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X-Linked Adrenoleukodystrophy (X-ALD) Pipeline Analysis Report 2025

Publisher Expert Market Research
Published May 25, 2025
Length 200 Pages
SKU # EMAR20147096

Description

X-linked adrenoleukodystrophy (X-ALD) ranks as the most common peroxisomal disorder that primarily affects the adrenal glands and the nervous system. It is estimated that the prevalence of X-linked adrenoleukodystrophy is between 1 in 14,000 and 1 in 17,000 male births. X-ALD is caused by the buildup of very long chain fatty acids (VLCFA) as a result of the failure of peroxisomal fatty acid beta-oxidation. Since this rare genetic disorder leads to progressive neurological decline and adrenal insufficiency, there is a growing demand for advanced treatment options that can address the unmet medical needs of the patient population.

Report Coverage

The X-Linked Adrenoleukodystrophy (X-ALD) Drug Pipeline Insight Report by Expert Market Research gives comprehensive insights into X-linked adrenoleukodystrophy (X-ALD) drugs currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for X-linked adrenoleukodystrophy (X-ALD). The report includes the analysis of over 100 pipeline drugs and 50+ companies. The X-linked adrenoleukodystrophy (X-ALD) pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials including their adverse effects on patients suffering from X-linked adrenoleukodystrophy (X-ALD).

The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to X-linked adrenoleukodystrophy (X-ALD).

X-Linked Adrenoleukodystrophy (X-ALD) Drug Pipeline Outlook

X-linked adrenoleukodystrophy (X-ALD), a rare progressive peroxisomal disorder, is characterized by the accumulation of very long chain fatty acids in the nervous system and adrenal cortex. The condition affects more males than females and is linked to the X chromosome. Behavioral and cognitive changes are the common symptoms of X-ALD. The treatment for this genetic disorder is focused on halting or slowing the progression of the disease and improving patient symptoms.

Corticosteroid replacement therapy is recommended for the treatment of adrenal insufficiency in X-ALD patients. To slow the progression of the disease in children, stem cell transplant is the only treatment available. Medications are often prescribed for managing symptoms such as muscle stiffness or seizures. The lack of effective treatments and the severe nature of the disease are encouraging significant investment and research to address the unmet medical needs of the affected patients.

X-Linked Adrenoleukodystrophy (X-ALD) – Drug Pipeline Therapeutic Assessment

This section of the report covers the analysis of X-Linked Adrenoleukodystrophy (X-ALD) drugs based on several segmentations including:

By Phase:
  • Late-Stage Products (Phase 3 and Phase 4)
  • Mid-Stage Products (Phase 2)
  • Early-Stage Products (Phase I)
  • Preclinical and Discovery Stage Products
By Drug Class:
  • Oligonucleotide
  • Peptide
  • Small Molecule
By Route of Administration:
  • Oral
  • Parenteral
  • Others
X-Linked Adrenoleukodystrophy (X-ALD) – Pipeline Assessment Segmentation, By Phases

The report covers phase I, phase II, phase III, phase IV, and early phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis , phase II covers a major share of the total clinical trials for X-linked adrenoleukodystrophy (X-ALD).

X-Linked Adrenoleukodystrophy (X-ALD) – Pipeline Assessment Segmentation, By Drug Classes

The drug molecule categories covered under X-linked adrenoleukodystrophy (X-ALD) pipeline analysis include oligonucleotide, peptide, and small molecule. The report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for X-linked adrenoleukodystrophy (X-ALD).

X-Linked Adrenoleukodystrophy (X-ALD) Clinical Trials Therapeutic Assessment – Competitive Dynamics

The EMR report for the X-linked adrenoleukodystrophy (X-ALD) drug pipeline covers the profile of key companies involved in clinical trials and their drugs under development. Below is the list of a few players involved in X-linked adrenoleukodystrophy (X-ALD) clinical trials:
  • Minoryx Therapeutics, S.L.
  • bluebird bio
  • Poxel SA
  • Viking Therapeutics, Inc.
  • SwanBio Therapeutics, Inc.
X-Linked Adrenoleukodystrophy (X-ALD) – Emerging Drugs Profile

Major drugs currently in the drug pipeline are as follows:

Drug: PXL065

Sponsored by Poxel SA, the objective of this open-label Phase 2a clinical trial is to evaluate the pharmacokinetics and pharmacodynamic parameters of PXL065, a novel drug candidate, after 12 weeks of treatment in around 12 male participants who are affected by adrenomyeloneuropathy, a type of x-linked adrenoleukodystrophy.

