced plasma cholestanol and urine 23S-pentol.

The most common adverse events associated with chenoidiol are diarrhea, headache, abdominal pain, constipation, hypertension, muscular weakness, and upper respiratory tract infection.

See the press release for full information.