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Adrenoleukodystrophy - Market Insight, Epidemiology, and Market Forecast - 2034
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Key Highlights:

DelveInsight's "Adrenoleukodystrophy Market Insights, Epidemiology and Market Forecast - 2034" report delivers an in-depth understanding of adrenoleukodystrophy, historical and forecasted epidemiology as well as the adrenoleukodystrophy market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.

Adrenoleukodystrophy market report provides real-world prescription pattern analysis, emerging drugs, market share of individual therapies, and historical and forecasted 7MM adrenoleukodystrophy market size from 2020 to 2034. The report also covers current adrenoleukodystrophy treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's underlying potential.

Geography Covered:

Study Period: 2020-2034

Adrenoleukodystrophy Treatment Market

Adrenoleukodystrophy Overview, Country-Specific Treatment Guidelines and Diagnosis

ALD is caused by mutations in the ABCD1 gene that prevent the body from breaking down very-long-chain fatty acids (VLCFAs). As a result, VLCFAs build up in the brain, nervous system, and adrenal glands. It is proposed that the presence of VLCFA in myelin induces myelin instability, which results in an immune-mediated process in which presentation of a lipid antigen may result in substantial myelin destruction. It is a progressive disorder of young males associated with elevated levels of very-long-chain fatty acids, due to defective B-oxidation and ABCD1 gene mutations encoding peroxisomal membrane protein X-ALD protein.

Diagnosing adrenoleukodystrophy (ALD) involves initially measuring VLCFA levels through a blood test. Elevated VLCFA levels suggest a potential ALD diagnosis. To confirm the diagnosis, a genetic test is typically ordered to identify mutations in the ABCD1 gene, which is associated with ALD. Other diagnostic tests for adrenoleukodystrophy (ALD) may include brain imaging (MRI), adrenal function tests, neurological evaluation, hormone testing, lipid panel, and sensory assessments to aid in diagnosis and assessment of disease progression.

The adrenoleukodystrophy market report provides an overview of adrenoleukodystrophy pathophysiology, diagnostic approaches, and detailed treatment algorithm along with a real-world scenario of a patient's journey beginning from the first symptom, the time taken for diagnosis to the entire treatment process.

Adrenoleukodystrophy Treatment

General supportive care and symptomatic treatment for patient and family, provided by pediatrician or neurologist, with appropriate specialist consultation, nursing, schools, rehabilitation, and social agencies, are the cornerstones for the care and treatment of patients with X-ALD. It is primarily managed by hormone replacement therapy, dietary therapy with Lorenzo's oil, and hematopoietic stem cell transplantation.

SKYSONA (elivaldogene autotemcel) became the first FDA approved therapy shown to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD).

Adrenoleukodystrophy Epidemiology

The Adrenoleukodystrophy epidemiology chapter in the report provides historical as well as forecasted in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan from 2024 to 2034. The adrenoleukodystrophy epidemiology is segmented with detailed insights into Total Prevalent Cases of Adrenoleukodystrophy (ALD), Total Diagnosed Prevalent Cases of Adrenoleukodystrophy (ALD), Gender-specific Diagnosed Prevalence of ALD, and Type-specific Diagnosed Prevalence of ALD.

Adrenoleukodystrophy Drug Chapters

The drug chapter segment of the adrenoleukodystrophy market report encloses a detailed analysis of adrenoleukodystrophy marketed drugs and late-stage (Phase III and Phase II) pipeline drugs. It also deep dives into the adrenoleukodystrophy clinical trial details, recent and expected market approvals, patent details, the latest news, and recent deals and collaborations.

Marketed Adrenoleukodystrophy Drugs

SKYSONA (elivaldogene autotemcel): Bluebird Bio

SKYSONA is an autologous hematopoietic stem cell-based gene therapy. In September 2022, the US FDA granted accelerated approval of SKYSONA (elivaldogene autotemcel), also known as eli-cel, to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD). The approval of SKYSONA was based on data from Bluebird Bio's Phase II/III study ALD-102 (Starbeam) (N=32) and Phase III ALD-104 (N=35) study. This indication is approved under accelerated approval based on 24-month major functional disability (MFD) free survival.\

Emerging Adrenoleukodystrophy Drugs

Leriglitazone (MIN-102): Minoryx Therapeutics/Neuraxpharm

Leriglitazone is Minoryx Therapeutics's novel orally bioavailable and selective PPAR gamma agonist with a potential first-in-class and best-in-class profile for CNS diseases. It has demonstrated brain penetration and a favorable safety profile. Leriglitazone showed clinical benefit in both adult X-ALD patients in ADVANCE and pediatric X-ALD patients in NEXUS trial. Data from ADVANCE showed that leriglitazone reduces the progression of lesions and the development of progressive cALD. Leriglitazone has been granted orphan drug status for X-ALD from the FDA and the EMA and Fast Track and Rare Pediatric Disease designation from the FDA for the treatment of X-ALD.

Minoryx Therapeutics and Neuraxpharm are seeking re-examination by the EMA's CHMP for conditional approval of leriglitazone as a treatment for cerebral adrenoleukodystrophy (cALD) after the initial marketing authorization recommendation was not granted.

