6yMirum’s Ctexli surpasses FDA milestone for rare metabolic condition

After Mirum Pharmaceuticals spent $210 million to acquire Travere Therapeutics’ bile acid portfolio they successfully received FDA approval about twenty-four months later. The FDA approved Ctexli marketed as chenodiol from Mirum Pharmaceuticals for adult CTX (cerebrotendinous xanthomatosis) patients.
Chenodiol performs as the first approved therapy for CTX treatment in the U.S. according to the FDA press release. CTX emerges as a genetic defect that results in body enzyme deficiencies impacting its ability to break down fats.
Bile acid shortage in CTX patient livers leads to abnormal cholesterol breakdown which causes the condition. Atypical cholesterol metabolite buildup results from this disorder and culminates in damage to brain tissue as well as skin tissue liver tissue and tendons.
Ctexli functions by providing missing bile acid levels which removes toxic cholesterol metabolite accumulations according to the FDA approval announcement. Clinical studies demonstrated that Ctexli at 250 mg tolerated thrice daily showed superior results compared to placebo concerning lowering metabolite cholesterol levels in patients having CTX.
The Food and Drug Administration requires that all patients receive a liver toxicity warning because they have elevated liver damage risk or existing liver conditions or bile duct problems. To manage potential risks during Ctexli treatment patients must have liver blood tests both at the start of therapy and annually as the regulator advises.
The official approval benefits Mirum decisively yet CTX patients already gained access to chenodiol off-label since the start of the last decade. The medication earned an orphan drug designation for CTX from the FDA in 2010 before receiving “medical necessity” status.
The acquisition agreement from 2023 involved Mirum paying Travere $210 million alongside up to $235 million in milestone bonuses to obtain both Chenodal (chenodiol) and Cholbam which received FDA approval in 2015 for rare bile acid synthesis disorder patients.
Company CEO Chris Peetz told Fierce Pharma that Mirum will act actively to promote CTX drug sales following its full approval along with improving disease screening. The CEO believed at that time that the company would succeed in identifying more patients with an untreated condition. Early treatment of the disease leads to diminished buildup of unchangeable defects in patients.
The pricing details and commercial plans for Ctexi from Mirum remain undisclosed while JMP Securities analyst Jonathan Wolleben estimates that new revenue will rise by $150 million to $200 million based on his evaluation reported by Reuters. According to the analyst the extended seven-year protection for gentrification makes this green light approval more valuable, the analyst said.