Alagille Syndrome Market Size, Share, and Industry Analysis, By Drug Type (Ileal Bile Acid Transporter (IBAT) Inhibitors, Antihistamines, Opioid Antagonists, Bile Acid Sequestrants, and Others), By Route of Administration (Oral and Parenteral), By Distribution Channel (Hospital Pharmacies, Drug Stores & Retail Pharmacies and Online Pharmacies), and Regional Forecast, 2025-2032

The global alagille syndrome market is experiencing significant growth due to the increasing number of R&D advancements, clinical trials, and various therapeutic improvements for the treatment of syndrome. The syndrome is a rare inherited condition characterized by liver damage caused by abnormalities in the bile ducts. Bile builds up in individuals suffering from Alagille Syndrome due to fewer number of bile ducts to drain the bile. The growing awareness regarding the condition, along with increasing diagnosis among the population, are some of the factors boosting the demand for novel therapies for this condition. 

• According to a 2023 article published by the National Center for Biotechnology Information (NCBI), the estimated prevalence of Alagille Syndrome ranges from 1 in 30,000 to 1 in 100,000 individuals.

Alagille Syndrome Market Driver

Rising Product Development to Fuel Market Growth

The rising prevalence of Alagille syndrome among the population, along with the growing diagnosis rate, is leading to an increasing demand for novel therapies for the condition. The increasing focus of companies on developing and introducing novel therapies for Alagille syndrome is expected to boost market growth.

• In February 2022, CANbridge Life Sciences Ltd. announced the approval of CAN108 for the treatment of Alagille syndrome under the Early and Pilot Implementation Policy in the Boao Lecheng International Medical Tourism Pilot Zone.

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According to the 2023 annual reports of key companies, the R&D expenditure of Mirum Pharmaceuticals Inc., accounted for 55.1% of its total revenue. Similarly, the R&D expenditure proportion (%) in total revenues for these 5 companies engaged in the Alagille syndrome market ranged from 2.2% to 55.1% in 2023.

Alagille Syndrome Market Restraint

 High Costs Associated with the Treatment May Hamper the Market Growth

The high costs of the medication may limit access to therapy and treatment for the patients, especially in emerging countries such as India, China, Brazil, and others. Along with this, the limited healthcare infrastructure can be responsible for the lower diagnosis rate of the condition among the population in these countries.

• According to a 2023 article published by the National Center for Biotechnology Information (NCBI), the treatment with Livmarli is expected to cost around USD 128,000 – USD 1.9 million per patient per year in the first year of the treatment

Alagille Syndrome Market Opportunity

Advancements in Molecular Genetic Testing to Create New Growth Opportunities

The advancements in molecular genetic testing have aided in increasing the rate of diagnosis of Alagille syndrome which presents a large potential patient pool requiring the treatment. In addition, there are several technological advancements in the field of genome sequencing, genetic testing, and others which eliminates the need for liver biopsy to diagnose Alagille syndrome among the population.

• In August 2022, Mayo Clinic Laboratories launched thirty-two new tests for various genetic conditions, including Alagille syndrome. The test utilizes next-generation sequencing to detect single nucleotide, small deletion-insertion, and copy number variants in two genes associated with Alagille syndrome.

Segmentation

Key Insights

The report covers the following key insights:

• Prevalence of Alagille Syndrome, By Key Countries, 2023
• Pipeline Analysis, By Key Players
• New Product Launches, By Key Players
• Key Industry Developments (Mergers, Acquisitions, Partnerships)
• Impact of COVID-19 on the Market 

Analysis by Drug Type

By drug type, the market is segmented into ileal bile acid transporter (IBAT) inhibitors, antihistamines, opioid antagonists, bile acid sequestrants, and others.

The ileal bile acid transporter (IBAT) inhibitors have shown promising results in the treatment of cholestatic diseases associated with Alagille syndrome. The growing focus of companies to develop and introduce therapies with IBAT inhibitor drug class is expected to spur the adoption of these products in the market.

