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Global Adrenoleukodystrophy Treatment Market - 2021-2028

Published: | DataM Intelligence | 180 Pages | Delivery time: 2 business days


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Global Adrenoleukodystrophy Treatment Market - 2021-2028
Published: November 16, 2021
DataM Intelligence
Content info: 180 Pages
Delivery time: 2 business days
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  • Description
  • Table of Contents

Market Overview

The Adrenoleukodystrophy Treatment Market size was valued at US$ YY billion in 2020 and is estimated to reach US$ YY billion by 2028, growing at a CAGR of YY % during the forecast period (2021-2028).

Adrenoleukodystrophy is a genetic disorder in which the membrane (myelin sheath) that protects nerve cells in the brain is damaged. It is a disease linked to the X chromosome.

Market Dynamics

Due to the increasing cases of X-linked recessive conditions and increasing R&D for adrenoleukodystrophy treatment is estimated to drive the Adrenoleukodystrophy Treatment Market in the forecast period.

The increasing cases of X-linked recessive conditions are expected to drive the adrenoleukodystrophy drugs market

Genetic problems related to mutations in genes on the X chromosome are referred to as X-linked recessive inheritance. When a male-only has one X chromosome, then a male having such a mutation will be affected. A girl with a gene mutation on one X chromosome but a normal gene on the other is usually unharmed. ALD is caused by a mutation in the ABCD1 gene on the X chromosome, an X-linked recessive disorder. Because a female has two X chromosomes, she can inherit the faulty gene and still have another X chromosome to compensate for the mutation. However, because males only have one X chromosome, the condition is caused by a gene defect. Female ALD carriers have a 25% (1 in 4) probability of producing a carrier daughter and a 25% (1 in 4) chance of having a boy with the illness each pregnancy. There are atleast 533 disorders due to the involvement of the genes on the X chromosome. The treatment for X-linked adrenoleukodystrophy (X-ALD) depends on the signs and symptoms present in each person. There are treatments available such as corticosteroids are used to correct hormone levels in cases of adrenal insufficiency. Physical treatment that may be beneficial to males suffering from adrenomyeloneuropathy (AMN). Bone marrow transplant is only suggested for boys with brain involvement, as evidenced by MRI but with minor neurological or psychosocial symptoms and a normal clinical neurologic assessment. There are numerous hazards associated with this treatment, and it is not suggested in cases of severe neurologic symptoms. Hence, with the rise of X-linked recessive conditions, it is estimated to increase adrenoleukodystrophy cases, increasing the demand for drugs in the market.

Increasing R&D for adrenoleukodystrophy treatment is estimated to drive the Adrenoleukodystrophy Treatment Market in the forecast period

There is no cure for adrenoleukodystrophy. If done when neurological symptoms first arise, stem cell transplantation may stop the course of ALD. Doctors will concentrate on reducing symptoms and decreasing the progression of disease. In a clinical trial conducted in recent times, boys with early-stage cerebral ALD were treated with gene therapy as an alternative to stem cell transplantation. Gene therapy's early findings are encouraging. In 88 percent of the boys who took part in the study, disease progression slowed. To examine the long-term results and safety of gene therapy for cerebral ALD more study is needed.

Data from nearly seven years of follow-up suggests one-time treatment with Eli-cel may durably stabilise cerebral adrenoleukodystrophy disease progression. For example, new data from the clinical development programme for bluebird bio-Inc.'s experimental elivaldogene autotemcel (Eli-cel, Lenti-DTM) gene therapy in patients with cerebral adrenoleukodystrophy has been released (CALD). The most severe form of adrenoleukodystrophy (ALD) is CALD, a rare X chromosome-linked condition that affects one out of every 21,000 male births worldwide. Approximately 40% of boys with ALD are estimated to develop CALD. The new Eli-cel trial data included the updated results from the Phase II/III Starbeam study (ALD-102) and the long-term follow-up study LTF-304. This includes the fact that 90 percent (27) of the 32 patients enrolled in ALD-102 were alive and free of major functional disabilities (MFDs) at Month 24, and that there was no evidence of MFDs in the 27 patients who completed ALD-102 and enrolled in LTF-304 after nearly seven years of follow-up. The company reported safety outcomes from the Phase III ALD-104 study. ALD-104 is assessing the safety and efficacy of Eli-cel in patients with CALD after myeloablative conditioning using fludarabine and busulfan. After Eli-cel infusion the primary safety endpoint is the proportion of patients with neutrophil engraftment. On May 23, 2018, bluebird bio, Inc. got approval from the U.S. Food and Drug Administration (FDA) for their Breakthrough Therapy designation to Lenti-D™ for the treatment of patients with cerebral adrenoleukodystrophy (CALD).

