Alagille Syndrome Market

• The Alagille Syndrome market is projected to grow based on various factors, such as increasing awareness, advancements in diagnostic technologies, and the introduction of novel treatment options for the condition.
• DelveInsight’s analysts estimated that there had been an increase in Alagille syndrome occurrences in recent years, which may be attributed to factors such as improved awareness or diagnostic advancements.
• Companies such as Ipsen, Takeda, and others have emerged as key players in the market, taking the lead in developing their respective assets for the treatment of Alagille Syndrome. With the anticipated launch of these therapies between 2023 and 2032, the overall Alagille Syndrome therapeutic market is expected to experience substantial growth at a significant compound annual growth rate (CAGR).

DelveInsight’s report titled “Alagille Syndrome Market Insights, Epidemiology, and Market Forecast—2032” comprehensively analyzes Alagille Syndrome. The report includes a detailed examination of the historical and projected epidemiology data that includes diagnosed prevalent cases of Alagille Syndrome further segmented by gender and age. The report offers an in-depth understanding of the various aspects related to the patient population, including diagnosis, prescriptions patterns, physician perspectives, market access, treatment, and future market developments for the seven major markets, including the United States, EU4 (Germany, France, Italy, and Spain) and the UK, and Japan from 2019 to 2032.

In order to gauge the Alagille Syndrome market’s overall potential and identify business opportunities, the report discusses current Alagille Syndrome treatment practices and algorithms as well as unmet medical needs.

Alagille Syndrome Overview

Alagille syndrome, also known as Alagille-Watson syndrome or arteriohepatic dysplasia, is a rare genetic disorder that primarily affects the liver and other organs caused by mutations in the JAG1 or NOTCH2 genes, which play a role in the development of various organs and tissues during embryonic development. The liver, heart, eyes, and kidneys are often affected in Alagille syndrome. Alagille syndrome impacts various organs, so it typically necessitates a multidisciplinary approach involving different specialists. These specialists may be involved in cardiac interventions, managing liver-related complications, monitoring growth and development, treating eye abnormalities, and providing supportive care and genetic counseling.

Alagille Syndrome Diagnosis and Treatment Algorithm

Diagnosing Alagille syndrome involves a combination of clinical evaluation, assessment of symptoms, and genetic testing to identify mutations in the JAG1 or NOTCH2 genes. Additionally, imaging studies such as ultrasound, echocardiogram, and liver biopsy may be performed to evaluate the extent of organ involvement. Diagnosing Alagille syndrome can pose several challenges due to its diverse and variable clinical presentation.

Treatment for Alagille syndrome is generally focused on managing the specific symptoms and complications that arise. This may involve medication to alleviate itching, surgical interventions to address heart defects or bile duct abnormalities, dietary modifications to ensure adequate nutrition and regular monitoring of liver function.

Treating Alagille syndrome involves several difficulties due to the involvement of multiple organs and the variability in symptom severity among patients. A primary challenge revolves around the scarcity of tailored therapies that specifically address the unique characteristics of Alagille syndrome.

Alagille Syndrome Epidemiology

The Alagille Syndrome epidemiology section provides insights into the historical and current Alagille Syndrome patient pool and forecasted trends for seven individual major countries. It helps recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the report also provides the diagnosed patient pool, its trends, and assumptions undertaken.

The epidemiology section of the Alagille syndrome report offers information on the patient populations, including historical and projected trends for each of the seven major markets. Examining key opinion leader views from physicians or clinical experts can assist in identifying the reasons behind historical and projected trends. The diagnosed patient pool, their trends, and the underlying assumptions are all included in this section of the report.

Key Findings

The offspring of a patient with Alagille syndrome has a 50% chance of inheriting a gene mutation, whereas among those affected with Alagille syndrome, 50–70% of individuals have a mutation de novo. No correlation has been found between a specific mutation and expressed phenotype. In more than 90% of cases, mutations in the JAG1 gene cause Alagille syndrome.

• As per the estimates by DelveInsight, the total cases of Alagille syndrome in the 7MM were around 18,000 cases in 2022.
• In 2022, among EU4 and the UK, Germany accounted for the highest number of cases, followed by France, according to estimates by DelveInsight.
• According to Diaz et al. (2019), it is estimated that the prevalence of Alagille syndrome varies from 1:30,000 to 1:100,000.
• In 2022, there was about a 4:3 ratio of males and females diagnosed with ALGS in the United States. Assessments, as per DelveInsight’s analysts, show that the overall prevalence of gender cases of ALGS will increase in the coming years by 2032.

