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
- Oligonucleotide
- Peptide
- Small Molecule
- Oral
- Parenteral
- Others
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.
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?
Global Rare Diseases Treatment Market
Global Clinical Trials Market
- 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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