Fabry Disease Drug Pipeline Analysis Report 2025
Description
Fabry disease (FD) is a rare, inherited lysosomal storage disorder caused by a deficiency in the enzyme alpha-galactosidase A, leading to the accumulation of globotriaosylceramide (GL-3) in cells. This condition primarily affects multiple organs, including the kidneys, heart, and nervous system. Its overall prevalence is estimated to be 1 in 40,000 to 170,000 births. There is a high unmet clinical need for better therapies as current treatments, such as enzyme replacement therapies (ERT), have limitations. The growing focus on gene therapy is likely to drive significant Fabry disease pipeline growth in the coming years, offering hope for improved treatment outcomes.
Report Coverage
The Fabry Disease Drug Pipeline Insight Report by Expert Market Research gives comprehensive insights into Fabry disease therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for Fabry disease. The Fabry disease report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The Fabry disease 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 the condition, and alignment with Fabry disease treatment guidelines to ensure optimal care practices.
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 Fabry disease.
Fabry Disease Drug Pipeline Outlook
Fabry disease is a rare genetic disorder caused by a deficiency of the enzyme alpha-galactosidase A, leading to the accumulation of globotriaosylceramide in cells. This buildup disrupts normal cell function and affects multiple organs, such as the kidneys, heart, and nervous system. The condition is inherited in an X-linked manner, resulting in a significant impact on males, but can also affect females.
Fabry disease is primarily treated by enzyme replacement therapy (ERT), which involves regular injections of the missing enzyme to reduce disease progression. Furthermore, pain management, kidney care, and cardiovascular treatments may be provided to address symptoms. Early diagnosis and intervention are essential to managing the condition and preventing serious complications.
Fabry Disease Epidemiology
The prevalence of classic Fabry disease is estimated to range from 1 in 40,000 to 170,000 births globally. In the United States, it affects approximately 1 in 17,000 to 1 in 117,000 males. In the United Kingdom, about 1 in 40,000 individuals are affected, with the condition impacting 1 in 17,000 Caucasian males. In India, approximately 70 million people are affected by rare genetic diseases, including Fabry disease.
Fabry Disease – Drug Pipeline Therapeutic Assessment
This section of the report covers the analysis of Fabry disease drug candidates based on several segmentations, including:
By Phase
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 III covers a major share of the total Fabry disease clinical trials.
Fabry Disease – Pipeline Assessment Segmentation, By Drug Classes
The drug molecule categories covered under the Fabry disease pipeline analysis include small molecules, biologics, gene therapy, enzyme replacement therapy, and substrate reduction therapy. The Fabry disease report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for Fabry disease.
Fabry Disease Clinical Trials Therapeutic Assessment – Competitive Dynamics
The EMR report for the Fabry disease drug pipeline covers the profile of key companies involved in clinical trials and their drugs under development. It provides a detailed Fabry disease therapeutic assessment, analyzing the competitive dynamics of the clinical trial landscape. Below is the list of a few players involved in Fabry disease clinical trials:
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for Fabry disease. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of Fabry disease drug candidates.
Drug: Pegunigalsidase Alfa
Pegunigalsidase alfa, a PEGylated enzyme replacement therapy, is being studied in a Phase III open-label extension trial sponsored by Chiesi Farmaceutici S.p.A. The objective of this study is to assess the long-term safety, tolerability, and efficacy of 1 mg/kg pegunigalsidase alfa, administered intravenously every two weeks to adult Fabry disease patients. This recombinant enzyme aims to improve pharmacokinetic stability and provide sustained therapeutic benefits for treating Fabry disease.
Drug: Lucerastat
Lucerastat is an oral glucosylceramide synthase inhibitor, designed to reduce Gb3 levels in key organs such as the kidneys and heart. Idorsia Pharmaceuticals Ltd. is sponsoring a Phase III study to evaluate the long-term safety and tolerability of Lucerastat in adult subjects with Fabry disease. The study aims to assess its potential as a long-term treatment option for Fabry disease, including its effects on kidney Gb3 inclusions.
Drug: AL01211
AL01211, developed by AceLink Therapeutics, Inc., is being evaluated in a Phase II study for males with classic Fabry disease who have never received treatment. The study aims to assess the drug's safety, pharmacodynamics, pharmacokinetics, and preliminary efficacy. AL01211, a potent glucosylceramide synthase (GCS) inhibitor, offers superior tissue penetration, particularly in the heart and kidneys, providing a more effective alternative to enzyme replacement therapy (ERT).