Get a more detailed overview of How Alagille Syndrome Epidemiology will evolve by 2032: Alagille Syndrome Epidemiology Forecast

Alagille Syndrome Market Outlook

The current treatment for Alagille Syndrome involves a comprehensive approach that includes management of liver-related complications through medications to improve bile flow and liver function, interventions for cardiac defects if present, monitoring and support for growth and development, regular eye examinations for any associated abnormalities, and the provision of supportive care and genetic counseling to address the overall well-being of individuals with Alagille Syndrome. LIVMARLI and BYLVAY have recently received approval for treating Alagille Syndrome.

The ongoing research and development efforts focused on identifying novel therapeutic interventions and genetic targets hold promise for developing innovative treatment options. The advent of precision medicine and personalized therapies tailored to an individual’s genetic profile further adds to the market’s potential growth. Collaborative efforts between pharmaceutical companies, research institutions, and advocacy groups will likely drive investment and support for Alagille Syndrome research and development.

According to DelveInsight, the Alagille Syndrome market in the 7MM is expected to change during the study period 2019–2032 significantly.

Alagille syndrome Drug Chapters

Marketed Alagille Syndrome Drugs

LIVMARLI (maralixibat): Mirum Pharmaceuticals

In September 2021, FDA-approved LIVMARLI (maralixibat) to treat cholestatic pruritus associated with Alagille syndrome. In September 2022, LIVMARLI was approved for the same in the European Union. It is an oral solution designed for daily use that acts as an inhibitor of the ileal bile acid transporter (IBAT) and is the only medication approved in both the US and Europe for the treatment of cholestatic pruritus specifically associated with Alagille syndrome.

In September 2021, Mirum and Takeda entered into an exclusive licensing agreement to develop and commercialize maralixibat in Japan.

In December 2022, Takeda received Orphan Drug Designation from the Ministry of Health, Labour, and Welfare for Maralixibat chloride (TAK-625) for the expected indication of Alagille syndrome (ALGS)

BYLVAY (odevixibat): Ipsen/Albireo

In June 2023, Ipsen received US FDA approval for BYLVAY (odevixibat) to treat patients aged 12 months and above with cholestatic pruritus caused by Alagille syndrome. It is a once-a-day, non-systemic ileal bile acid transport inhibitor (IBATi) that acts locally in the small intestine and has minimal systemic exposure. Ipsen picked up this drug in its January 2023 acquisition of Albireo.

Emerging Alagille Syndrome Drugs

The pipeline for Alagille Syndrome is relatively dry. Travere Therapeutics, in collaboration with the National Institutes of Health (NIH), National Center for Advancing Translational Sciences (NCATS), and the patient advocacy foundation Alagille Syndrome Alliance (ALGSA), is engaged in a cooperative research and development agreement (CRADA) to conduct research activities jointly focused on identifying and developing potential innovative therapeutics specifically for Alagille syndrome. Their research is in the preclinical stage.

Explore more about the emerging therapies and key companies actively working in the market: Alagille Syndrome Pipeline Insights

Alagille Syndrome Market Segmentation

DelveInsight’s ‘Alagille Syndrome Market Insights, Epidemiology, and Market Forecast – 2032’ report provides a detailed outlook of the current and future Alagille Syndrome market, segmented within countries, by therapies, and by classes. Further, the Alagille Syndrome market of each region is then segmented by each therapy to provide a detailed view of the current and future market share of all therapies.

Alagille Syndrome Market Size by Countries

The total Alagille Syndrome market is analyzed for individual countries (the United States Market, EU4 (Germany, France, Italy, and Spain) and the UK market, and Japan). The United States accounted for a larger portion of the Alagille Syndrome market in 7MM in 2022 due to the high prevalence of the condition and the higher cost of treatments. This dominance is predicted to continue with the potential early entry of new products.

Alagille Syndrome Market Size by Therapies

Currently, limited approved therapies are specifically designed for Alagille Syndrome, leading to a relatively small market size. However, the landscape is evolving with increasing awareness, advancements in research, and ongoing clinical trials. The potential introduction of targeted therapies, including ileal bile acid transporter inhibitors and other novel treatment modalities, will likely expand the market. The current treatment landscape involves approved and off-label therapies. LIVMARLI and BYLVAY are the approved treatments, and off-label might include rifampicin, antihistamines, opioid antagonists, cholestyramine, etc., for symptomatic relief. As more therapies emerge and gain regulatory approvals, the market size for Alagille Syndrome therapies is projected to increase, providing new options and improved outcomes for individuals living with this rare genetic disorder.

