Adrenoleukodystrophy (ALD) is a rare genetic condition that causes the buildup of very long chain fatty acids (VLCFAs) in the brain. When VLCFAs accumulate, they destroy the protective myelin sheath around nerve cells, responsible for brain function. Without the myelin sheath, the nerves can no longer relay information to and from the brain.
The defective gene in ALD, commonly referred to as a genetic mutation, can cause several different but related conditions: adrenomyelopathy (AMN), Addison’s disease and — the most common and most devastating form — cerebral ALD. Cerebral ALD strikes boys between ages 4 and 10, leading to permanent disability and death usually within four to eight years. There are three distinct types of X-linked adrenoleukodystrophy: a childhood cerebral form, an adrenomyeloneuropathy type, and a form called Addison disease only.
DelveInsight’s “Adrenoleukodystrophy – Market Insights, Epidemiology, and Market Forecast-2030“ report delivers an in-depth understanding of the Adrenoleukodystrophy , historical and forecasted epidemiology as well as the Adrenoleukodystrophy market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.
The Adrenoleukodystrophy market report provides current treatment practices, emerging drugs, Adrenoleukodystrophy market share of the individual therapies, current and forecasted Adrenoleukodystrophy market Size from 2017 to 2030 segmented by seven major markets. The Report also covers current Adrenoleukodystrophy treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses the underlying potential of the market.
- The United States
- EU5 (Germany, France, Italy, Spain, and the United Kingdom)
Study Period: 2017-2030
Adrenoleukodystrophy Disease Understanding and Treatment Algorithm
Adrenoleukodystrophy (ALD) is an X-linked recessive genetic disorder caused by the abnormality in the ABCD1 gene present on the X chromosome that leads to the accumulation of very-long-chain fatty acids (VLCFAs) in the brain, nervous system, and adrenal gland. The accumulation of VLCFAs leads to the deterioration of the myelin sheath, and without the sheath, neurons cannot conduct action potentials, this leads to the development of seizures and hyperactivity. Currently, no drug is approved for the treatment of Adrenoleukodystrophy and the available treatment options include hormone replacement therapy, dietary therapy with Lorenzo’s oil and Hematopoietic stem cell transplantation, using either umbilical cord or bone marrow stem cells.
Signs and Symptoms
Signs and symptoms of the adrenomyeloneuropathy type appear between early adulthood and middle age. Affected individuals develop progressive stiffness and weakness in their legs (paraparesis), experience urinary and genital tract disorders, and often show changes in behavior and thinking ability. Most people with the adrenomyeloneuropathy type also have adrenocortical insufficiency. In some severely affected individuals, damage to the brain and nervous system can lead to early death.
ALD is an X-linked recessive condition caused by a mutation in the ABCD1 gene on the X chromosome. Because a female has two X chromosomes, if she inherits the faulty gene, then she still has another X chromosome to offset the mutation. However, because males only have one X chromosome, the gene abnormality causes the disease. With each pregnancy, female ALD carriers have a 25 percent (1 in 4) chance of having a carrier daughter and a 25 (1 in 4) percent chance of having a son affected with the disease.
The Adrenoleukodystrophy epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by Total Prevalent Population of Adrenoleukodystrophy in the 7MM, Gender-specific Prevalence of Adrenoleukodystrophy in the 7MM, Gender-specific Diagnosed Prevalence of Adrenoleukodystrophy in the 7MM, and Type-specific Diagnosed Prevalence of ALD in Males in the 7MM. The epidemiology section covers the 7MM countries, the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from the year 2017 to 2030.
Adrenoleukodystrophy Drug Chapters
Lenti D: Bluebird Bio
Lenti D is an investigational gene therapy that is in Phase II/III stage of development for the treatment of cerebral ALD (CALD). Currently, bluebird bio is enrolling patients for a Phase III study (ALD-104) designed to assess the efficacy and safety of Lenti-D after myeloablative conditioning using busulfan and fludarabine in patients with CALD. Additionally, the company is conducting a long-term safety and efficacy follow-up study (LTF-304) for patients who have participated in ALD-102 and were treated with Lenti-D.
Leriglitazone: Minoryx Therapeutics, S.L.
Leriglitazone (MIN-102) is a metabolite of pioglitazone which shows an excellent brain penetration and safety profile, allowing PPAR gamma engagement in the CNS above the level that can be safely achieved with pioglitazone and other glitazones. The drug candidate is in Phase II/III stage of development for the treatment of both cerebral ALD and Adrenomyeloneuropathy (AMN).