Drug: MIN-102

Minoryx Therapeutics, S.L. is conducting a Phase II multi-center clinical trial to investigate the effects of MIN-102, an orally bioavailable and selective PPAR gamma agonist, in an estimated 13 male pediatric patients suffering from cerebral X-linked adrenoleukodystrophy.

Reasons To Buy This Report

The X-Linked Adrenoleukodystrophy (X-ALD) Drug Pipeline Insight Report provides a strategic overview of the latest and future landscape of treatments for X-linked adrenoleukodystrophy (X-ALD). It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into market trends, regulatory environments, and potential growth opportunities within X-linked adrenoleukodystrophy (X-ALD) pipeline insights.

Key Questions Answered in the X-Linked Adrenoleukodystrophy (X-ALD) – Pipeline Insight Report
  • What is the current landscape of X-linked adrenoleukodystrophy (X-ALD) pipeline drugs?
  • How many companies are developing X-linked adrenoleukodystrophy (X-ALD) drugs?
  • How many phase III and phase IV drugs are currently present in X-linked adrenoleukodystrophy (X-ALD) pipeline drugs?
  • Which companies/institutions are leading the X-linked adrenoleukodystrophy (X-ALD) drug development?
  • What is the efficacy and safety profile of X-linked adrenoleukodystrophy (X-ALD) pipeline drugs?
  • What are the opportunities and challenges present in the X-linked adrenoleukodystrophy (X-ALD) drug pipeline landscape?
  • Which company is conducting major trials for X-linked adrenoleukodystrophy (X-ALD) drugs?
  • What geographies are covered for X-linked adrenoleukodystrophy (X-ALD) clinical trials?
  • What are emerging trends in X-linked adrenoleukodystrophy (X-ALD) clinical trials?
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Table of Contents

