Cerebral Adrenoleukodystrophy (cALD) Market Forecasts by DelveInsight Signal Groundbreaking Growth by 2032 | bluebird bio, Minoryx Therapeutics, Orpheris, POXEL SA

The Cerebral Adrenoleukodystrophy (cALD) Market is undergoing a significant transformation with advancements in gene therapy and stem cell transplantation offering promising treatment avenues. Research efforts are focused on developing targeted therapies to address the underlying genetic mutations responsible for cALD progression. Additionally, collaborations between academia, and pharmaceutical companies are driving awareness and accelerating the development of novel interventions, ushering in a new era of hope for patients and families affected by cALD.

DelveInsight's "Cerebral Adrenoleukodystrophy (cALD) Market Insights, Epidemiology, and Market Forecast 2032" report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Cerebral Adrenoleukodystrophy Market Size and Share in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).

The market report covers emerging drugs, treatment practices, market share of individual Cerebral Adrenoleukodystrophy therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Cerebral Adrenoleukodystrophy treatment practice/algorithm, market drivers & barriers, and unmet medical needs to curate the best of opportunities and assess the underlying potential of the market.

Cerebral Adrenoleukodystrophy (cALD): An Overview
Cerebral Adrenoleukodystrophy (cALD) is an X-chromosome-linked, recessively inherited genetic disease that affects the brain. It is the childhood-onset form of Adrenoleukodystrophy (ALD) and the most severe one.

Cerebral ALD is caused by a mutation in the ABCD1 gene. These mutations of ABCD1 lead to the accumulation of very long-chain fatty acids (VLCFA) in the brain and the adrenal gland, resulting in demyelination of nerve fibers that impair information transmission from the brain to the rest of the body. Untreated, CALD progresses rapidly and can cause permanent cognitive and motor disabilities and death.

CALD typically presents in early childhood. Boys start showing symptoms between 4 and 10 years of age. More than half of the patients begin showing symptoms such as behavioral problems, psychological instability, poor memory, and among kids, difficulty in reading, writing, and comprehension then progress to different types of disabilities, such as progressive psychomotor disorder, diminution in vision and audition, and adrenocortical dysfunction.

Currently, there is no way to predict which boys with ALD will develop CALD. Fortunately, however, the early stages of CALD can be detected by brain MRI in boys with known ALD, even when they don't have any noticeable symptoms, Current guidelines recommend that all boys diagnosed with ALD are monitored regularly with MRIs. Newborn screening for ALD is vital to enable early diagnosis. It detects elevated VLCFA levels in the blood, a clear indicator of ALD. With newborn screening, boys at risk for CALD can be identified and treated early.

As there is no cure for CALD. The available treatment options aim to stop the progress of the disease and to provide supportive care to the patients, the current treatment options include therapies like Gene therapy and stem cell replacement therapy. other off-label drugs are also being used such as oral corticoids which aim to provide relief from pain and inflammations.

Cerebral Adrenoleukodystrophy (cALD) Market Key Facts
• In 2022, the total prevalent population of ALD in the 7MM was 56,000.
• As per DelveInsight's analysis, based on the type of ALD, nearly 50% of the affected population suffer from Cerebral ALD.
• Out of the total diagnosed cases in the US, CALD cases were further divided based on age, Childhood CALD (1,000 cases), Adolescent CALD (200 cases), and Adult CALD (100 cases) in 2022.
• As per DelveInsight's analysis, a higher percentage of ALD prevalence was observed for females, in comparison to males, in the United States during the study period (2019-2032). However, the diagnosed prevalent cases of ALD were estimated to be ~2,700 cases and ~2,500 cases for males and females, respectively, in 2022.
• According to the U.S. National Library of Medicine, the prevalence of ALD is 1 in 20,000 to 50,000 individuals worldwide. In the U.S., 1 in every 21,000 men is thought to be affected by ALD while 1 in 16,800 women are thought to be ALD carriers.
• According to the National Organization for Rare Disorders (NORD), the prevalence of ALD is estimated to be between 1 in 10,000 and 1 in 17,000 individuals in the general population.
• According to Raymond et al. (2019), CALD develops in approximately 40% of affected boys and in a smaller number of adult men.

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Cerebral Adrenoleukodystrophy (cALD) Market
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted market size by analyzing the impact of current and emerging Cerebral Adrenoleukodystrophy therapies in the market. It also provides a detailed assessment of the Cerebral Adrenoleukodystrophy market drivers & barriers, unmet needs, and emerging technologies.