Reasons To Buy This Report
The Fabry Disease Drug Pipeline Insight Report provides a strategic overview of the latest and future landscape of treatments for Fabry disease. 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 Fabry disease collaborations, regulatory environments, and potential growth opportunities.
Key Questions Answered in the Fabry Disease – Pipeline Insight Report
Fabry Disease Treatment Market
Fabry Disease Epidemiology
Global Clinical Trials Market
Report Coverage
The Fabry Disease Drug Pipeline Insight Report by Expert Market Research gives comprehensive insights into Fabry disease therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for Fabry disease. The Fabry disease report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The Fabry disease 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 the condition, and alignment with Fabry disease treatment guidelines to ensure optimal care practices.
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 Fabry disease.
Fabry Disease Drug Pipeline Outlook
Fabry disease is a rare genetic disorder caused by a deficiency of the enzyme alpha-galactosidase A, leading to the accumulation of globotriaosylceramide in cells. This buildup disrupts normal cell function and affects multiple organs, such as the kidneys, heart, and nervous system. The condition is inherited in an X-linked manner, resulting in a significant impact on males, but can also affect females.
Fabry disease is primarily treated by enzyme replacement therapy (ERT), which involves regular injections of the missing enzyme to reduce disease progression. Furthermore, pain management, kidney care, and cardiovascular treatments may be provided to address symptoms. Early diagnosis and intervention are essential to managing the condition and preventing serious complications.
Fabry Disease Epidemiology
The prevalence of classic Fabry disease is estimated to range from 1 in 40,000 to 170,000 births globally. In the United States, it affects approximately 1 in 17,000 to 1 in 117,000 males. In the United Kingdom, about 1 in 40,000 individuals are affected, with the condition impacting 1 in 17,000 Caucasian males. In India, approximately 70 million people are affected by rare genetic diseases, including Fabry disease.
Fabry Disease – Drug Pipeline Therapeutic Assessment
This section of the report covers the analysis of Fabry disease drug candidates 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
- Small Molecules
- Biologics
- Gene Therapy
- Enzyme Replacement Therapy
- Substrate Reduction Therapy
- 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 III covers a major share of the total Fabry disease clinical trials.
Fabry Disease – Pipeline Assessment Segmentation, By Drug Classes
The drug molecule categories covered under the Fabry disease pipeline analysis include small molecules, biologics, gene therapy, enzyme replacement therapy, and substrate reduction therapy. The Fabry disease report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for Fabry disease.
Fabry Disease Clinical Trials Therapeutic Assessment – Competitive Dynamics
The EMR report for the Fabry disease drug pipeline covers the profile of key companies involved in clinical trials and their drugs under development. It provides a detailed Fabry disease therapeutic assessment, analyzing the competitive dynamics of the clinical trial landscape. Below is the list of a few players involved in Fabry disease clinical trials:
- Sanofi
- Idorsia Pharmaceuticals Ltd.
- 4D Molecular Therapeutics
- UniQure Biopharma B.V.
- Bio Sidus SA
- ISU Abxis Co., Ltd.
- AceLink Therapeutics, Inc.
- Chiesi Farmaceutici S.p.A.
- Amicus Therapeutics
- Sangamo Therapeutics
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for Fabry disease. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of Fabry disease drug candidates.
Drug: Pegunigalsidase Alfa
Pegunigalsidase alfa, a PEGylated enzyme replacement therapy, is being studied in a Phase III open-label extension trial sponsored by Chiesi Farmaceutici S.p.A. The objective of this study is to assess the long-term safety, tolerability, and efficacy of 1 mg/kg pegunigalsidase alfa, administered intravenously every two weeks to adult Fabry disease patients. This recombinant enzyme aims to improve pharmacokinetic stability and provide sustained therapeutic benefits for treating Fabry disease.
Drug: Lucerastat
Lucerastat is an oral glucosylceramide synthase inhibitor, designed to reduce Gb3 levels in key organs such as the kidneys and heart. Idorsia Pharmaceuticals Ltd. is sponsoring a Phase III study to evaluate the long-term safety and tolerability of Lucerastat in adult subjects with Fabry disease. The study aims to assess its potential as a long-term treatment option for Fabry disease, including its effects on kidney Gb3 inclusions.