200 Pages
1 Preface
1.1 Introduction
1.2 Objectives of the Study
1.3 Research Methodology & Assumptions
2 Executive Summary
3 Overview of X-Linked Adrenoleukodystrophy (X-ALD)
3.1 Signs and Symptoms
3.2 Causes
3.3 Risk Factors
3.4 Types of X-Linked Adrenoleukodystrophy
3.5 Diagnosis
3.6 Treatment
4 Patient Profile: X-Linked Adrenoleukodystrophy (X-ALD)
4.1 Patient Profile Overview
4.2 Patient Psychology and Emotional Impact Factors
4.3 Risk Assessment and Treatment Success Rate
5 X-Linked Adrenoleukodystrophy (X-ALD): Epidemiology Snapshot
5.1 X-Linked Adrenoleukodystrophy (X-ALD) Incidence by Key Markets
5.2 X-Linked Adrenoleukodystrophy (X-ALD) – Patients Seeking Treatment in Key Markets
6 X-Linked Adrenoleukodystrophy (X-ALD): Market Dynamics
6.1 Market Drivers and Constraints
6.2 SWOT Analysis
7 X-Linked Adrenoleukodystrophy (X-ALD): Key Facts Covered
7.1 Top Countries Contributing to Clinical Trials in Asia-Pacific
7.2 Top Countries Contributing to Clinical Trials in Europe
7.3 Top Countries Contributing to Clinical Trials in North America
7.4 Top Countries Contributing to Clinical Trials in Other Regions
8 X-Linked Adrenoleukodystrophy (X-ALD), Drug Pipeline Assessment
8.1 Assessment by Treatment Type
8.2 Assessment by Route of Administration
8.3 Assessment by Drug Class
9 EMR Drug Pipeline Comparative Analysis
9.1 List of X-Linked Adrenoleukodystrophy (X-ALD) Pipeline Drugs
9.1.1 By Company
9.1.2 By Phase
9.1.3 By Indication
9.1.4 By Trial Status
9.1.5 By Funder Type
9.2 EMR Attribute Scoring Analysis of Pipeline Drugs (Top Drugs)
10 X-Linked Adrenoleukodystrophy (X-ALD) Drug Pipeline - Late-Stage Products (Phase III and IV) (Top Drugs)
10.1 Comparative Analysis for Late-Stage Drugs
10.1.1 Study Type
10.1.2 Recruitment Status
10.1.3 Company
10.1.4 Funder Type
10.2 Product Level Analysis*
10.2.1 Drug: Pramipexole
10.2.1.1 Product Description
10.2.1.2 Trial ID
10.2.1.3 Sponsor Name
10.2.1.4 Study Type
10.2.1.5 Drug Class
10.2.1.6 Eligibility Criteria
10.2.1.7 Study Record Dates
10.2.1.7.1 First Submitted
10.2.1.7.2 First Posted
10.2.1.7.3 Last Update Posted
10.2.1.7.4 Last Verified
10.2.1.8 Indication
10.2.1.9 Study Design
10.2.1.10 Recruitment Status
10.2.1.11 Enrollment (Estimated)
10.2.1.12 Location Countries
10.2.1.13 Recent Results
10.2.2 Drug: Leriglitazone
10.2.3 Other Drugs
11 X-Linked Adrenoleukodystrophy (X-ALD) Drug Pipeline - Mid-Stage Products (Phase II) (Top Drugs)
11.1 Comparative Analysis for Mid-Stage Drugs
11.1.1 Study Type
11.1.2 Recruitment Status
11.1.3 Company
11.1.4 Funder Type
11.2 Product Level Analysis*
11.2.1 Drug: MIN-102
11.2.1.1 Product Description
11.2.1.2 Trial ID
11.2.1.3 Sponsor Name
11.2.1.4 Study Type
11.2.1.5 Drug Class
11.2.1.6 Eligibility Criteria
11.2.1.7 Study Record Dates
11.2.1.7.1 First Submitted
11.2.1.7.2 First Posted
11.2.1.7.3 Last Update Posted
11.2.1.7.4 Last Verified
11.2.1.8 Indication
11.2.1.9 Study Design
11.2.1.10 Recruitment Status
11.2.1.11 Enrollment (Estimated)
11.2.1.12 Location Countries
11.2.1.13 Recent Results
11.2.2 Drug: PXL065
11.2.3 Genetic: SBT101
11.2.4 Other Drugs
12 X-Linked Adrenoleukodystrophy (X-ALD) Drug Pipeline - Early-Stage Products (Phase I) (Top Drugs)
12.1 Comparative Analysis for Early-Stage Drugs
12.1.1 Study Type
12.1.2 Recruitment Status
12.1.3 Company
12.1.4 Funder Type
12.2 Product Level Analysis*
12.2.1 Drug: VK0214
12.2.1.1 Product Description
12.2.1.2 Trial ID
12.2.1.3 Sponsor Name
12.2.1.4 Study Type
12.2.1.5 Drug Class
12.2.1.6 Eligibility Criteria
12.2.1.7 Study Record Dates
12.2.1.7.1 First Submitted
12.2.1.7.2 First Posted
12.2.1.7.3 Last Update Posted
12.2.1.7.4 Last Verified
12.2.1.8 Indication
12.2.1.9 Study Design
12.2.1.10 Recruitment Status
12.2.1.11 Enrollment (Estimated)
12.2.1.12 Location Countries
12.2.2 Other Drugs
13 X-Linked Adrenoleukodystrophy (X-ALD) Drug Pipeline - Preclinical and Discovery Stage Products (Top Drugs)
13.1 Comparative Analysis for Preclinical and Discovery Stage Drugs
13.1.1 Study Type
13.1.2 Recruitment Status
13.1.3 Company
13.1.4 Funder Type
13.2 Product Level Analysis*
13.2.1 Drug 1
13.2.1.1 Product Description
13.2.1.2 Trial ID
13.2.1.3 Sponsor Name
13.2.1.4 Study Type
13.2.1.5 Drug Class
13.2.1.6 Eligibility Criteria
13.2.1.7 Study Record Dates
13.2.1.7.1 First Submitted
13.2.1.7.2 First Posted
13.2.1.7.3 Last Update Posted
13.2.1.7.4 Last Verified
13.2.1.8 Indication
13.2.1.9 Study Design
13.2.1.10 Recruitment Status
13.2.1.11 Enrollment (Estimated)
13.2.1.12 Location Countries
13.2.2 Other Drugs
14 X-Linked Adrenoleukodystrophy (X-ALD), Key Drug Pipeline Companies
14.1 Minoryx Therapeutics, S.L.
14.1.1 Company Snapshot
14.1.2 Pipeline Product Portfolio
14.1.3 Financial Analysis
14.1.4 Recent News and Developments
14.2 bluebird bio
14.2.1 Company Snapshot
14.2.2 Pipeline Product Portfolio
14.2.3 Financial Analysis
14.2.4 Recent News and Developments
14.3 Poxel SA
14.3.1 Company Snapshot
14.3.2 Pipeline Product Portfolio
14.3.3 Financial Analysis
14.3.4 Recent News and Developments
14.4 Viking Therapeutics, Inc.
14.4.1 Company Snapshot
14.4.2 Pipeline Product Portfolio
14.4.3 Financial Analysis
14.4.4 Recent News and Developments
14.5 SwanBio Therapeutics, Inc.
14.5.1 Company Snapshot
14.5.2 Pipeline Product Portfolio
14.5.3 Financial Analysis
14.5.4 Recent News and Developments
15 Regulatory Framework for Drug Approval, By Region
16 Terminated or Suspended Pipeline Products

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