FDA Approves Mirum Pharmaceuticals' Drug As First For Genetic Disorder With Inability To Break-Down Cholesterol

The U.S. Food and Drug Administration (FDA) approved Mirum Pharmaceuticals, Inc’s MIRM drug to treat CTX, a very rare lipid storage disease.

CTX is a genetic metabolic disorder caused by a mutation in a gene called CYP27A1, resulting in a deficiency of the enzyme that is important to the body's ability to break down fats.

Ctexli (chenodiol) is the first FDA-approved drug to treat cerebrotendinous xanthomatosis (CTX) in adults.

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Due to reduced bile acid production in the liver, patients with CTX are unable to break down cholesterol in a normal way, resulting in deposition of atypical cholesterol metabolites in various places in the body including the brain, liver, skin and tendons, leading to damage to those organs and tissues.

Ctexli works to replace deficient levels of one of the bile acids, reducing the abnormal deposits of cholesterol metabolites thought to be responsible for clinical abnormalities in CTX.

The efficacy of Ctexli 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, significantly reduced plasma cholestanol and urine 23S-pentol (cholesterol metabolites 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.

In January, Mirum Pharmaceuticals reported preliminary net product sales of approximately $99 million in the fourth quarter, including approximately $64 million in Livmarli net sales and approximately $35 million from Cholbam and Chenodal.

The company expects 2025 global net product sales of approximately $420 million to $435 million and positive cash flow from operations.

Price Action: Mirium stock is up 0.10% at $53.22 at last check Friday.

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