DelveInsight’s ‘Alagille Syndrome (ALGS) – Market Insights, Epidemiology, and Market Forecast—2030’ report deliver an in-depth understanding of the Alagille Syndrome (ALGS), historical and forecasted epidemiology as well as the market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.
The Alagille Syndrome (ALGS) market report provides current treatment practices, emerging drugs and their market share of the individual therapies, current and forecasted Alagille Syndrome (ALGS) symptoms market size from 2017 to 2030 segmented by seven major markets. The report also covers current Alagille Syndrome (ALGS) symptoms treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate the best of the opportunities. It assesses the underlying potential of the market.
Alagille Syndrome (ALGS) Overview
Alagille syndrome, also known as Alagille-Watson syndrome is a genetic disorder. It is an autosomal dominant disorder that is caused by a mutation in either the JAG1 or NOTCH2 gene. These genes are located on chromosomes 20 and 1, respectively, with more than 90% of the individuals having a mutation in JAG1. Although it is inherited in an autosomal dominant fashion in about half of cases, the mutation occurs as a new change ("de novo") in the individual and is not inherited from a parent. This disease is characterized by syndromic bile duct paucity and arteriohepatic dysplasia. This disease is characterized as a rare disease with an incidence of 1 in 30,000 children; it is equally prevalent in both genders.
However, several unmet needs of patients need to be addressed. It is essential that to provide holistic care to patients, novel diagnostic tools, and therapies that address the underlying cause of the disease need to be developed. Several challenges do exist like the rarity of the disease and poor research in the field.
It covers the details of conventional and current medical therapies and diagnosis available in the Alagille Syndrome (ALGS) market for the treatment of the condition. It also provides the country-wise treatment guidelines and algorithms across the United States, Europe, and Japan.
The DelveInsight Alagille Syndrome (ALGS) market report gives a thorough understanding of Alagille Syndrome (ALGS) symptoms by including details such as disease definition, symptoms, causes, physiology, and diagnosis. It also provides Alagille Syndrome (ALGS) symptoms of treatment algorithms and treatment guidelines for Alagille Syndrome (ALGS) symptoms in the US, Europe, and Japan.
A diagnosis of ALGS is made based upon identification of characteristic symptoms, a detailed patient history, a thorough clinical evaluation and a variety of specialized tests as the symptoms are highly variable and obtaining a diagnosis can be difficult. Surgical removal and microscopic study of liver tissue (liver biopsy) can reveal bile duct paucity. Although bile duct paucity is considered a key characteristic of Alagille syndrome, this finding is not always present in infants with the disorder.
The treatment of Alagille syndrome is directed toward the specific symptoms that are apparent in each individual. Treatment may require the coordinated efforts of a team of specialists. Pediatricians, gastroenterologists, cardiologists, ophthalmologists, and other
healthcare professionals may need to systematically and comprehensively plan an effective child’s treatment.
The Alagille Syndrome (ALGS) symptoms epidemiology division provides insights about the historical and current patient pool along with the forecasted trend for every seven 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 DelveInsight report also provides the diagnosed patient pool and their trends along with assumptions undertaken.
The diagnosed prevalent cases of Alagille Syndrome (ALGS) is increasing in 7MM during the study period, i.e., 2017–2030.
The disease epidemiology covered in the report provides historical as well as forecasted Alagille Syndrome (ALGS) symptoms epidemiology segmented as the Total Prevalent cases of Alagille Syndrome (ALGS), Gender-specific cases of Alagille Syndrome (ALGS), Age-specific cases of Alagille Syndrome (ALGS). The report includes the prevalent scenario of Alagille Syndrome (ALGS) symptoms in 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2017 to 2030.
The epidemiology segment also provides the Alagille Syndrome (ALGS) epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
The total prevalent cases of Alagille Syndrome (ALGS) associated in 7MM countries was 17,486 in 2017.
The drug chapter segment of the Alagille Syndrome (ALGS) report encloses the detailed analysis of Alagille Syndrome (ALGS) early-stage (Phase-I, II) pipeline drugs. It also helps understand the Alagille Syndrome (ALGS) clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.