The report gives complete details of the market trend for each marketed Cerebral Adrenoleukodystrophy drug and late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action (MOA), competition with other therapies, brand value, their impact on the market and view of the key opinion leaders.

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Cerebral Adrenoleukodystrophy (cALD) Epidemiology
The epidemiology section covers detailed insights into the historical, and current Cerebral Adrenoleukodystrophy patient pool and forecasted epidemiology trends for every seven major countries (7MM) from 2019 to 2032.

The Report Covers the Cerebral Adrenoleukodystrophy Epidemiology Segmented as -
• Gender-specific Prevalent Cases of ALD in the 7MM [2019-2032]
• Diagnosed Prevalent Cases of ALD in Males in the 7MM [2019-2032]
• Diagnosed Prevalent Cases of CALD in the 7MM [2019-2032]
• Age-specific Prevalent Cases of CALD in the 7MM [2019-2032]
• Total Treated Cases of CALD in the 7MM [2019-2032]

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Cerebral Adrenoleukodystrophy (cALD) Drugs Uptake and Pipeline Development Activities
The drugs' uptake section focuses on the rate of uptake of the potential drugs recently launched in the Cerebral Adrenoleukodystrophy market or expected to be launched during the study period. The analysis covers the market share by Cerebral Adrenoleukodystrophy drugs, patient uptake by therapies, and sales of each drug.

The report's drugs uptake section helps in understanding the drugs with the most rapid uptake and the reasons behind the maximal use of new drugs. It allows the comparison of the drugs based on market share and size, which again will be helpful in investigating factors important in understanding the market dynamics and in making financial and regulatory decisions.

The report also covers the Cerebral Adrenoleukodystrophy Pipeline Development Activities and provides valuable insights about different therapeutic candidates in Phase II and Phase III stages and the key companies in the market involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

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Cerebral Adrenoleukodystrophy (cALD) Therapeutics Assessment
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. Currently, there are no approved therapies for the treatment of CALD, and it is primarily managed by hormone replacement therapy, dietary therapy with Lorenzo's oil, and hematopoietic stem cell transplantation.

The accumulation of VLCFA in the adrenal gland is believed to cause apoptosis and the ultimate atrophy of the adrenal cortex. The accumulated VLCFA results in the esterification of cholesterol that may further impair cortisol secretion due to a relative shortage of substrate for steroid genesis (pathway of steroid hormones synthesis). The impaired adrenal gland function leads to the deficiency of steroid hormones (mainly cortisol and aldosterone), which is known as adrenal insufficiency.

Several major pharma and biotech companies are developing therapies for Cerebral Adrenoleukodystrophy. The companies which have their Cerebral Adrenoleukodystrophy drug candidates in the most advanced stage, i.e. phase II/III include Minoryx Therapeutics.

In September 2022, 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). The approval of SKYSONA was based on data from Bluebird Bio's Phase II/II 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. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Leading Companies in the Cerebral Adrenoleukodystrophy (cALD) Therapeutics Market Include
• bluebird bio, Inc.
• Minoryx Therapeutics
• Orpheris, Inc.
• POXEL SA
And Many Others

Emerging and Marketed Cerebral Adrenoleukodystrophy (cALD) Therapies Covered in the Report Include
• MIN-102: Minoryx Therapeutics
• PXL770: POXEL SA
And Many More

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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Cerebral Adrenoleukodystrophy Competitive Intelligence Analysis
4. Cerebral Adrenoleukodystrophy Market Overview at a Glance
5. Cerebral Adrenoleukodystrophy Background and Overview
6. Cerebral Adrenoleukodystrophy Patient Journey
7. Cerebral Adrenoleukodystrophy Epidemiology and Patient Population
8. Cerebral Adrenoleukodystrophy Treatment Algorithm, Current Treatment, and Medical Practices
9. Cerebral Adrenoleukodystrophy Unmet Needs
10. Key Endpoints of Cerebral Adrenoleukodystrophy Treatment
11. Cerebral Adrenoleukodystrophy Marketed Products
12. Cerebral Adrenoleukodystrophy Emerging Therapies
13. Cerebral Adrenoleukodystrophy Seven Major Market Analysis
14. Attribute Analysis
15. Cerebral Adrenoleukodystrophy Market Outlook (7 major markets)
16. Cerebral Adrenoleukodystrophy Access and Reimbursement Overview
17. KOL Views on the Cerebral Adrenoleukodystrophy Market.
18. Cerebral Adrenoleukodystrophy Market Drivers
19. Cerebral Adrenoleukodystrophy Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
*The table of contents is not exhaustive; the final content may vary.

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