Insufficient outcomes of available treatments for adrenoleukodystrophy and the drugs is estimated to hamper the market

A stem cell transplant, in which the patient receives blood stem cells from a genetically matched donor, is the only successful therapy option for cerebral ALD. The goal is to provide healthy stem cells that can produce the protein that is missing in ALD males. There is a long preparation phase that takes place before the stem cell transplant. Chemotherapy is used to prepare the body for receiving the donor cells. Although it has been established that a donor stem cell transplant can slow the progression of ALD, it comes with its own set of problems and risks: Finding a donor match with a comparable or identical genetic composition might be tricky. Because the immune system is suppressed to allow the recipient's body to accept the transplanted new stem cells, there is a high risk of infection. The illness continues to worsen for 6 to 18 months after transplant; therefore, early therapy is critical.

VLCFA build-up in primary cultured human XALD fibroblasts is normalised when cellular cholesterol is altered by either cholesterol depletion or treatment with the cholesterol-lowering medication lovastatin. On the other hand, high cholesterol levels may enhance VLCFA levels in cultured human fibroblasts from XALD patients or controls. Abcd1deficient XALD mice have higher plasma cholesterol levels than wildtype controls fed high cholesterol. Unlike wild-type mice, Abcd1 defective mice's plasma cholesterol levels could not be raised further by high dietary cholesterol. However, neither lovastatin nor simvastatin medication was effective. These studies concluded that lovastatin should not be prescribed as a therapy to lower levels of VLCFA in patients with X-ALD.

COVID-19 Impact Analysis

COVID-19 has affected the healthcare industry. To stop its development, government-imposed lockdown. People are fearful they will experience negative health and occupational effects from the COVID-19 pandemic. COVID-19 has a substantial impact on clinical trials. The impact includes: eroding sponsors' financial ability to pay clinical trials, social distancing, hindering the recruitment of non-COVID-19 clinical trial participants, deterring participants from visiting clinical sites owing to fear of COVID-19 infections, and forcing trial staff to work remotely. All the manufacturing, as well as supply chains, have been hit due to the COVID-19 restrictions. Hence, the Adrenoleukodystrophy Treatment Market is estimated to see a negative impact.

Segment Analysis

Lorenzo's oil is estimated to dominate the adrenoleukodystrophy drugs market

Erucic acid and oleic acid are two compounds that makeup Lorenzo's oil. Lorenzo's oil is used as a medication. It is used to treat two related inherited nerve system disorders. Adrenoleukodystrophy (ALD), which affects children, and adrenomyeloneuropathy, occurs in adults are two very rare disorders. Lorenzo's oil is a 4:1 blend of glyceryl trioleate (GTO) and glyceryl trierucate (GTE). Lorenzo's oil is taken orally and is generally well tolerated, though it has been linked to a slight reduction in platelets and an increase in liver transaminases.

In the United States, Lorenzo's oil is only available to patients participating in a clinical trial. There is currently an effort to obtain FDA approval of Lorenzo's oil as a prescription drug. Lorenzo's oil is jointly manufactured by SHS International and Croda International of Britain. SHS International is the worldwide distributor of the oil. Lorenzo's oil is still in phase-3 clinical trials.

Childhood Cerebral ALD segment is estimated to dominate the Adrenoleukodystrophy Treatment Market

Adrenocortical insufficiency, a variety of neurocognitive and behavioural impairments are common signs of childhood cerebral X-linked adrenoleukodystrophy (XALD). Adrenoleukodystrophy in children is known as cerebral adrenoleukodystrophy (CALD). Mutations in the ABCD1 gene cause this serious neurological disorder. Mutations in this gene cause defects in cells that metabolise very-long-chain fatty acids, causing them to build up in the CNS and the blood. This buildup is responsible for the breakdown of the protein coat, that insulates the nerve fibres to protect them. Without the protein coat, the nerve cells are fragile and easily damaged.

The European Commission (EC) has granted marketing authorization to SKYSONATM (elivaldogene autotemcel, Lenti-DTM), one-time gene therapy for the treatment of early cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age who have an ABCD1 genetic mutation and for whom a human leukocyte antigen (HLA)-matched sibling hematopoietia has not been found. SKYSONA is the first one-time gene therapy licenced in the European Union (EU) to treat CALD. This rare neurodegenerative illness strikes children and causes rapid, irreversible loss of neurologic function, as well as death.