PXL770: Poxel

PXL770 is a first-in-class direct AMPK activator. Clinical Phase I and IIa development has demonstrated target engagement and translation of several metabolic efficacy parameters to humans which suggests the likelihood of broader translation for this mechanism. In early 2022, the US FDA granted Fast Track and Orphan Drug Designation to PXL770 for the treatment of patients with adrenomyeloneuropathy (AMN).

A Phase IIa clinical POC biomarker studies of PXL770 is planned to initiate as soon as possible, subject to financing. The initial focus will be on ALD patients with adrenomyeloneuropathy (AMN), the largest subtype of ALD.

Adrenoleukodystrophy Market Outlook

Adrenoleukodystrophy Companies, such as Bluebird Bio, Minoryx Therapeutics/Neuraxpharm, Poxel, and others are evaluating their lead candidates in different stages of clinical development. They aim to investigate their products for the treatment of adrenoleukodystrophy.

The United States dominates the Adrenoleukodystrophy market, constituting about 40% of the market share, surpassing the market presence of the EU4 (Germany, Spain, Italy, France), the United Kingdom, and Japan.

Leriglitazone (MIN-102) is expected to capture a significant market share during the forecast period.

The Adrenoleukodystrophy market growth is driven by a growing pipeline of potential therapies, heightened awareness of newborn screening initiatives, and a rising incidence of cerebral adrenoleukodystrophy (CALD) cases.

Adrenoleukodystrophy Drugs Uptake

This section focuses on the uptake rate of potential Adrenoleukodystrophy drugs expected to be launched in the market during 2024-2034, which depends on the competitive landscape, safety, and efficacy data along with order of entry. It is important to understand that the key players evaluating their novel therapies in the pivotal and confirmatory trials should remain vigilant when selecting appropriate comparators to stand the greatest chance of a positive opinion from regulatory bodies, leading to approval, smooth launch, and rapid uptake.

Adrenoleukodystrophy Activities

The Adrenoleukodystrophy market report provides insights into different therapeutic candidates in Phase III and Phase II stages. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities

The Adrenoleukodystrophy market report covers information on collaborations, acquisitions and mergers, licensing, and patent details for adrenoleukodystrophy emerging therapies.

KOL Views

To keep up with the real-world scenario in current and emerging Adrenoleukodystrophy market trends, we take opinions from Key Industry leaders working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts were contacted for insights on the evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake along with challenges related to accessibility.

DelveInsight's analysts connected with 10+ KOLs to gather insights; however, interviews were conducted with 5+ KOLs in the 7MM. Centers such as Albert Einstein College of Medicine, Penn State Hershey Medical Center, University Hospital Heidelberg, University of Minnesota, etc., were contacted. Their opinion helps understand and validate current and emerging treatment patterns of adrenoleukodystrophy. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of gaps in disease diagnosis, patient awareness, physician acceptability, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided.

Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

In efficacy, the trial's primary and secondary outcome measures are evaluated; one of the most important primary outcome measures is time to death and requirement for permanent ventilator support.

Further, the therapies' safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

Adrenoleukodystrophy Market Access and Reimbursement

Reimbursement of rare disease therapies can be limited due to lack of supporting policies and funding, challenges of high prices, lack of specific approaches to evaluating rare disease drugs given limited evidence, and payers' concerns about budget impact. The high cost of rare disease drugs usually has a limited effect on the budget due to the small number of eligible patients being prescribed the drug. The US FDA has approved several rare disease therapies in recent years. From a patient perspective, health insurance and payer coverage guidelines surrounding rare disease treatments restrict broad access to these treatments, leaving only a small number of patients who can bypass insurance and pay for products independently.

The Adrenoleukodystrophy market report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of currently used therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Scope of the Adrenoleukodystrophy Market Report

Adrenoleukodystrophy Market Report Insights

Adrenoleukodystrophy Market Report Key Strengths

Adrenoleukodystrophy Market Report Assessment

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Table of Contents

1. Key Insights

2. Executive Summary of Adrenoleukodystrophy (ALD)

3. Competitive Intelligence Analysis for Adrenoleukodystrophy (ALD)

4. Adrenoleukodystrophy (ALD) Market Overview at a Glance

5. Adrenoleukodystrophy (ALD): Disease Background and Overview

6. Adrenoleukodystrophy Patient Journey

7. Adrenoleukodystrophy (ALD) Epidemiology and Patient Population

8. Adrenoleukodystrophy Treatment Algorithm, Current Treatment, and Medical Practices

9. Adrenoleukodystrophy (ALD) Unmet Needs

10. Key Endpoints of Adrenoleukodystrophy (ALD) Treatment

11. Adrenoleukodystrophy (ALD) Marketed Products

12. Adrenoleukodystrophy (ALD) Emerging Therapies

13. Adrenoleukodystrophy (ALD): Seven Major Market Analysis

14. Attribute analysis

15. 7MM: Adrenoleukodystrophy Market Outlook

16. Access and Reimbursement Overview of Adrenoleukodystrophy (ALD)

17. KOL Views

18. Adrenoleukodystrophy Market Drivers

19. Adrenoleukodystrophy Market Barriers

20. Appendix

21. DelveInsight Capabilities

22. Disclaimer

23. About DelveInsight

The table of contents is not exhaustive; the final content may vary.

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