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Alagille Syndrome
Conference Roundup

New treatments on the horizon for management of ALGS

Posted on July 7, 2021
Rare Disease

There is no approved treatment for ALGS, instead, the management of this rare disease aims to alleviate symptoms, specifically pruritus. During an industry symposium event at the 6th World Congress of Paediatric Gastroenterology, Hepatology and Nutrition, Dr Emmanuel Gonzales discussed treatments on the horizon for patients with ALGS.

Pruritus is commonly controlled with medications such as ursodeoxycholic acid, rifampicin, antihistamines, bile salt binding agents, amongst others, however, side effects are often troublesome.

Cholestatic clinical manifestation can result in substantial risk for liver transplantation in patients with ALGS.

Two novel approaches to improve outcomes in ALGS that are currently being investigated are maralixibat (Mirum Pharmaceuticals) and odevixibat (Albireo).

In the ITCH trial, maralixibat was found to possibly reduce pruritus with adverse events similar to those reported in patients treated with placebo.

In the ICONIC phase 2 study of maralixibat in ALGS, a significant reduction in serum bile acid levels was maintained over long term and a significant and sustained improvement in pruritus was demonstrated, with 84% of patients experiencing a clinically meaningful decrease in pruritus during the 48-week period. Overall, treatment was well tolerated.

Dr Gonzales said more detailed results would be published in a medical journal.

In a phase 2 study, odevixibat was investigated in children diagnosed with pruritus due to chronic cholestasis. In the 24 patients included, odevixibat was well tolerated, and in the 6 patients with ALGS a trend in decreased serum bile acid levels was seen. A correlation was observed in this study, Dr Gonzales said, between the reduction in serum bile acid levels and reduction in pruritus.

Odevixibat is currently being investigated in the phase 3 ASSERT trial.

Dr Gonzales concluded his presentation by saying that ASBT inhibitors have demonstrated clinical results and are a promising therapy for patients with ALGS.

Reference
Gonzales E. Can novel treatments offer new hope for children with ALGS? Industry Symposium Presented at: 6th World Congress of Paediatric Gastroenterology, Hepatology and Nutrition.

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