Fabry Disease Clinical Trial Pipeline Gains Momentum: 18+ Companies Lead the Charge in Pioneering New Treatments | DelveInsight
DelveInsight’s “Fabry Disease Pipeline Insight 2025” report provides comprehensive insights about 18+ companies and 18+ pipeline drugs in the Fabry Disease pipeline landscape. It covers the Fabry Disease pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Fabry Disease therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
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Key Takeaways from the Fabry Disease Pipeline Report
- On January 21, 2026- Chiesi Farmaceutici S.p.A. conducted a CLI-06657AA1-03 (formerly PB-102-F51) is to evaluate the long-term safety, tolerability, and efficacy of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry patients who have successfully completed PB-102-F50.
- On January 15, 2026- Amicus Therapeutics announced a Phase 3b study to evaluate the safety, PK, PD, and efficacy of 12 months of migalastat treatment in pediatric subjects 2 to < 12 years of age with Fabry disease and with amenable GLA variants. Subjects must be either naïve to enzyme replacement therapy (ERT) or have stopped ERT at least 14 days before Baseline visit.
- DelveInsight’s Fabry Disease pipeline report depicts a robust space with 18+ active players working to develop 18+ pipeline therapies for Fabry Disease treatment.
- The leading Fabry Disease Companies such as Idorsia Pharmaceuticals, Protalix, Sanofi Genzyme, Sangamo Therapeutics, 4D Molecular Therapeutics, Resverlogix Corp, AVROBIO, Freeline Therapeutics, Ozmosis Research Inc., CellGenTech, Inc., uniQure, Codexis, Canbridge, Eleva GmbH, MP6 Therapeutics, Amicus Therapeutics, Sigilon Therapeutics and others.
- Promising Fabry Disease Therapies such as AGALSIDASE BETA (GZ419828), Acetaminophen, Diphenhydramine, Migalastat HCl 20 mg, AMT-191, 4D-310, AL01211, Pegunigalsidase Alfa and others.
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The Fabry Disease Pipeline Report provides a disease overview, pipeline scenario, and therapeutic assessment of the key pipeline therapies in this domain. The Fabry Disease Pipeline Report also highlights the unmet needs with respect to the Fabry Disease.
Fabry Disease Overview
Fabry disease is a rare, inherited genetic disorder caused by a deficiency or absence of the enzyme alpha-galactosidase A. This enzyme is responsible for breaking down a fatty substance called globotriaosylceramide (GL-3 or Gb3). When the enzyme does not work properly, GL-3 accumulates in cells throughout the body, leading to progressive organ damage. Fabry disease is X-linked, meaning it primarily affects males, though females can also experience symptoms ranging from mild to severe. Common symptoms often begin in childhood or adolescence and may include burning pain in the hands and feet (acroparesthesia), heat and exercise intolerance, reduced sweating, gastrointestinal issues, and characteristic skin lesions called angiokeratomas. As the disease progresses, it can cause serious complications involving the kidneys, heart, and brain, such as kidney failure, cardiomyopathy, arrhythmias, and stroke.
Fabry Disease Emerging Drugs Profile
- Pegunigalsidase Alfa: Protalix Biotherapeutics
Pegunigalsidase alfa (PRX-102) is an investigational, plant cell culture-expressed, and chemically modified stabilized version of the recombinant α-Galactosidase-A enzyme. Protein sub-units are covalently bound via chemical cross-linking using short PEG moieties, resulting in a molecule with unique pharmacokinetic parameters. In clinical studies, PRX–102 has been observed to have a circulatory half-life of approximately 80 hours. PRX–102 has been designed to potentially address the continued unmet clinical need in Fabry patients. In May 2020, the companies filed an application with the U.S. Food and Drug Administration (FDA) seeking the accelerated approval of PRX-102, given at a dose of 1 mg/kg every other week, for the treatment of adults with Fabry. PRX-102 received orphan drug designation in Europe and fast-track designation in the U.S. Both designations are meant to speed up the therapy’s development and review process. After granting it priority review, the agency rejected the application in April 2022 due to issues with facility inspections and manufacturing processes, partially caused by travel restrictions during the COVID-19 pandemic. Protalix and Chiesi have requested a meeting with the FDA to discuss the regulatory path toward PRX-102’s approval in the U.S. The companies plan to file a similar regulatory application with the European Medicines Agency later this year to seek the therapy’s approval in the EU.
