Adrenoleukodystrophy Market

Market Overview:

The 7 major adrenoleukodystrophy markets reached a value of US$ 239.3 Million in 2023. Looking forward, IMARC Group expects the 7MM to reach US$ 290.2 Million by 2034, exhibiting a growth rate (CAGR) of 1.77% during 2024-2034.

The adrenoleukodystrophy market has been comprehensively analyzed in IMARC's new report titled "Adrenoleukodystrophy Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Adrenoleukodystrophy (ALD) is a rare and hereditary disorder that affects the adrenal glands and nervous system. It falls under the category of leukodystrophies, a group of genetic disorders impacting the myelin sheath, the protective layer of nerve fibers in the brain. The symptoms of ALD can manifest in various ways, depending on the type and severity of the disease. Common initial signs include behavioral changes, difficulty concentrating, and deteriorating school performance. As the disease progresses, individuals suffering from the disorder may experience muscle weakness, visual disturbances, impaired coordination, and even severe cognitive and motor problems. In some cases, ALD can also lead to adrenal insufficiency, resulting in hormonal imbalances. Diagnosing the ailment often involves a combination of clinical evaluation, neuroimaging studies, such as MRI, and specialized biochemical tests to assess the levels of certain fatty acids in the blood. Physicians might also perform genetic testing to confirm the presence of mutations associated with ALD.

The escalating cases of genetic variations within the ABCD1 gene, culminating in the disruption of peroxisomal functions and the subsequent accumulation of very long-chain fatty acids (VLCFA), are primarily driving the adrenoleukodystrophy market. In addition to this, the inflating utilization of combination treatments, including glyceryl trioleate and glyceryl trierucate, which aim to reduce the buildup of VLCFAs and mitigate neurological damage in patients, is also creating a positive outlook for the market. Moreover, the widespread adoption of supportive care strategies, such as physical and occupational therapies tailored to address mobility challenges and enhance daily functioning, is further bolstering the market growth. These therapies aid in fortifying muscle strength, refining coordination, and augmenting gross motor skills, thus increasing the overall standard of living for individuals suffering from ALD. Additionally, the rising usage of potent medications, like corticosteroids, to manage adrenal gland dysfunctions and alleviate the progression of the disease is acting as another significant growth-inducing factor. Apart from this, the emerging popularity of gene therapy for the treatment of the illness by introducing functional genetic material to rectify or replace the defective ABCD1 gene responsible for the disorder is expected to drive the adrenoleukodystrophy market during the forecast period.

IMARC Group's new report provides an exhaustive analysis of the adrenoleukodystrophy market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report, the United States has the largest patient pool for adrenoleukodystrophy and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the adrenoleukodystrophy market in any manner.

Recent Developments:

• In May 2023, Minoryx Therapeutics reported that the US Food and Drug Administration (FDA) had approved its Phase 3 clinical trial (CALYX) of lead candidate leriglitazone to treat adult males with X-linked adrenoleukodystrophy (X-ALD) patients with cerebral adrenoleukodystrophy.

Key Highlights:

• Adrenoleukodystrophy incidence is higher in patients of Latino or African origin.
• X-linked adrenoleukodystrophy is the most common peroxisomal condition, with an estimated birth frequency of 1 in 17,000 (male and female).
• In the United States, one in every 21,000 men is thought to have adrenoleukodystrophy, whereas one in every 16,800 women is expected to be a carrier.
• Men are more likely to develop adrenoleukodystrophy.
• Most cases are identified in childhood; however, a considerable proportion of cases appear in young adults (usually in their late 20s).

Drugs:

SKYSONA is an autologous hematopoietic stem cell-based gene therapy designed to reduce the course of neurologic impairment in boys aged 4 to 17 who have early, active cerebral adrenoleukodystrophy (CALD).

Leriglitazone (MIN-102) is an orally bioavailable and selective PPARγ agonist with a promising profile for CNS disorders. It has shown brain penetration and a positive safety profile. It demonstrated strong preclinical proof-of-concept in animal models of several disorders by altering pathways that lead to mitochondrial failure, oxidative stress, neuroinflammation, demyelination, and axonal degeneration.

PXL065 is the deuterium-stabilized (R)-enantiomer of pioglitazone, which lacks PPARγ agonist action and related side effects such as weight gain and edema. PXL065 (formerly DRX-065) has shown preclinical efficacy for X-linked adrenoleukodystrophy.

Time Period of the Study

• Base Year: 2023
• Historical Period: 2018-2023
• Market Forecast: 2024-2034

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan
   

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the adrenoleukodystrophy market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the adrenoleukodystrophy market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current adrenoleukodystrophy marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the adrenoleukodystrophy market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
• What was the country-wise size of the adrenoleukodystrophy market across the seven major markets in 2023 and what will it look like in 2034?
• What is the growth rate of the adrenoleukodystrophy market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?
   

Epidemiology Insights

• What is the number of prevalent cases (​2018-2034​) of adrenoleukodystrophy across the seven major markets?
• What is the number of prevalent cases (​2018-2034​) of adrenoleukodystrophy by age across the seven major markets?
• What is the number of prevalent cases (​2018-2034​) of adrenoleukodystrophy by gender across the seven major markets?
• How many patients are diagnosed (​2018-2034​) with adrenoleukodystrophy across the seven major markets?
• What is the size of the adrenoleukodystrophy patient pool (2018-2023) across the seven major markets?
• What would be the forecasted patient pool (2024-2034) across the seven major markets?
• What are the key factors driving the epidemiological trend of adrenoleukodystrophy?
• What will be the growth rate of patients across the seven major markets?
   

Adrenoleukodystrophy: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for adrenoleukodystrophy drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the adrenoleukodystrophy market?
• What are the key regulatory events related to the adrenoleukodystrophy market?
• What is the structure of clinical trial landscape by status related to the adrenoleukodystrophy market?
• What is the structure of clinical trial landscape by phase related to the adrenoleukodystrophy market?
• What is the structure of clinical trial landscape by route of administration related to the adrenoleukodystrophy market?