Maralixibat: Mirum Pharmaceuticals
LUM001, also known as Maralixibat, is an apical sodium-dependent bile acid transporter (ASBT) inhibitor that helps in the treatment of Alagille syndrome. Alagille syndrome is caused by a mutation in JAG1 or NOTCH2 and has impaired bile duct development. As a result of which the liver function is compromised.
A4250, also known as Odevixibat, is an Ileal bile acid transporter inhibitor that is being developed by Albireo; for Alagille Syndrome. This disease has wide implications ranging from liver and heart dysfunction and is caused by a mutation in the JAG1 and NOTCH2 gene.
Products detail in the report…
The Alagille Syndrome (ALGS) market outlook of the report helps build the detailed comprehension of the historic, current and forecasted Alagille Syndrome (ALGS) market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology.
This segment gives a thorough detail of Alagille Syndrome (ALGS) market trend of each marketed drug and early-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria’s, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.
According to DelveInsight, Alagille Syndrome (ALGS) market in 7MM is expected to grow in the study period 2017–2030.
The first-line therapy would be ursodiol or a bile salt–binding resin such as cholestyramine. Antihistamines may be used as adjuvants, on an as-needed basis for symptomatic relief, especially overnight, whereas Rifampin and colesevelam would be considered as second- or third-line therapy. The use of opioid antagonists or statins is not well established in pediatrics, but anecdotally has been used with success. Partial external biliary diversion (PEBD) has been successful in several patients with AGS and is now a viable option for intractable pruritus and xanthomata. Biliary diversion should be considered in all children with AGS when medical management of pruritus fails. It should be noted, however, that the outcome of PEBD is poor in the setting of hepatic fibrosis, and therefore, a liver biopsy should be considered before offering this therapy.
This section includes a glimpse of the Alagille Syndrome (ALGS) market in 7MM. The market size of Alagille Syndrome (ALGS) in the seven major markets was USD 35.1 million in 2017.
The United States Market Outlook
This section provides the total Alagille Syndrome (ALGS) market size and market size by therapies in the United States.
The United States accounts for the largest market size of Alagille Syndrome (ALGS) in comparison to the EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan.
EU-5 Countries: Market Outlook
The total Alagille Syndrome (ALGS) market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are provided in this section.
Japan Market Outlook
The total Alagille Syndrome (ALGS) market Size and market Size by therapies in Japan are also mentioned.
This section focusses on the rate of uptake of the potential drugs recently launched or expected to get launched in the market during the study period 2017–2030. The analysis covers Alagille Syndrome (ALGS) market uptake by drugs; patient uptake by therapies; and sales of each drug.
This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allow the comparison of the drugs based on market share and size which again will be useful in investigating factors important in the market uptake and in making financial and regulatory decisions.
Alagille Syndrome (ALGS) Pipeline Development Activities
The report provides insights into the therapeutic candidate in Phase I, and II stages. It also analyses Alagille Syndrome (ALGS) key
players involved in developing targeted therapeutics.
Major players include Mirum Pharmaceuticals and Albireo.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition, and merger, licensing and patent details for Alagille Syndrome (ALGS) emerging therapies.
Approaching reimbursement, proactively, can have a positive impact both during the early stages of product development and well after product launch. In the report, we take reimbursement into consideration to identify economically attractive indications and market opportunities. When working with finite resources, the ability to select the markets with the fewest reimbursement barriers can be a critical business and price strategy.
Several drugs are coming in the market to treat Alagille syndrome-like Maralixibat, Odevixibat, etc. Pricing and reimbursement for these drugs is an issue of high need for approach producers, administrators, social insurance experts, industry pioneers, scholastics, and patients. Vagrant medications are probably not going to give esteem to cash due to their high cost for regularly unassuming adequacy. In any case, extra criteria are utilized to advise repayment choices in a few nations. These criteria may incorporate the earnestness of the sickness; the accessibility of different treatments to treat the illness, and the cost to the patient if the drug is not repaid. There is a requirement for a straightforward and proof-based approach toward vagrant medication pricing and reimbursement. Such an approach ought to be focused on showing the relative adequacy, cost-adequacy, and monetary feasibility of vagrant medications to educate pricing and reimbursement choices.
To keep up with current market trends, we take KOLs and SME’s opinion working in Alagille Syndrome (ALGS) domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns or Alagille Syndrome (ALGS) market trend. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs.