Geographical Analysis

North America region is expected to dominate the Adrenoleukodystrophy Treatment Market

The increasing clinical trials and presence of major key players in this region are expected to dominate the market.

Minoryx Therapeutics is developing MIN-102, an oral medication for the treatment of CALD and AMN. Both Europe and the United States have designated MIN-102 as an orphan medication. In March 2017, phase 1 trials on the safety of the medication MIN-102 in healthy male volunteers came to an end. The trials also looked into the drug's capacity to go to where it was required in the brain. According to the findings, participants tolerated MIN-102 well, both at the usual dose and at considerably greater amounts than were required to produce an impact. They also discovered that the medication might reach the brain. MIN-102 is now undergoing a Phase 2/3 clinical trial in adult patients with AMN. Another orally administered medication being explored to treat ALD is STEM CELL/BONE MARROW TRANSPLANT. It functions as a TR agonist by enhancing thyroid receptor beta (TR) activation. TR has a variety of functions, but the researchers discovered that increasing its activity resulted in higher levels of the ABCD2 gene, which was able to lower VLCFA levels. Reduced VLCFA levels should assist to slow the disease's course. The United States Food and Medication Administration has designated STEM CELL/BONE MARROW TRANSPLANT, a medicine being researched by Viking Therapeutics, as an orphan drug after promising results from preclinical testing (not in humans) (FDA).

THERAPY is being tested in a Phase 1/2 trial in children with cerebral adrenoleukodystrophy (CCALD). The safety of THERAPY for persons with CCALD will be investigated in NeuroVia's Phase 1/2 trial. The trial will also look at the drug's pharmacokinetics, or how the body uses the medication and how it enters, passes through, and exits the body. Hence, with all the drugs in the pipeline, this region is estimated to dominate in the forecast period.

Competitive Landscape

Major key players in the Adrenoleukodystrophy Treatment Market are Bluebird Bio Inc, NeuroVia, Inc., Orpheris, Inc., Minoryx, MedDay Pharmaceuticals, SwanBio Therapeutics and Viking Therapeutics

NeuroVia, Inc.

Overview: NeuroVia, Inc. is dedicated to meeting unmet medical requirements in neurological illnesses, with the primary goal of preventing the onset of debilitating neurological abnormalities caused by X-ALD.

Product Portfolio: The company comprises original medicines, value-added medicines, standard generics and consumer healthcare.

Key Development: In 2018, NeuroVia, Inc. initiated Phase 1/2 clinical study of their lead drug candidate, THERAPY, in patients with childhood cerebral adrenoleukodystrophy (CCALD).

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Target Audience

Service Providers/ Buyers

Industry Investors/Investment Bankers

Education & Research Institutes

Research Professionals

Emerging Companies


Market Segmentation

Adrenoleukodystrophy Treatment Market - By Treatment

Lorenzo's oil


Stem cell therapy

Gene therapy

Stem Cell/Bone Marrow Transplant

Adrenoleukodystrophy Treatment Market - By Application

Childhood Cerebral ALD

Adrenomyeloneuropathy (AMN)



Adrenoleukodystrophy Treatment Market - By Region

North America


South America

Asia Pacific

Middle East and Africa

Table of Contents
Product Code: DMPH4273

Table of Contents

1. Adrenoleukodystrophy Treatment Market Methodology and Scope

  • 1.1. Research Methodology
  • 1.2. Research Objective and Scope of the Report

2. Adrenoleukodystrophy Treatment Market - Market Definition and Overview

3. Adrenoleukodystrophy Treatment Market - Executive Summary

  • 3.1. Market Snippet by Treatment
  • 3.2. Market Snippet by Application
  • 3.3. Market Snippet by Region

4. Adrenoleukodystrophy Treatment Market - Market Dynamics

  • 4.1. Market Impacting Factors
    • 4.1.1. Drivers
      • Due to the increasing cases of X-linked recessive conditions
      • Increasing R&D for adrenoleukodystrophy treatment
    • 4.1.2. Restraints:
      • Insufficient outcomes of available treatments for adrenoleukodystrophy and the drugs
    • 4.1.3. Opportunity
    • 4.1.4. Impact Analysis

5. Adrenoleukodystrophy Treatment Market - Industry Analysis

  • 5.1. Porter's Five Forces Analysis
  • 5.2. Epidemiology Analysis
  • 5.3. Supply Chain Analysis
  • 5.4. Pricing Analysis
  • 5.5. Regulatory Analysis
  • 5.6. Reimbursement Analysis
  • 5.7. Unmet Needs

