Alagille Syndrome Treatment Market: Introduction
- Alagille syndrome is a rare genetic disorder that affects various organs including the liver, kidney, heart, eyes, and blood vessels. In this condition, bile accumulates in the liver and causes liver damage. Alagille syndrome is associated with liver diseases, hepatomegaly, and biliary atresia. This syndrome is caused due to mutation of the JAG1 gene and can be passed from parent to child. The symptoms of Alagille syndrome usually can be seen shortly after birth or in early infancy. Alagille syndrome can be treated through medication and surgery.
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Key Drivers, Restraints, and Opportunities of Global Alagille Syndrome Treatment Market
- A surge in R&D activities and clinical trials coupled with the rising investment and funding gained by key market player for R&D of novel drugs for the treatment of Alagille syndrome is a major factor boosting the market. Several key players are focusing on the development of new drugs for the treatment of Alagille syndrome. These compounds are under clinical trials and are expected to receive USFDA approval in the future.
- For instance, in 2024, Mirum Pharmaceuticals, Inc., announced submission of the first portion of its rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for maralixibat. Maralixibat, a novel, minimally absorbed, orally administered apical sodium-dependent bile acid transporter (ASBT) inhibitor is being assessed cholestatic pruritus treatment in patients suffering from Alagille syndrome (ALGS). Therefore, the robust pipeline of drugs for treatment of Alagille syndrome is anticipated to augment the market.
- Rising awareness about Alagille syndrome is also projected to propel the Alagille syndrome treatment market during the forecast period. However, a lack of a healthcare budget in some middle-income economies is estimated to restrain the market.
- The COVID-19 virus outbreak spread to almost all countries worldwide. There was an imposition of a travel ban and shutdown of manufacturing units, offices & marketplaces by governments in order to curb the spread of the virus. The COVID has also resulted in disruption across communities, patients, and clinicians. The drug development process is also facing disruption as the capacity of lab experts working remotely is reduced. Moreover, the clinical trials for drugs are severely impacted by the disruptions in both new enrollments and existing patients on therapies.
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North America to Capture Major Share of Global Alagille Syndrome Treatment Market
- North America is expected to account for the major share of the global Alagille syndrome treatment market. The dominance is attributed to the presence of key market players in the region. Moreover, a surge in the adoption of inorganic strategies by market players is projected to augment the market.
- The Alagille syndrome treatment market in Asia Pacific is projected to expand at a rapid pace during the forecast period due to various programs introduced by market players in order to commercialize their products
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Key Players Operating in Global Alagille Syndrome Treatment Market
The key market players have been focusing on organic growth strategies such as product launches, product approval, and others. These activities have created opportunities for the expansion of the business and customer base of these players. Major players operating in the global Alagille syndrome treatment market is listed below:
- Albireo Pharma, Inc.
- Bioarray S.L.
- Mirum Pharmaceuticals
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