We perform Competitive and Market Intelligence analysis of the Alagille Syndrome (ALGS) Market by using various Competitive Intelligence tools that includes – SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.
Market Insights:
Epidemiology Insights:
1. Key Insights
2. Executive Summary of Alagille Syndrome
3. Competitive Intelligence Analysis for Alagille Syndrome
4. Alagille Syndrome: Market Overview at a Glance
4.1. Alagille Syndrome Total Market Share (%) Distribution in 2017
4.2. Alagille Syndrome Total Market Share (%) Distribution in 2030
5. Alagille Syndrome: Disease Background and Overview
5.1. Introduction
5.2. Sign and Symptoms
5.3. Pathophysiology
5.4. Risk Factors
5.5. Diagnosis
6. Patient Journey
7. Alagille Syndrome Epidemiology and Patient Population
7.1. Epidemiology Key Findings
7.2. Assumptions and Rationale: 7MM
7.3. Epidemiology Scenario: 7MM
7.3.1. Alagille Syndrome Epidemiology Scenario in the 7MM (2017-2030)
7.4. United States Epidemiology
7.4.1. Alagille Syndrome Epidemiology Scenario in the United States (2017-2030)
7.5. EU-5 Country-wise Epidemiology
7.5.1. Germany Epidemiology
7.5.1.1. Alagille Syndrome Epidemiology Scenario in Germany (2017-2030)
7.5.2. France Epidemiology
7.5.2.1. Alagille Syndrome Epidemiology Scenario in France (2017-2030)
7.5.3. Italy Epidemiology
7.5.3.1. Alagille Syndrome Epidemiology Scenario in Italy (2017-2030)
7.5.4. Spain Epidemiology
7.5.4.1. Alagille Syndrome Epidemiology Scenario in Spain (2017-2030)
7.5.5. United Kingdom Epidemiology
7.5.5.1. Alagille Syndrome Epidemiology Scenario in the United Kingdom (2017-2030)
7.5.6. Japan Epidemiology
7.5.6.1. Alagille Syndrome Epidemiology Scenario in Japan (2017-2030)
8. Treatment Algorithm, Current Treatment, and Medical Practices
8.1. Alagille Syndrome Treatment and Management
8.2. Alagille Syndrome Treatment Algorithm
9. Unmet Needs
10. Key Endpoints of Alagille Syndrome Treatment
11. Marketed Products
11.1. List of Marketed Products in the 7MM
11.2. Drug Name: Company Name
11.2.1. Product Description
11.2.2. Regulatory Milestones
11.2.3. Other Developmental Activities
11.2.4. Pivotal Clinical Trials
11.2.5. Summary of Pivotal Clinical Trial
List to be continued in report
12. Emerging Therapies
12.1. Key Cross
12.2. Drug Name: Company Name
12.2.1. Product Description
12.2.2. Other Developmental Activities
12.2.3. Clinical Development
12.2.4. Safety and Efficacy
12.2.5. Product Profile
List to be continued in report
13. Alagille Syndrome: Seven Major Market Analysis
13.1. Key Findings
13.2. Alagille Syndrome Market Size in 7MM
13.3. Alagille Syndrome Market Size by Therapies in the 7MM
14. Attribute analysis
15. 7MM: Market Outlook
15.1. United States: Market Size
15.1.1. Alagille Syndrome Total Market Size in the United States
15.1.2. Alagille Syndrome Market Size by Therapies in the United States
15.2. EU-5 countries: Market Size and Outlook
15.3. Germany Market Size
15.3.1. Alagille Syndrome Total Market Size in Germany
15.3.2. Alagille Syndrome Market Size by Therapies in Germany
15.4. France Market Size
15.4.1. Alagille Syndrome Total Market Size in France
15.4.2. Alagille Syndrome Market Size by Therapies in France
15.5. Italy Market Size
15.5.1. Alagille Syndrome Total Market Size in Italy
15.5.2. Alagille Syndrome Market Size by Therapies in Italy
15.6. Spain Market Size
15.6.1. Alagille Syndrome Total Market Size in Spain
15.6.2. Alagille Syndrome Market Size by Therapies in Spain
15.7. United Kingdom Market Size
15.7.1. Alagille Syndrome Total Market Size in the United Kingdom
15.7.2. Alagille Syndrome Market Size by Therapies in the United Kingdom
15.8. Japan Market Outlook
15.8.1. Japan Market Size
15.8.2. Alagille Syndrome Total Market Size in Japan
15.8.3. Alagille Syndrome Market Size by Therapies in Japan
16. Access and Reimbursement Overview of Alagille Syndrome
17. KOL Views
18. Market Drivers
19. Market Barriers
20. Appendix
20.1. Bibliography
20.2. Report Methodology
21. DelveInsight Capabilities
22. Disclaimer
23. About DelveInsight
*The table of contents is not exhaustive; the final content may vary.