6. Adrenoleukodystrophy Treatment Market - Pipeline Analysis

  • 6.1. Phase-1 Clinical Trial
    • 6.1.1. VK0214
  • 6.2. Phase-2 Clinical Trial
    • 6.2.1. MIN-102
  • 6.3. Phase-3 Clinical Trial
  • 6.4. Key developments

7. Adrenoleukodystrophy Treatment Market - COVID-19 Analysis

  • 7.1. Analysis of Covid-19 on the Market
    • 7.1.1. Before COVID-19 Market Scenario
    • 7.1.2. Present COVID-19 Market Scenario
    • 7.1.3. After COVID-19 or Future Scenario
  • 7.2. Pricing Dynamics Amid Covid-19
  • 7.3. Demand-Supply Spectrum
  • 7.4. Government Initiatives Related to the Market During Pandemic
  • 7.5. Manufacturers Strategic Initiatives
  • 7.6. Conclusion

8. Adrenoleukodystrophy Treatment Market - By Treatment

  • 8.1. Introduction
    • 8.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Segment
    • 8.1.2. Market Attractiveness Index, By Treatment Segment
  • 8.2. Lorenzo's oil *
    • 8.2.1. Introduction
    • 8.2.2. Market Size Analysis, US$ Million, 2019-2028 and Y-o-Y Growth Analysis (%), 2020-2028
  • 8.3. Therapy
  • 8.4. Stem Cell/Bone Marrow Transplant

9. Adrenoleukodystrophy Treatment Market - By Application

  • 9.1. Introduction
    • 9.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Application Segment
    • 9.1.2. Market Attractiveness Index, By Application Segment
  • 9.2. Childhood Cerebral ALD*
    • 9.2.1. Introduction
    • 9.2.2. Market Size Analysis, US$ Million, 2019-2028 and Y-o-Y Growth Analysis (%), 2020-2028
  • 9.3. Adrenomyeloneuropathy (AMN)
  • 9.4. Other

10. Adrenoleukodystrophy Treatment Market - By Region

  • 10.1. Introduction
    • 10.1.1. Market Size Analysis, US$ Million, 2019-2028 and Y-o-Y Growth Analysis (%), 2020-2028, By Region
    • 10.1.2. Market Attractiveness Index, By Region
  • 10.2. North America
    • 10.2.1. Introduction
    • 10.2.2. Key Region-Specific Dynamics
    • 10.2.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
    • 10.2.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Application
    • 10.2.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • U.S.
      • Canada
      • Mexico
  • 10.3. Europe
    • 10.3.1. Introduction
    • 10.3.2. Key Region-Specific Dynamics
    • 10.3.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
    • 10.3.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Application
    • 10.3.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • Germany
      • U.K.
      • France
      • Italy
      • Spain
      • Rest of Europe
  • 10.4. South America
    • 10.4.1. Introduction
    • 10.4.2. Key Region-Specific Dynamics
    • 10.4.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
    • 10.4.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Application
    • 10.4.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • Brazil
      • Argentina
      • Rest of South America
  • 10.5. Asia Pacific
    • 10.5.1. Introduction
    • 10.5.2. Key Region-Specific Dynamics
    • 10.5.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
    • 10.5.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Application
    • 10.5.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • China
      • India
      • Japan
      • Australia
      • Rest of Asia Pacific
  • 10.6. Middle East and Africa
    • 10.6.1. Introduction
    • 10.6.2. Key Region-Specific Dynamics
    • 10.6.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
    • 10.6.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Application

11. Adrenoleukodystrophy Treatment Market - Competitive Landscape

  • 11.1. Key Developments and Strategies
  • 11.2. Company Share Analysis
  • 11.3. Product Benchmarking

12. Adrenoleukodystrophy Treatment Market - Company Profiles

  • 12.1. NeuroVia, Inc.
    • 12.1.1. Company Overview
    • 12.1.2. Product Portfolio and Description
    • 12.1.3. Key Highlights
    • 12.1.4. Financial Overview
  • 12.2. Bluebird Bio Inc
  • 12.3. Orpheris, Inc.
  • 12.4. Minoryx
  • 12.5. SwanBio Therapeutics
  • 12.6. Viking Therapeutics (*LIST NOT EXHAUSTIVE)

13. Adrenoleukodystrophy Treatment Market - DataM

  • 13.1. Appendix
  • 13.2. About Us and Services
  • 13.3. Contact Us