Adrenoleukodystrophy Treatment Market Size, Share, Industry, Forecast and Outlook (2024-2031)

Global Adrenoleukodystrophy Treatment Market is segmented By Type (Childhood Cerebral ALD, Adrenomyeloneuropathy (AMN), Addison's disease), By Treatment (Stem Cell Transplant, Corticosteroid Replacement Therapy, Medications, Gene Therapy, Lorenzo's Oil, Others), By End-user (Hospitals, Specialty Clinics, Others), and By Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) – Share, Size, Outlook, and Opportunity Analysis, 2024-2031

Report Overview

The Global Adrenoleukodystrophy Treatment Market reached US$ 323.36 million in 2023 and is expected to reach US$ 673.03 million by 2031 growing with a CAGR of 9.6% during the forecast period 2024-2031.

Adrenoleukodystrophy is a disease caused by a mutation in the ABCD1 gene that is located on the X chromosome. It is a neurodegenerative disease in which the protective myelin sheath covering the nerve fibers in the brain and spinal cord gradually deteriorates. This demyelination causes injury to the brain leading to the behavioral and neurological symptoms seen in the patients. The disease also affects the adrenal glands causing them not to produce enough steroid hormones.

The most common type of adrenoleukodystrophy is X-linked ALD caused by a genetic defect on the X chromosome. It mostly affects males more severely than females. The global adrenoleukodystrophy treatment market has witnessed significant growth in recent years due to the adoption of stem cell transplantation treatments. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only therapeutic approach to arrest cerebral demyelination of X‐linked adrenoleukodystrophy.

Market Scope

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Market Dynamics: Drivers

Increasing Research & Developments and Rising FDA Approvals of Drugs for Adrenoleukodystrophy Treatment 

The demand for the global adrenoleukodystrophy treatment market is driven by multiple factors. One of the primary factors is the increasing research & developments and rising FDA approvals of drugs for the treatment.

Adrenoleukodystrophy (ALD) is a rare genetic disorder that affects the white matter of the nervous system and the adrenal cortex. According to the National Organization for Rare Disorders in September 2022, the prevalence of ALD is estimated to be between 1 in 10,000 and 1 in 17,000 individuals in the general population.

Moreover, government initiatives and advanced research and developments in treating adrenoleukodystrophy help to drive this market growth. For instance, in August 2023, Orpheris announced that the drug Acetylcysteine zidrimer is under clinical development in Phase II for X-linked adrenoleukodystrophy (X-ALD).

In addition, key players in the industry strategies such as partnerships & collaborations and product launches & approvals would drive this market growth. For instance, in November 2022, Minoryx Therapeutics and Neuraxpharm collaborated on concluding the European regulatory process to obtain approval for leriglitazone for the treatment of adult male patients with X-ALD. Leriglitazone is the first approved treatment approved by the European Medicines Agency (EMA.

In May 2022, POXEL SA  announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to PXL065 and PXL770 for the treatment of patients with adrenomyeloneuropathy (AMN), the most common form of X-linked adrenoleukodystrophy (ALD). 

Restraints

Factors such as the high cost of the treatment, side effects associated with stem cell transplants, a limited number of treatment options, and lack of awareness among people, are expected to hamper the market.

Market Segment Analysis

The global adrenoleukodystrophy treatment market is segmented based on type, treatment, end users, and region.

The childhood cerebral (cALD) segment accounted for approximately 38.5% of the global adrenoleukodystrophy treatment market share 

The childhood cerebral (cALD) segment is expected to hold the largest market share over the forecast period. Childhood cerebral ALD (cALD )is the most devastating form of ALD. X-ALD (X-linked adrenoleukodystrophy) is an orphan neurodegenerative disease. The global incidence of X-ALD is approximately 6-8/100,000 live births. ALD patients, boys, and adult men, at any point in their lifetime, can develop cALD, characterized by demyelinating brain lesions that rapidly progress, leading to acute neurological decline and death.

Moreover, key players in the industry on ongoing advanced research & developments and a rising number of clinical trials in the treatment of adrenoleukodystrophy would drive this segment growth. For instance, in April 2023, Minoryx Therapeutics, announces 24-week interim results from NEXUS. This open-label registration-enabling clinical trial is assessing the safety and efficacy of lead candidate leriglitazone, a novel, brain-penetrant and selective PPAR gamma agonist, to treat pediatric patients with cerebral adrenoleukodystrophy (cALD) – a phenotype of X-linked adrenoleukodystrophy (X-ALD). 