Drug: AL01211
AL01211, developed by AceLink Therapeutics, Inc., is being evaluated in a Phase II study for males with classic Fabry disease who have never received treatment. The study aims to assess the drug's safety, pharmacodynamics, pharmacokinetics, and preliminary efficacy. AL01211, a potent glucosylceramide synthase (GCS) inhibitor, offers superior tissue penetration, particularly in the heart and kidneys, providing a more effective alternative to enzyme replacement therapy (ERT).
Reasons To Buy This Report
The Fabry Disease Drug Pipeline Insight Report provides a strategic overview of the latest and future landscape of treatments for Fabry disease. 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 Fabry disease collaborations, regulatory environments, and potential growth opportunities.
Key Questions Answered in the Fabry Disease – Pipeline Insight Report
- Which companies/institutions are leading the Fabry disease drug development?
- What is the efficacy and safety profile of Fabry disease pipeline drugs?
- Which company is leading the Fabry disease pipeline development activities?
- What is the current Fabry disease commercial assessment?
- What are the opportunities and challenges present in the Fabry disease drug pipeline landscape?
- What is the efficacy and safety profile of Fabry disease pipeline drugs?
- Which company is conducting major trials for Fabry disease drugs?
- Which companies/institutions are involved in Fabry disease collaborations aimed at providing enhanced therapeutic alternatives for patients?
- What are the geographies covered for clinical trials in Fabry disease?
Fabry Disease Treatment Market
Fabry Disease Epidemiology
Global Clinical Trials Market
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 Fabry Disease
- 3.1 Signs and Symptoms
- 3.2 Causes
- 3.3 Risk Factors
- 3.4 Diagnosis
- 3.5 Treatment
- 4 Patient Profile: Fabry Disease
- 4.1 Patient Profile Overview
- 4.2 Patient Psychology and Emotional Impact Factors
- 4.3 Risk Assessment and Treatment Success Rate
- 5 Fabry Disease: Epidemiology Snapshot
- 5.1 Fabry Disease Incidence by Key Markets
- 5.2 Fabry Disease – Patients Seeking Treatment in Key Markets
- 6 Fabry Disease: Market Dynamics
- 6.1 Market Drivers and Constraints
- 6.2 SWOT Analysis
- 7 Fabry Disease: 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 Fabry Disease, 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 Fabry Disease 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 Fabry Disease 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 Biological: Replagal
- 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: Pegunigalsidase Alfa
- 10.2.3 Drug: Lucerastat
- 10.2.4 Other Drugs
- 11 Fabry Disease 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: AL01211
- 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 Other Drugs
- 12 Fabry Disease 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: AMT-191
- 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 Biological: 4D-310
- 12.2.3 Other Drugs
- 13 Fabry Disease 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 Fabry Disease, Key Drug Pipeline Companies
- 14.1 Sanofi
- 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 Idorsia Pharmaceuticals Ltd.
- 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 4D Molecular Therapeutics
- 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 UniQure Biopharma B.V.
- 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 Bio Sidus SA
- 14.5.1 Company Snapshot
- 14.5.2 Pipeline Product Portfolio
- 14.5.3 Financial Analysis
- 14.5.4 Recent News and Developments
- 14.6 ISU Abxis Co., Ltd.
- 14.6.1 Company Snapshot
- 14.6.2 Pipeline Product Portfolio
- 14.6.3 Financial Analysis
- 14.6.4 Recent News and Developments
- 14.7 AceLink Therapeutics, Inc.
- 14.7.1 Company Snapshot
- 14.7.2 Pipeline Product Portfolio
- 14.7.3 Financial Analysis
- 14.7.4 Recent News and Developments
- 14.8 Chiesi Farmaceutici S.p.A.
- 14.8.1 Company Snapshot
- 14.8.2 Pipeline Product Portfolio
- 14.8.3 Financial Analysis
- 14.8.4 Recent News and Developments
- 14.9 Amicus Therapeutics
- 14.9.1 Company Snapshot
- 14.9.2 Pipeline Product Portfolio
- 14.9.3 Financial Analysis
- 14.9.4 Recent News and Developments
- 14.10 Sangamo Therapeutics
- 14.10.1 Company Snapshot
- 14.10.2 Pipeline Product Portfolio
- 14.10.3 Financial Analysis
- 14.10.4 Recent News and Developments
- 15 Regulatory Framework for Drug Approval, By Region
- 16 Terminated or Suspended Pipeline Products
Pricing
Currency Rates
Questions or Comments?
Our team has the ability to search within reports to verify it suits your needs. We can also help maximize your budget by finding sections of reports you can purchase.
