Safety and efficacy of odexibat (Belvi) in the treatment of pediatric patients with PFIC

Odevixibat (trade name: Bylvay) is an oral intestinal bile acid transporter (IBAT) inhibitor, mainly used to treat pruritus symptoms in patients with progressive familial intrahepatic cholestasis (PFIC). PFICIt is a group of rare inherited liver diseases that usually develop in infancy and early childhood and present with cholestasis, jaundice, severe itching and liver failure. By inhibiting IBAT, odexibat reduces the reabsorption of bile acids and increases their excretion in feces, thereby reducing serum bile acid levels and relieving itching symptoms.

In clinical studies, odexibat has been shown to have significant efficacy in PFIC pediatric patients. In the PEDFIC 1 study, odexibat significantly reduced serum bile acid levels and improved pruritus symptoms. PEDFIC 2Studies further confirmed its efficacy in patients with different PFIC subtypes, including PFIC1, PFIC2 and other rare subtypes. In a 72-week open-label extension study, patients treated with odexibat experienced significant improvements in pruritus scores, serum bile acid levels, and growth and development.

The safety of odexibat has also been fully evaluated. In clinical trials, the most common adverse reaction was mild to moderate diarrhea, with an incidence of approximately 12%. Other adverse reactions include abdominal pain, abnormal liver function indicators and vitamin A, D, E, K deficiencies. Most adverse reactions are reversible and can be alleviated after dose adjustment or symptomatic treatment. Therefore, the safety profile of odexibat in PFIC pediatric patients is good.

In real-world applications, odexibat also demonstrated good efficacy and safety. A study of pediatric patients with different PFIC subtypes showed that odexibat could effectively reduce serum bile acid levels and improve pruritus symptoms, requiring dose adjustment in some patients to achieve a better therapeutic response. These results support the use of odexibat in patients with various PFIC subtypes.

In summary, odexibat, as an IBAT inhibitor, has significant efficacy and good safety in PFIC pediatric patients. It improves patients' quality of life by reducing serum bile acid levels and improving pruritus symptoms. However, further studies are needed to evaluate its efficacy and safety across different PFIC subtypes, long-term use, and in combination with other treatments. In addition, patients should regularly monitor liver function and vitamin levels when using odexibat to detect and deal with possible adverse reactions in a timely manner.

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