FDA approves first treatment for rare lipid storage disease

Key takeaways:

• Mirum Pharmaceuticals received first treatment approval for treatment of cerebrotendinous xanthomatosis.
• Ctexli resulted in significant reduction in plasma cholestanol and urine 23S-pentol.

The FDA has approved Ctexli for the treatment of cerebrotendinous xanthomatosis (CTX), a rare lipid storage disease, in adults.

According to a press release, Ctexli (chenodiol) is the first FDA-approved drug to treat CTX. The application was granted priority review, fast track and orphan drug designations.

"CTX is a progressive multisystemic disorder that significantly impacts patients and previously lacked approved treatments,” said Janet Maynard, MD, MHS, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine at the FDA’s Center for Drug Evaluation and Research. “Today’s approval provides a safe and effective treatment option for CTX.”

The double-blind, placebo-controlled randomized trial demonstrated 250 mL Ctexli three times per day orally resulted in significant reduction in plasma cholestanol and urine 23S-pentol, cholesterol metabolites that are highly increased in patients with CTX, compared with placebo.

At 24 weeks, the most common side effects of Ctexli were diarrhea, headache, abdominal pain, constipation, hypertension, muscular weakness and upper respiratory tract infection.

Approval for Ctexli was granted to Mirum Pharmaceuticals Inc.