List of Tables
Table 1: Features of Alagille Syndrome
Table 2: Molecular Genetic Testing Used in Alagille Syndrome
Table 3: Recommended Evaluations Following Initial Diagnosis in Individuals with Alagille Syndrome
Table 4: Classic Criteria, based on five body systems, for a diagnosis of Alagille syndrome
Table 5: Genes of Interest in the Differential Diagnosis of Alagille Syndrome (ALGS)
Table 6: Total cases of Alagille Syndrome in 7MM (2017–2030)
Table 7: Total Prevalent cases of Alagille Syndrome in the United States (2017–2030)
Table 8: Gender-specific cases of Alagille Syndrome in the US (2017–2030)
Table 9: Age-specific cases of Alagille Syndrome in the US (2017–2030)
Table 10: Total Prevalent cases of Alagille Syndrome in Germany (2017–2030)
Table 11: Gender-specific cases of Alagille Syndrome in Germany (2017–2030)
Table 12: Age-specific cases of Alagille Syndrome in Germany (2017–2030)
Table 13: Total Prevalent cases of Alagille Syndrome in France (2017–2030)
Table 14: Gender-specific cases of Alagille Syndrome in France (2017–2030)
Table 15: Age-specific cases of Alagille Syndrome in France (2017–2030)
Table 16: Total Prevalent cases of Alagille Syndrome in Italy (2017–2030)
Table 17: Gender-specific cases of Alagille Syndrome in Italy (2017–2030)
Table 18: Age-specific cases of Alagille Syndrome in Italy (2017–2030)
Table 19: Total Prevalent cases of Alagille Syndrome in Spain (2017–2030)
Table 20: Gender-specific cases of Alagille Syndrome in Spain (2017–2030)
Table 21: Age-specific cases of Alagille Syndrome in Spain (2017–2030)
Table 22: Total Prevalent cases of Alagille Syndrome in the United Kingdom (2017–2030)
Table 23: Gender-specific cases of Alagille Syndrome in the UK (2017–2030)
Table 24: Age-specific cases of Alagille Syndrome in the UK (2017–2030)
Table 25: Total Prevalent cases of Alagille Syndrome in Japan (2017–2030)
Table 26: Gender-specific cases of Alagille Syndrome in Japan (2017–2030)
Table 27: Age-specific cases of Alagille Syndrome in Japan (2017–2030)
Table 28: Treatment of Liver Manifestations in Individuals with Alagille Syndrome
Table 29: LUM001, Clinical Trial Description, 2020
Table 30: A4250, Clinical Trial Description, 2020
Table 31: Market Size of Alagille Syndrome in 7MM in USD Million (2017–2030)
Table 32: US Market Size of Alagille Syndrome in USD Million (2017–2030)
Table 33: US Market Size of Alagille Syndrome in USD Million (2017–2030)
Table 34: Market Size of Alagille Syndrome in Germany, USD Millions (2017–2030)
Table 35: Germany Market Size of Alagille Syndrome in USD Million (2017–2030)
Table 36: Market Size of Alagille Syndrome associated in France, USD Millions (2017–2030)
Table 37: France Market Size of Alagille Syndrome in USD Million (2017–2030)
Table 38: Market Size of Alagille Syndrome in Italy, USD Millions (2017–2030)
Table 39: Italy Market Size of Alagille Syndrome in USD Million (2017–2030)
Table 40: Market Size of Alagille Syndrome in Spain, USD Millions (2017–2030)
Table 41: Spain Market Size of Alagille Syndrome in USD Million (2017–2030)
Table 42: Market Size of Alagille Syndrome in the UK, USD Millions (2017–2030)
Table 43: UK Market Size of Alagille Syndrome in USD Million (2017–2030)
Table 44: Market Size of Alagille Syndrome in Japan, USD Millions (2017–2030)
Table 45: Japan Market Size of Alagille Syndrome in USD Million (2017–2030)
List of Figures
