CTX is a rare autosomal inherited, progressive cholesterol metabolism disorder that affects multiple parts of the body. In CTX, the deficiency of chenodeoxycholic acid (CDCA) can lead to the accumulation of bile alcohols, which in turn can cause the accumulation of toxic cholesterol. Cholesterol is a major driver of patient symptom burden and disease progression, including irreversible neurological dysfunction. If left untreated, CTX patients may experience symptoms that disrupt their lives and worsen over time, including chronic diarrhea, bilateral cataracts in adolescents, xanthoma tendineae, and neurodegeneration.
This approval is mainly based on the positive results of the Phase 3 Resilience study, which evaluated the efficacy of Ctexli in adult patients with CTX. The study achieved the primary endpoint, which was a highly statistically significant decrease in patients' bile alcohol levels (p<0.0001). The ROSE study also showed that Ctexli treatment significantly improved the serum cholesterol levels of patients. The most common adverse events are diarrhea and headache, most of which are mild or moderate and are not considered treatment-related.
The active ingredient of Ctexli tablets is chenodeoxycholic acid, a naturally occurring bile acid initially approved for the treatment of patients with radiation-induced opaque gallstones in the gallbladder.
<Reprinted from WuXi AppTec>
