The United States Food and Drug Administration has granted approval for a novel treatment targeting cerebrotendinous xanthomatosis (CTX), a rare genetic condition that leads to harmful fat accumulation throughout the body. The medication, developed by Mirum
Pharmaceuticals and marketed under the name Ctexli, received regulatory clearance on Friday for adult patients.
CTX stems from a genetic mutation that interferes with the body’s ability to produce an essential enzyme involved in cholesterol breakdown. This disruption causes abnormal cholesterol metabolites to build up in various organs, resulting in progressive damage and neurological decline over time.
The therapeutic came under Mirum’s ownership through a strategic acquisition made in 2023, when the company purchased Travere Therapeutics’ bile acid portfolio in a $210 million transaction. Ctexli, which contains synthetic chenodeoxycholic acid as its active ingredient, had previously been authorized for treating radiolucent gallstones.
Clinical validation for the drug’s approval came from a Phase 3 trial examining its effects in adult CTX patients. The study protocol involved administering 250mg doses three times per day over a 24-week period. Results demonstrated that, compared to placebo, the treatment significantly reduced levels of plasma cholestanol and bile alcohols – the toxic metabolites that accumulate in CTX patients.
The FDA-approved labeling includes important safety information regarding potential liver toxicity. Patients will need to undergo liver function testing both before initiating treatment and at regular intervals during the therapeutic course. Additional reported side effects include headaches, digestive issues like constipation and diarrhea, elevated blood pressure, muscle weakness, and upper respiratory infections.
Chris Peetz, CEO of Mirum Pharmaceuticals, emphasized the significance of this approval, stating it creates new opportunities for identifying and treating adult CTX patients in the US. He expressed hope that the availability of Ctexli would lead to earlier diagnosis and help prevent some of the devastating long-term effects associated with the condition.
The company has integrated the medication into its existing patient support infrastructure to facilitate access. Regarding cost
considerations, Mirum indicated that Ctexli’s pricing would align with the previous version of the drug used in gallstone treatment. The company projects that most patients will face out-of-pocket costs of $10 or less.
This regulatory approval grants Mirum exclusive rights to market the drug for CTX treatment. The company’s strategic acquisition of the treatment through the Travere deal has positioned them to address an important unmet need in this rare disease space. The approval marks a significant milestone in providing therapeutic options for CTX patients, who previously had limited treatment alternatives.
The development represents a key advancement in addressing rare genetic disorders through targeted therapeutic approaches. By specifically addressing the underlying metabolic disruption in CTX, Ctexli offers hope for better disease management in affected adults. The combination of demonstrated clinical efficacy and the company’s commitment to patient access suggests potential for meaningful impact in the CTX community.
Through their established patient support platform, Mirum aims to ensure that eligible patients can readily access the newly approved treatment. The company’s approach to pricing and patient assistance programs reflects an effort to balance commercial viability with healthcare accessibility for those affected by this rare genetic condition.