• In June 2023, Ipsen Pharma received the U.S. FDA approval for its Bylvay, an ileal bile acid transport (IBAT) inhibitor, for the treatment of cholestatic pruritus in patients from 12 months of age with Alagille syndrome (ALGS).

Analysis by Route of Administration

By routes of administration, the market is divided into oral and parenteral.  

The oral segment accounts for a major market share in 2023. The increased preference of oral drugs among the patients, owing to its convenience and versatility in the formulation of dosage forms, is a prominent factor fueling the growth of the segment in the market. Along with this, the growing focus of companies to develop and introduce novel oral drugs for the treatment is expected to spur the growth of the segment.

• According to clinicaltrials.gov, Takeda Pharmaceutical Company Limited is conducting a Phase III study to evaluate the safety and efficiency of its oral drug, TAK-625, in the treatment of Alagille syndrome in patients. 

Analysis by Distribution Channel

Based on distribution channel, the market is divided into hospital pharmacies, drug stores & retail pharmacies, and online pharmacies.

The hospital pharmacies segment will hold a leading share in the global market in 2023. The rising number of hospitals globally catering to the rising number of patients visiting hospitals for diagnosis and treatment plays a vital role in the segment growth.

• According to an article published by PubMed in March 2023, data from the Central Register of Pharmacies, as of September 2022, revealed that there are a total of 565 hospital pharmacies in Poland.

Regional Analysis

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By region, the market has been studied across North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa.

Among regions, North America dominates the market in 2023. The rising number of newborns diagnosed with Alagille syndrome is resulting in a growing demand for novel therapies for the condition among the population.

• In February 2023, a research article released by the National Institutes of Health (NIH) noted that a comprehensive study documented a total of 1,543 children with Alagille syndrome (ALGS). The study findings indicated that 29% of the patients were identified in North America.

The Europe market is projected to grow owing to the rising incidence of the syndrome in countries such as the U.K., Germany, France, and others, which is leading to a rising demand for novel and effective therapies.

• In April 2022, the European Medical Journal published an article highlighting the reported incidence of Alagille Syndrome (ALGS) as 1 in 30,000 to 50,000 in Europe. This underscores the rarity of this disorder and the critical need for accurate diagnosis and proper treatment. Managing this condition effectively requires specialized medical attention and care.

The rising incidence of Alagille syndrome (ALGS) in the Asia Pacific region has prompted the development of advanced therapeutic interventions to address the specific needs of individuals affected by this condition.

• In June 2024, Takeda announced the submission of a new drug application (NDA) to the Ministry of Health, Labor, and Welfare (MHLW) for maralixibat chloride in partnership with Mirum Pharmaceuticals. This application pertains to "maralixibat," an ileal bile acid transporter (IBAT) inhibitor intended for the treatment of Alagille syndrome.

Key Players Covered

The global Alagille syndrome market is consolidated with the presence of certain key players with approved product offerings.

The report includes the profiles of the following key players:

• CANbridge Life Sciences Ltd. (China)
• Mirum Pharmaceuticals, Inc. (U.S.)
• Ipsen Pharma (France)
• Takeda Pharmaceutical Company Limited. (Japan)
• AbbVie Inc. (U.S.)
• GLENMARK PHARMACEUTICALS LTD. (India)
• Strides Pharma Science Limited (India)
• Zydus Group (India)
• Alkermes (Ireland)

Key Industry Developments

• In August 2024, the researchers at the University of Pennsylvania and Children’s Hospital of Philadelphia (CHOP) developed a high-throughput assay to increase the accuracy of the diagnosis of Alagille syndrome.
• In March 2023, Ipsen acquired Albireo Pharma, Inc., enhancing its Rare Disease portfolio with promising therapeutics for rare liver conditions and strengthening its scientific and commercial capabilities.