Similarly, in November 2023, Minoryx Therapeutics announced that the US Food and Drug Administration (FDA) approved its Phase 3 clinical trial (CALYX) of lead candidate leriglitazone to treat adult male X-linked Adrenoleukodystrophy (X-ALD) patients with cerebral Adrenoleukodystrophy (CALD). The CALYX protocol has received both FDA and central IRB approval.

Market Geographical Share

North America accounted for approximately 41.3% of the global adrenoleukodystrophy treatment market share 

North America region is expected to hold the largest market share over the forecast period owing to factors such as increasing research and developments, drug approvals, and technological advancements in adrenoleukodystrophy treatment.

Moreover, the rising prevalence of ALD in the U.S. propels this market growth. According to the U.S. National Library of Medicine, in October 2022, 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.  

As per a newborn screening conducted in the state of New York, the incidence of ALD at birth was estimated to be 1 in 15,000. Among the 700,000 newborns screened in three years, 45 babies were diagnosed with ALD.

In addition, major players in the industry strategies such as FDA approvals and research & development in the treatment of adrenoleukodystrophy would drive this market growth. For instance, in September 2022, Bluebird Bio received FDA accelerated approval for SKYSONA Gene Therapy for early, active cerebral adrenoleukodystrophy (CALD). This therapy slows down the progression of neurologic dysfunction in boys 4-17 years of age with early and active cerebral adrenoleukodystrophy (CALD).

Also,  in January 2022, SwanBio Therapeutics announced that its Investigational New Drug (IND) application for its lead candidate SBT101 for the treatment of adrenomyeloneuropathy (AMN) is cleared by the U.S. Food and Drug Administration (FDA). SBT101 is the first AAV-based gene therapy in development specifically designed for people living with adrenomyeloneuropathy. 

Market Segmentation

By Type

• Childhood Cerebral ALD
• Adrenomyeloneuropathy (AMN)
• Addison's Disease

By Treatment

• Stem Cell Transplant
• Corticosteroid Replacement Therapy
• Medications
• Gene Therapy
• Lorenzo's Oil
• Others

By End Users

• Hospitals
• Specialty Clinics
• Others

By Region

• North America
  • U.S.
  • Canada
  • Mexico
• Europe
  • Germany
  • U.K.
  • France
  • Italy
  • Spain
  • Rest of Europe
• South America
  • Brazil
  • Argentina
  • Rest of South America
• Asia-Pacific
  • China
  • India
  • Japan
  • South Korea
  • Rest of Asia-Pacific
• Middle East and Africa

Market Competitive Landscape

The major global players in the adrenoleukodystrophy treatment market include Bluebird Bio Inc., Viking Therapeutics, Magenta Therapeutics, NeuroVia, Minoryx Therapeutics, Poxel S.A., Orpheris, SwanBio Therapeutics, Sperogenix Therapeutics, and Autobahn Therapeutics among others.

Key Developments

• In May 2024, Minoryx Therapeutics announced that the EMA’s Committee for Medicinal Products for Human Use (CHMP) has recommended not to grant marketing authorization for NEZGLYAL (leriglitazone) as a treatment for cALD following the completion of the re-examination procedure.
• In February 2024, Neuraxpharm Group and Minoryx Therapeutics, a late-stage biotech company focused on the development of therapies for orphan central nervous system disorders, reaffirm their commitment on Rare Disease Day 2024 to find better treatments for patients suffering from a range of rare diseases, such as leukodystrophy, including X-linked adrenoleukodystrophy (X-ALD) and more specifically cerebral adrenoleukodystrophy (cALD).
• In June 2022, POXEL SA announced its participation at the ULF (United Leukodystrophy Foundation) Scientific Symposium and Family Conference on June at Illinois, USA by using PXL065, two proprietary promising candidates that are focused on the treatment of adrenoleukodystrophy (ALD) which is a rare and genetic disease in adults.

Why Purchase the Report?

• To visualize the global adrenoleukodystrophy treatment market segmentation based on the type, treatment, end users and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development. 
• Excel data sheet with numerous data points of global adrenoleukodystrophy treatment market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.

The global adrenoleukodystrophy treatment market report would provide approximately 62 tables, 55 figures, and 184 Pages.

Target Audience 2024

• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies