Adrenoleukodystrophy Market to Rise by 2034 | bluebird bio, Minoryx Therapeutics, S.L., MedDay Pharmaceuticals, Magenta Therapeutics, Orpheris, SOM Biotech expected to boost market

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, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.

The Adrenoleukodystrophy market report provides current treatment practices, emerging drugs, the market share of the individual therapies, and the current and forecasted Adrenoleukodystrophy market size from 2020 to 2034, segmented by seven major markets. The Report also covers current Adrenoleukodystrophy treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying potential of the Adrenoleukodystrophy market.

Key highlights from the adrenoleukodystrophy market report:

In 2023, the United States held the largest share of the Adrenoleukodystrophy (ALD) market, estimated at approximately USD 400 million, surpassing the combined markets of the EU4 (Germany, Spain, Italy, France), the United Kingdom, and Japan.
ALD is an X-linked genetic disorder caused by a defect in the peroxisomal beta-oxidation of very long-chain fatty acids (VLCFAs). It is classified into three main types based on age of onset and symptom severity: childhood cerebral adrenoleukodystrophy (CALD), adrenomyeloneuropathy (AMN), and Addison's disease.
According to DelveInsight, CALD accounted for about 50% of diagnosed ALD cases in the United States in 2023. This rare disorder predominantly affects males, particularly during childhood, with around 60% of ALD cases diagnosed in males within the seven major markets (7MM).
Currently, there is no cure for ALD. One effective treatment for cerebral ALD is stem cell transplantation, where patients receive blood stem cells from a genetically matched donor. Hematopoietic stem cell therapy is beneficial for X-ALD when initiated early; however, treatment options for X-AMN remain limited.
The ALD market in the 7MM is expected to grow, driven by increased awareness of newborn screening programs, a rising number of cerebral adrenoleukodystrophy (CALD) cases, and anticipated approvals of new therapies from 2024 to 2034.
In September 2022, Bluebird Bio received accelerated approval from the US FDA for SKYSONA, a gene therapy for early, active CALD. Several emerging therapies, including Leriglitazone (MIN-102) and PXL770, are in development to address unmet medical needs in the market. However, in January 2024, Minoryx Therapeutics announced that the EMA's Committee for Medicinal Products for Human Use (CHMP) recommended not granting marketing authorization for Nezglyal (leriglitazone) as a treatment for X-ALD.

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Adrenoleukodystrophy Overview

Adrenoleukodystrophy (ALD) is a rare genetic disorder that primarily affects the nervous system and adrenal glands. It is characterized by the accumulation of very long-chain fatty acids (VLCFAs) due to a mutation in the ABCD1 gene, which impairs the ability of cells to break down these fatty acids. This accumulation leads to damage to myelin, the protective sheath surrounding nerve fibers, and affects adrenal function.

Causes

ALD is caused by mutations in the ABCD1 gene located on the X chromosome. This gene is responsible for producing a protein that helps transport VLCFAs into peroxisomes, where they are metabolized. The disorder is inherited in an X-linked recessive manner, meaning it predominantly affects males, while females may be carriers and experience milder symptoms.

Signs and Symptoms

Symptoms of ALD can vary widely, depending on the age of onset and severity. They typically fall into three main categories:

1. Cerebral ALD (most severe form, usually in childhood):
- Behavioral changes (e.g., hyperactivity, aggressiveness)
- Learning difficulties or cognitive decline
- Vision problems or blindness
- Seizures
- Motor skill deterioration (e.g., difficulty walking)

2. Adrenal Insufficiency:
- Fatigue
- Weight loss
- Low blood pressure
- Hyperpigmentation (darkening of the skin)
- Salt cravings

3. Adrenomyeloneuropathy (AMN) (later-onset form, typically in adulthood):
- Gradual loss of mobility
- Weakness or stiffness in the legs
- Numbness or tingling in the limbs
- Bladder and bowel dysfunction

Diagnosis

1. Clinical Evaluation: A thorough medical history and physical examination focusing on symptoms.

2. Blood Tests:
- Measurement of VLCFA levels in the blood; elevated levels indicate ALD.
- Hormonal testing to assess adrenal function.

3. Genetic Testing: To confirm mutations in the ABCD1 gene.

4. MRI: Brain imaging to identify changes in white matter, especially in the cerebral form of ALD.

Treatment Options

1. Hormone Replacement Therapy: For patients with adrenal insufficiency, glucocorticoids (e.g., hydrocortisone) may be prescribed to manage hormone levels.

2. Dietary Management: A low VLCFA diet can help reduce the levels of harmful fatty acids in the body.

3. Lorenzo's Oil: A mixture of specific fatty acids (oleic acid and erucic acid) that may help lower VLCFA levels, although its effectiveness can vary.

4. Stem Cell Transplant: This treatment can be effective, particularly for cerebral ALD if performed early in the disease. It involves transplanting healthy stem cells to replace damaged ones.

5. Gene Therapy: Research is ongoing into gene therapy approaches that may correct the underlying genetic defect.

6. Supportive Care: Physical therapy, occupational therapy, and psychological support to help manage symptoms and improve quality of life.

Prognosis

The prognosis for individuals with ALD varies widely based on the age of onset and the severity of symptoms. Early diagnosis and intervention can significantly improve outcomes, particularly in the cerebral form of the disease. Regular follow-up with healthcare providers is essential for managing symptoms and complications.

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Adrenoleukodystrophy Market

The Adrenoleukodystrophy market outlook of the report helps to build a detailed comprehension of the historical, current, and forecasted Adrenoleukodystrophy market trends by analyzing the impact of current Adrenoleukodystrophy therapies on the market and unmet needs, and drivers, barriers, and demand for better technology.

This segment gives a thorough detail of the Adrenoleukodystrophy market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, 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 Adrenoleukodystrophy market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

According to DelveInsight, the Adrenoleukodystrophy market in 7MM is expected to witness a major change in the study period 2020-2034.

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Adrenoleukodystrophy Epidemiology

The findings indicate that adrenoleukodystrophy (ALD) is more prevalent in males than in females across the seven major markets (7MM). According to DelveInsight's estimates, more than 30% of the diagnosed cases of ALD in 2023 were in the United States.

Among the EU4 countries and the UK, Germany had the highest prevalence of ALD, accounting for approximately 25% of cases, followed by France and the UK. DelveInsight's consultants project that cerebral ALD will represent the majority of cases in the United States in 2023.

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Adrenoleukodystrophy Drugs Uptake

This section focuses on the uptake rate of the potential Adrenoleukodystrophy drugs recently launched in the Adrenoleukodystrophy market or expected to be launched in 2020-2034. The analysis covers the Adrenoleukodystrophy market uptake by drugs, patient uptake by therapies, and sales of each drug.

Adrenoleukodystrophy Drugs Uptake helps in understanding the drugs with the most rapid uptake and the reasons behind the maximal use of new drugs and allows the comparison of the drugs based on Adrenoleukodystrophy market share and size, which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

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Adrenoleukodystrophy Therapeutics Assessment

Major pharma companies such as bluebird bio, Minoryx Therapeutics, S.L., MedDay Pharmaceuticals, Magenta Therapeutics, Orpheris Inc., SOM Biotech, and others are working proactively in the Adrenoleukodystrophy Therapeutics market to develop novel therapies which will drive the Adrenoleukodystrophy treatment markets in the upcoming years.

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Adrenoleukodystrophy Report Key Insights

1. Adrenoleukodystrophy Patient Population
2. Adrenoleukodystrophy Market Size and Trends
3. Key Cross Competition in the Adrenoleukodystrophy Market
4. Adrenoleukodystrophy Market Dynamics (Key Drivers and Barriers)
5. Adrenoleukodystrophy Market Opportunities
6. Adrenoleukodystrophy Therapeutic Approaches
7. Adrenoleukodystrophy Pipeline Analysis
8. Adrenoleukodystrophy Current Treatment Practices/Algorithm
9. Impact of Emerging Therapies on the Adrenoleukodystrophy Market

Table of Contents

1. Key Insights
2. Executive Summary
3. Adrenoleukodystrophy Competitive Intelligence Analysis
4. Adrenoleukodystrophy Market Overview at a Glance
5. Adrenoleukodystrophy Disease Background and Overview
6. Adrenoleukodystrophy Patient Journey
7. Adrenoleukodystrophy Epidemiology and Patient Population
8. Adrenoleukodystrophy Treatment Algorithm, Current Treatment, and Medical Practices
9. Adrenoleukodystrophy Unmet Needs
10. Key Endpoints of Adrenoleukodystrophy Treatment
11. Adrenoleukodystrophy Marketed Products
12. Adrenoleukodystrophy Emerging Therapies
13. Adrenoleukodystrophy Seven Major Market Analysis
14. Attribute Analysis
15. Adrenoleukodystrophy Market Outlook (7 major markets)
16. Adrenoleukodystrophy Access and Reimbursement Overview
17. KOL Views on the Adrenoleukodystrophy Market
18. Adrenoleukodystrophy Market Drivers
19. Adrenoleukodystrophy Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer

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