- Venglustat: Sanofi
GSLs are cellular building blocks whose abnormal accumulation is implicated in several rare diseases, responsible for both cell dysfunction and disease progression. Venglustat is a novel, oral investigational therapy that has the potential to slow the progression of certain diseases by inhibiting abnormal GSL accumulation. Venglustat is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority. Venglustat continues in Phase II for Gaucher, Fabry, Tay-Sachs and Sandhoff diseases. In 2015, the FDA fast-tracked this drug for Fabry disease.
- 4D 310: 4D Molecular Therapeutics
4D-310 is a novel adeno-associated virus (AAV) gene therapy comprised of two active components: the capsid (4D-C102) and the transgene cassette, which encodes a codon-optimized full length human GLA transgene driven by the CAG promoter. 4D-310 has been engineered so that it cannot replicate. The drug is being investigated in Phase I/II stage of development for the treatment of patients with Fabry Disease.
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The Fabry Disease Pipeline Report Provides Insights into
- The report provides detailed insights about companies that are developing therapies for the treatment of Fabry Disease with aggregate therapies developed by each company for the same.
- It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Fabry Disease Treatment.
- Fabry Disease Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
- Fabry Disease Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
- Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Fabry Disease market
Fabry Disease Companies
Idorsia Pharmaceuticals, Protalix, Sanofi Genzyme, Sangamo Therapeutics, 4D Molecular Therapeutics, Resverlogix Corp, AVROBIO, Freeline Therapeutics, Ozmosis Research Inc., CellGenTech, Inc., uniQure, Codexis, Canbridge, Eleva GmbH, MP6 Therapeutics, Amicus Therapeutics, Sigilon Therapeutics and others.
The Fabry Disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
- Oral
- Parenteral
- Intravitreal
- Subretinal
- Topical.
- Molecule Type
Fabry Disease Products have been categorized under various Molecule types such as,
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
Unveil the future of Fabry Disease Treatment. Learn about new drugs, pipeline developments, and key companies with DelveInsight’s expert analysis @ Fabry Disease Market Drivers and Barriers
Scope of the Fabry Disease Pipeline Report
- Coverage- Global
- Fabry Disease Companies- Idorsia Pharmaceuticals, Protalix, Sanofi Genzyme, Sangamo Therapeutics, 4D Molecular Therapeutics, Resverlogix Corp, AVROBIO, Freeline Therapeutics, Ozmosis Research Inc., CellGenTech, Inc., uniQure, Codexis, Canbridge, Eleva GmbH, MP6 Therapeutics, Amicus Therapeutics, Sigilon Therapeutics and others.
- Fabry Disease Therapies- AGALSIDASE BETA (GZ419828), Acetaminophen, Diphenhydramine, Migalastat HCl 20 mg, AMT-191, 4D-310, AL01211, Pegunigalsidase Alfa and others.
- Fabry Disease Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
- Fabry Disease Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
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Table of Contents
- Introduction
- Executive Summary
- Fabry Disease: Overview
- Pipeline Therapeutics
- Therapeutics Assessment
- Fabry Disease – DelveInsight’s Analytical Perspective
- In-depth Commercial Assessment
- Fabry Disease Collaboration Deals
- Late Stage Products (Preregistration)
- Pegunigalsidase Alfa: Protalix Biotherapeutics
- Mid Stage Products (Phase II)
- Venglustat: Sanofi
- Early stage products (Phase I/II)
- 4D 310: 4D Molecular Therapeutics
- Inactive Products
- Fabry Disease Key Companies
- Fabry Disease Key Products
- Fabry Disease- Unmet Needs
- Fabry Disease- Market Drivers and Barriers
- Fabry Disease- Future Perspectives and Conclusion
- Fabry Disease Analyst Views
- Fabry Disease Key Companies
- Appendix
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