Figure 1: Epidemiology and Market Methodology
Figure 2: Flow diagram of genetic investigations and management for suspected ALGS patients
Figure 3: Frequency of symptoms
Figure 4: Total cases of Alagille Syndrome in 7MM (2017–2030)
Figure 5: Total Prevalent cases of Alagille Syndrome in the United States (2017–2030)
Figure 6: Gender-specific cases of Alagille Syndrome in the US (2017–2030)
Figure 7: Age-specific cases of Alagille Syndrome in the US (2017–2030)
Figure 8: Total Prevalent cases of Alagille Syndrome in Germany (2017–2030)
Figure 9: Gender-specific cases of Alagille Syndrome in Germany (2017–2030)
Figure 10: Age-specific cases of Alagille Syndrome in Germany (2017–2030)
Figure 11: Total Prevalent cases of Alagille Syndrome in France (2017–2030)
Figure 12: Gender-specific cases of Alagille Syndrome in France (2017–2030)
Figure 13: Age-specific cases of Alagille Syndrome in France (2017–2030)
Figure 14: Total Prevalent cases of Alagille Syndrome in Italy (2017–2030)
Figure 15: Gender-specific cases of Alagille Syndrome in Italy (2017–2030)
Figure 16: Age-specific cases of Alagille Syndrome in Italy (2017–2030)
Figure 17: Total Prevalent cases of Alagille Syndrome in Spain (2017–2030)
Figure 18: Gender-specific cases of Alagille Syndrome in Spain (2017–2030)
Figure 19: Age-specific cases of Alagille Syndrome in Spain (2017–2030)
Figure 20: Total Prevalent cases of Alagille Syndrome in the United Kingdom (2017–2030)
Figure 21: Gender-specific cases of Alagille Syndrome in the UK (2017–2030)
Figure 22: Age-specific cases of Alagille Syndrome in the UK (2017–2030)
Figure 23: Total Prevalent cases of Alagille Syndrome in Japan (2017–2030)
Figure 24: Gender-specific cases of Alagille Syndrome in Japan (2017–2030)
Figure 25: Age-specific cases of Alagille Syndrome in Japan (2017–2030)
Figure 26: Treatment protocol for cholestatic pruritus in Alagille syndrome. MARS¼ molecular adsorbent recirculation system; PEBD¼ partial external biliary diversion
Figure 27: Unmet Needs
Figure 28: Market Size of Alagille Syndrome in USD Million (2017–2030)
Figure 29: Market Size of Alagille Syndrome in the US, USD Millions (2017–2030)
Figure 30: Market Size of Alagille Syndrome in the US by therapies, USD Millions (2017–2030)
Figure 31: Market Size of Alagille Syndrome in Germany, USD Millions (2017–2030)
Figure 32: Market Size of Alagille Syndrome in Germany by therapies, USD Millions (2017–2030)
Figure 33: Market Size of Alagille Syndrome in France, USD Millions (2017–2030)
Figure 34: Market Size of Alagille Syndrome in France by therapies, USD Millions (2017–2030)
Figure 35: Market Size of Alagille Syndrome in Italy, USD Millions (2017–2030)
Figure 36: Market Size of Alagille Syndrome in Italy by therapies, USD Millions (2017–2030)
Figure 37: Market Size of Alagille Syndrome in Spain, USD Millions (2017–2030)
Figure 38: Market Size of Alagille Syndrome in Spain by therapies, USD Millions (2017–2030)
Figure 39: Market Size of Alagille Syndrome in the UK, USD Millions (2017–2030)
Figure 40: Market Size of Alagille Syndrome in the UK by therapies, USD Millions (2017–2030)
Figure 41: Market Size of Alagille Syndrome in Japan, USD Millions (2017–2030)
Figure 42: Market Size of Alagille Syndrome in Japan by therapies, USD Millions (2017–2030)
Figure 43: Market Drivers
Figure 44: Market Barriers
Mirum Pharmaceuticals
Albireo
And Many Others
