Case study explores off-label use of odevixibat in ALGS

Odevixibat, a drug designed to reduce bile acid levels, has long been recognized as a potential breakthrough therapy for progressive familial intrahepatic cholestasis. However, a recent case study sheds light on its efficacy in addressing symptoms associated with Alagille syndrome, providing renewed hope for patients suffering from this challenging condition.

A male pediatric patient initially presented with elevated liver enzyme and bile acid levels, accompanied by bile duct hypoplasia, mild liver fibrosis, and pruritus, prompting a diagnosis of progressive familial intrahepatic cholestasis. Traditional treatments involving ursodeoxycholic acid and naltrexone failed to produce the desired outcome.

The patient was treated with odevixibat, an investigational drug primarily designed for progressive familial intrahepatic cholestasis, and experienced significant improvements in both serum bile acid levels and pruritus after several weeks.

Genetic testing confirmed the patient had Alagille syndrome and odevixibat was continued off-label. Serum bile acid levels dropped to within the normal limit and pruritus was relieved.

Reference
Ganschow R, Maucksch C. Odevixibat Treatment of Alagille Syndrome: A Case Report. JPGN Rep. 2023;4(2):e301. doi: 10.1097/PG9.0000000000000301. PMID: 37200711; PMCID: PMC10187842.