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Long-Term Outlook for Alagille Syndrome

Current
Therapies

Off-label pharmacologic agents are often used to manage symptoms of Alagille syndrome with limited success. As a result, surgical biliary diversion and liver transplant represented the only effective treatment option for most liver symptoms of Alagille syndrome, but they present several issues. In the case of a surgical biliary diversion, individuals typically require a stoma, which many view as stigmatizing. In the case of liver transplants, issues include lifelong immunosuppressive therapy or surgical complications. These are practical limitations to treatment.1

Recently, a class of medicines called IBAT inhibitors have been approved by the FDA to treat the itch in certain liver disorders.1-3

Current Therapies
Treatment Landscape

Treatment Goals

For patients with Alagille syndrome, there is a need for more noninvasive treatment options.1,4

The goals of these treatments should be to1,4:

Reducethe accumulation of bile acids in the liver and bloodstream

Relievecholestatic pruritus

Reducexanthomas

Limitthe progression of liver disease

Improvelong-term prognosis

Promising
Research

There is promising research underway for additional treatment options. These may play a crucial role in reducing the symptoms of Alagille syndrome.1

Significant scientific discovery for better treatments of Alagille syndrome could mean other children won’t have to suffer like my daughter. –Jazmyn R.
Promising Research

Alagille Syndrome Resources

ASBT=apical sodium-dependent bile acid transporter; IBAT=ileal bile acid transporter.
References: 1. Kamath BM, Stein P, Houwen RHJ, Verkade HJ. Potential of ileal bile acid transporter inhibition as a therapeutic target in Alagille syndrome and progressive familial intrahepatic cholestasis. Liver Int. 2020;40(8):1812-1822. doi:10.1111/liv.14553 2. Novel Drug Approvals for 2021. U.S. Food and Drug Administration. Accessed August 15, 2022. https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2021 3. Albireo Completes Enrollment in Pivotal Phase 3 ASSERT Study of Bylvay™ (odevixibat) in Alagille Syndrome.;2022. Available at: https://ir.albireopharma.com/news-releases/news-release-details/albireo-completes-enrollment-pivotal-phase-3-assert-study. Accessed August 15, 2022. 4. Spinner NB, Gilbert MA, Loomes KM, Krantz ID. Alagille syndrome. In: Adam MP, Ardinger HH, Pagon RA, et al, eds. GeneReviews. Seattle (WA): University of Washington, Seattle. Published online: May 19, 2000. Updated: December 12, 2019. https://www.ncbi.nlm.nih.gov/books/NBK1273/pdf/Bookshelf_NBK1273.pdf