Alagille syndrome is a genetic disorder, in which a person have fewer bile ducts than normal in liver. Alagille syndrome can be passed from parent to child or can happen naturally. Alagille syndrome not only affects the liver, it can also cause skin, heart, facial, kidney, and eye abnormalities. While there is no known cure for Alagille syndrome, there are treatments that can help control the symptoms. Possible treatments may include medications such as Ursodeoxycholic acid, Cholestyramine, and Rifampicin that increases the flow of bile and careful management of diet to minimize nutrition and vitamin related problems. In severe cases, a liver transplant may be necessary.
Several pharmaceutical companies manufacture drugs to control the symptoms such as Teva Pharmaceutical Industries Ltd., AbbVie, Inc., Glenmark Pharmaceuticals and others. There are two drugs under the clinical trials that have the potential to treat Alagille syndrome more effectively than other medications. The two drugs are Odevixibat and Maralixibat. Furthermore, the U.S. FDA is providing support to the manufacturers by giving orphan drug designation or breakthrough designation to the new drugs.
Market Dynamics
Key players in the market are gaining huge amount of funding for the research of Alagille syndrome treatment for new drugs, which is expected to boost the Alagille syndrome treatment market growth over the forecast period. For instance, the Alagille Syndrome Alliance, an international nonprofit started in 1993, offers funding for Research Grant Program for ALGS and Science for research and development of novel therapies for Alagille syndrome treatment and encourage ALGS research in new investigators. The grant offers US$ 30,000 for each year for development of novel drug compounds.
However, lack of novel therapies is expected to restrain the Alagille syndrome treatment market growth. For instance, according to the National Organization for Rare Disorders, Alagille syndrome is caused by mutations in one of two genes – the JAG1 gene or the NOTCH2 gene. Mutations of the JAG1 gene have been identified in over 88% of cases of Alagille syndrome. Mutations in the NOTCH2 gene account for less than 1% of cases. These mutations are inherited in an autosomal dominant pattern, and finding cure for genetic disorders require extensive R&D, which is often not achieved.
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