Note: Detailed market segment assessment will be provided in the final report.

Alagille Syndrome Drugs Uptake

This section focuses on the sales uptake of potential drugs that have recently launched or are anticipated to be launched in the Alagille Syndrome market between 2019 and 2032. It estimates the market penetration of the drugs for a given country, examining their impact within and across classes and segments. It also touches upon the financial and regulatory decisions contributing to the probability of success (PoS) of the drugs in the market.

The emerging therapies are analyzed based on various attributes such as safety and efficacy in randomized clinical trials, order of entry, and other market dynamics, and the unmet need they fulfill in the Alagille Syndrome market.

Note: Detailed assessment of drug uptake and attribute analysis will be provided in the full report on Alagille Syndrome.

Alagille Syndrome Market Access and Reimbursement

DelveInsight’s ‘Alagille Syndrome Market Insights, Epidemiology, and Market Forecast – 2032’ report provides a descriptive overview of the market access and reimbursement scenario of Alagille Syndrome.

This section includes a detailed analysis of the country-wise healthcare system for each therapy, enlightening the market access, reimbursement policies, and health technology assessments.

Alagille Syndrome KOL Views

To keep up with current market trends and fulfill gaps in secondary findings, we interview KOLs and SMEs’ working in the Alagille Syndrome domain. Their opinion helps to understand and validate current and emerging therapies and treatment patterns or Alagille Syndrome market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Alagille Syndrome: KOL Insights

DelveInsight’s analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. These KOLs were from organizations, institutes, and hospitals, such as the Children’s Hospital of Philadelphia in the US, Deutsche Leberhilfe in Germany, and others.

“Due to the complexity of the disease, it is extremely difficult for scientists to research. ALGS typically has three or more characteristics: organ involvement, facial characteristics, skeletal characteristics, and liver or heart involvement.”

“The current pharmacological development is very promising, especially for patients suffering from chronic pruritus in inflammatory dermatoses, chronic kidney diseases, and hepatobiliary diseases. However, there are still several pruritic diseases in which neither mediators nor specific target populations (e.g., children) nor stages of diseases have been identified; however, it can be assumed that within the next 10 years, major changes in the possibilities of antipruritic treatment will take place.”

Note: Detailed assessment of KOL Views will be provided in the full report of Alagille Syndrome.

Competitive Intelligence Analysis

We perform Competitive and Market Intelligence analysis of the Alagille Syndrome Market using various Competitive Intelligence tools, including SWOT analysis, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Alagille Syndrome Pipeline Development Activities

The Alagille Syndrome market report provides insights into therapeutic candidates in Phase II and III stages. It also analyses Alagille Syndrome key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers collaborations, acquisitions and mergers, licensing, patent details, and other information for emerging Alagille Syndrome therapies.

Alagille Syndrome Market Report Insights

• Alagille Syndrome Patient Population
• Alagille Syndrome Therapeutic Approaches
• Alagille Syndrome Pipeline Analysis
• Alagille Syndrome Market Size and Trends
• Alagille Syndrome Market Opportunities
• Impact of Upcoming Alagille Syndrome Therapies

Alagille Syndrome Report Key Strengths

• Alagille Syndrome 10 years Forecast
• 7MM Coverage
• Alagille Syndrome Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Alagille Syndrome Market
• Alagille Syndrome Drugs Uptake

Alagille Syndrome Market Report Assessment

• Alagille Syndrome Current Treatment Practices
• Alagille Syndrome Unmet Needs
• Alagille Syndrome Pipeline Product Profiles
• Alagille Syndrome Market Attractiveness

Key Questions

• What are the key findings of the market across 7MM, and what country will have the largest Alagille Syndrome market size during the forecast period (2023–2032)?
• What are the challenges in the management of Alagille Syndrome?
• At what CAGR is the Alagille Syndrome market, and is epidemiology expected to grow in the 7MM during the forecast period (2023–2032)?
• In what ways would the unmet needs impact market dynamics and subsequently influence the analysis of related trends? Are there any approved medications specifically for Alagille Syndrome?
• What would be the forecasted patient pool of Alagille Syndrome in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), the UK, and Japan?
• Which medications have received approval for the treatment of Alagille Syndrome? How would the expiration of their patents impact the market?
• What is the outlook for the Alagille Syndrome market?
• How does genetic counseling play a role in the Alagille Syndrome market?