Alagille syndrome (ALGS) is a rare, genetic condition that can affect different parts of the body including liver, heart, kidneys, eyes, face, and bones. Moreover, liver problems results from having fewer small bile ducts than normal in the liver. This leads to bile building-up inside the liver, which in turn causes liver scarring and damage. This condition can be referred to as Alagille-Watson syndrome, arteriohepatic dysplasia (AHD), cardiovertebral syndrome or syndromic bile duct paucity.
Major treatment drugs used for Alagille syndrome include Ursodeoxycholic acid, which is one of the most prescribed medications for the treatment of pruritus (itching) associated with the Alagille syndrome. Other medications for Alagille syndrome such as cholestyramine, rifampin, and naltrexone can be used in combination with therapies, if required. Furthermore, enteral feeding devices such as nasogastric feeds or gastrostomy tube may be required to maintain caloric intake majorly in children with Alagille syndrome, who cannot absorb fats and vitamins. The major treatment drugs for Alagille syndrome in pipeline include Odevixibat and Maralixibat.
Alagille Syndrome Treatment Market – Impact of Coronavirus (Covid-19) Pandemic
The COVID-19 virus outbreak has spread across the globe and it has created profound disruption across communities, patients, and clinicians. The drug development process is facing profound disruption, as the lab specialists adjust to remote work environments and lab capacity is reduced. Clinical trials for drugs are also severely affected with disruptions in both new enrollment and in existing patients on therapies. According to the clinicaltrial.gov, as of April 2020, there were over 2,850 trials and around 900,000 patients enrolled at trial sites in regions that were in partial or complete lockdown due to COVID-19 restrictions. The R&D labs are operating at below 50% of normal capacity.
Alagille syndrome treatment market is estimated to be valued at US$ 69.6 million in 2020 and is expected to exhibit a CAGR of 45.2% during the forecast period (2020-2027).
|Base Year:||2019||Market Size in 2019:||US$ 69.6 Mn|
|Historical Data for:||2017 to 2019||Forecast Period:||2020 to 2027|
|Forecast Period 2020 to 2027 CAGR:||45.2%||2027 Value Projection:||US$ 948.7 Mn|
Teva Pharmaceutical Industries Ltd., AbbVie, Inc., Glenmark Pharmaceuticals Limited, Par Pharmaceuticals, Inc., Mylan Pharmaceuticals, Inc., Sanofi S.A., Novartis International AG, Akorn, Inc., Albireo Pharma, Inc., and Mirum Pharmaceuticals
|Restraints & Challenges:||
Figure 1. Alagille Syndrome Treatment Market Share (%) Analysis, By Drug, 2020 and 2027
Robust Pipeline for Alagille Syndrome and Increasing Investments and Funding for Research of Alagille Syndrome Drugs are Expected to drive Growth of the Global Alagille Syndrome Treatment Market during the Forecast Period
Major players in the market are focusing on developing new drug compounds for the treatment of Alagille syndrome, which are under clinical trials and are expected to receive approval form USFDA in the near future. Thus, the robust pipeline of drugs for Alagille syndrome treatment is expected to significantly contribute to growth of the global Alagille syndrome treatment market over the forecast period. For instance, on September 1, 2020, Mirum Pharmaceuticals, Inc., a biopharmaceutical company focusing on the development and commercialization of novel therapies for debilitating liver diseases, announced to submit the first portion of its rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for maralixibat. Maralixibat is a novel, minimally absorbed, orally administered apical sodium dependent bile acid transporter (ASBT) inhibitor being evaluated for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS). Mirum expects to complete the NDA submission in the first quarter of 2021.
Furthermore, increasing funding and investments for the treatment of Alagille syndrome is expected to drive the global Alagille syndrome treatment market growth. For instance, on December 14, 2020, Mirum Pharmaceuticals Inc. announced that they will receive around US$ 210 million funding from Oberland Capital Management LLC for its experimental liver disease drug maralixibat. The sum is split between US$ 200 million under a revenue-based funding agreement and US$ 10 million under an equity investment agreement signed between the companies. Mirum Pharmaceuticals Inc. is expected to initially receive US$ 60 million, including the US$ 10 million equity investment, by December 31, 2020. Oberland will release US$ 65 million upon the acceptance of the company U.S. FDA NDA application for maralixibat.
Alagille Syndrome Treatment Market – Restraints
Higher economic growth in low- and middle-income countries has not closed the gap in health spending, and global inequity in health spending has remained largely unchanged. For instance, according to a report on ‘New Perspectives on Global Health Spending for Universal Health Coverage’ by WHO published in 2015, high-income countries with only 16% of the global populace account for 80% of the global health spending. However, 76% of the world’s populace live in middle-income countries, but they account for less than 20% of global health spending. Low-income countries such as Afghanistan., Bangladesh, Burkina Faso. Burundi and others, with over half billion people, accounted for less than 1% of the world’s health spending in 2015. As per the same source, around 50 countries with a total population of 2.7 billion spent less than US$ 100 per capita, in 2015.
Alagille Syndrome Treatment Market – Regional Analysis
Among regions, North America is expected to hold dominant position in the global alagille syndrome treatment market over the forecast period, owing to the presence of major market players such as Mirum Pharmaceuticals, Albireo Pharma, Inc., and others. Moreover, increasing partnerships and collaborations by market players are expected to drive the market growth. For instance, on October 15, 2020, Mirum Pharmaceuticals entered into a partnership with EVERSANA, an independent provider of services to the lifescience industry for the commercialization and distribution of Maralixibat, indicated for the treatment of alagille syndrome, in the U.S.
In addition, Asia Pacific Alagille syndrome treatment market is expected to witness significant growth during the forecast period, owing to various programs launched by market players for commercialization of their products, which is expected to drive the Asia Pacific Alagille syndrome treatment market growth over the forecast period. For instance, on November 5, 2020, Mirum Pharmaceuticals announced extention of Expanded Access Program (EAP) in Australia for maralixibat, indicated for the treatment of patients suffering with cholestatic pruritus associated with Alagille syndrome (ALGS).
Figure 2. Alagille Syndrome Treatment Market Value (US$ Mn) & Y-o-Y Growth (%), 2016-2027
Alagille Syndrome Treatment Market - Competitive Landscape
Key players operating in the global Alagille syndrome treatment market include Teva Pharmaceutical Industries Ltd., AbbVie, Inc., Glenmark Pharmaceuticals Limited, Par Pharmaceuticals, Inc., Mylan Pharmaceuticals, Inc., Sanofi S.A., Novartis International AG, Akorn, Inc., Albireo Pharma, Inc., and Mirum Pharmaceuticals.
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.
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.
Key features of the study:
“*” marked represents similar segmentation in other categories in the respective section.