Products detail in the report…
Adrenoleukodystrophy Market Outlook
The Adrenoleukodystrophy market outlook of the report helps to build a detailed comprehension of the historic, current and forecasted Adrenoleukodystrophy market trends by analyzing the impact of current and emerging therapies on the market, unmet needs, drivers and barriers and demand of better technology.
This segment gives a thorough detail of Adrenoleukodystrophy market trend of each current available and late-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, Adrenoleukodystrophy market in 7MM is expected to change in the study period 2017–2030.
Scope of the Report
- The report covers the descriptive overview of Adrenoleukodystrophy , explaining its causes, signs and symptoms, pathophysiology, diagnosis and currently available therapies
- Comprehensive insight has been provided into the Adrenoleukodystrophy epidemiology and treatment in the 7MM
- Additionally, an all-inclusive account of both the current and emerging therapies for Adrenoleukodystrophy are provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape
- A detailed review of Adrenoleukodystrophy market; historical and forecasted is included in the report, covering drug outreach in the 7MM
- The report provides an edge while developing business strategies, by understanding trends shaping and driving the global Adrenoleukodystrophy market
Table of contents:
1. Key Insights
2. Adrenoleukodystrophy (ALD): Market Overview at a Glance
2.1. Total Market Share (%) Distribution of ALD in 2017
2.2. Total Market Share (%) Distribution of ALD in 2030
3. Adrenoleukodystrophy: Disease Background and Overview
3.2. Clinical aspects of ALD
3.3. Causes of ALD
3.4. Inheritance pattern of ALD
3.6. Pathogenesis and Pathophysiology of ALD
3.7.1. ALD Newborn Screening
3.7.2. Tools for evaluating Cerebral ALD
3.7.3. Diagnostic algorithm
3.7.4. HSCT and presymptomatic diagnosis of ALD in Japan
4. Epidemiology and Patient Population
4.1. Key Findings
4.2. Epidemiology Methodology
4.3. KOL Views
4.4. Total Prevalent Population of Adrenoleukodystrophy in the 7MM
5. United States Epidemiology
5.1. Assumptions and Rationale
5.2. Total Prevalent Population of Adrenoleukodystrophy (ALD) in the United States
5.3. Gender-specific Prevalence of Adrenoleukodystrophy in the United States
5.4. Gender-specific Diagnosed Prevalence of Adrenoleukodystrophy in the United States
5.5. Type-specific Diagnosed Prevalence of ALD in Males in the United States
6. EU5 Epidemiology
7. Japan Epidemiology
7.1. Assumptions and Rationale
7.2. Total Prevalent Population of Adrenoleukodystrophy in Japan
7.3. Gender-specific Prevalence of Adrenoleukodystrophy in Japan
7.4. Gender-specific Diagnosed Prevalence of Adrenoleukodystrophy in Japan
7.5. Type-specific Diagnosed Prevalence of ALD in Males in Japan
8. Current Treatment and Medical Practices
8.1. Subtype-specific Treatment
8.2. Treatment Guidelines
8.2.1. Recommendations for Treatment of X-linked ALD in the United States
8.2.2. Adrenoleukodystrophy treatment: European Leukodystrophies Association
8.3. Early intervention and long-term follow-up system for presymptomatic ALD patients in Japan
9. Unmet Needs
10. Emerging Therapies
11. Failed Therapy
11.1. NV1205: NeuroVia/Archer Daniels Midland
11.1.1. Drug Description
11.1.2. Other Development Activities
11.1.3. Clinical Development
11.1.4. Safety and Efficacy
11.1.5. Advantages and Disadvantages
11.1.6. Product Profile
12. Adrenoleukodystrophy (ALD): 7MM Market Analysis
12.1. Key Findings
12.2. Market Methodology
12.3. Market Size of Adrenoleukodystrophy in the 7MM
12.4. Market Size of Adrenoleukodystrophy by Therapies in the 7MM
13. 7MM: Market Outlook
14. United States
14.1. United States Market Size
14.1.1. Total Market size of Adrenoleukodystrophy in the United States
14.1.2. Market Size of Adrenoleukodystrophy by Therapies in the US
15. EU-5 countries
16.1. Japan Market Size
16.1.1. Total Market size of Adrenoleukodystrophy in Japan
16.1.2. Market Size of Adrenoleukodystrophy by therapies in Japan
17. Market Drivers
18. Market Barriers
19.2. Report Methodology
20. DelveInsight Capabilities
22. About DelveInsight
DelveInsight is a leading Business Consultant, and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing end to end comprehensive solutions to improve their performance.
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