Fabry Disease Drug Pipeline Analysis Report 2025

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 the publisher 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

By Drug Class

• Small Molecules
• Biologics
• Gene Therapy
• Enzyme Replacement Therapy
• Substrate Reduction Therapy

By Route of Administration

• Oral
• Parenteral
• Others

Fabry Disease - 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 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 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

Fabry Disease Emerging Drugs Profile

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?

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