Dive Brief:
- The Food and Drug Administration on Friday approved a drug from Mirum Pharmaceuticals to treat a rare genetic disorder that causes toxic depositions of fat in different organs of the body.
- Called Ctexli, the drug is cleared for adults cerebrotendinous xanthomatosis, or CTX. A genetic mutation in people with this disease disrupts production of an enzyme needed to break down cholesterol. As a result, abnormal cholesterol metabolites accumulate in the body, leading to progressive damage and neurological deterioration.
- Mirum gained Ctexli through a 2023 deal with Travere Therapeutics that handed Mirum Travere’s bile acid portfolio for $210 million. A synthetic form of chenodeoxycholic acid, the drug was previously approved for the treatment of radiolucent gallstones in the gallbladder.
Dive Insight:
Ctexli’s approval was supported by data from a Phase 3 study testing the drug in adults with CTX. Over 24 weeks, trial participants were given 250 milligrams of Ctexli three times daily. Treatment led to a significant reduction compared to placebo in plasma cholestanol and bile alcohols — the toxic cholesterol metabolites.
The label granted by the FDA includes a warning for liver toxicity. People taking Ctexli will need to have liver tests performed both before and during treatment. Other side effects of treatment include headache, constipation, diarrhea, hypertension, muscle weakness and upper respiratory tract infection.
The approval gives Mirum a form of regulatory exclusivity on the drug’s use treating CTX.
“The FDA’s approval of Ctexli is tremendous as it unlocks an opportunity to better identify and treat adult patients with CTX in the United States,’ Mirum CEO Chris Peetz said in a statement. “Our hope is that patients are diagnosed sooner and have a chance to avoid some of the debilitating and lasting symptoms associated with CTX.”
Ctexli will be available through Mirum’s patient support platform. In a statement, Mirum said Ctexli would be priced "in line" with the cost of the prior version of the drug used to treat gallstones. The "majority of patients" will pay $10 or less for access to treatment, the company said.
