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“Fabry Disease Pipeline Insight, 2026“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Fabry Disease Treatment Landscape.

The Fabry Disease Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.

Some of the key takeaways from the Fabry Disease Pipeline Report:

• Companies across the globe are diligently working toward developing novel Fabry Disease treatment therapies with a considerable amount of success over the years.

• Fabry Disease companies working in the treatment market are Chiesi and Protalix Biotherapeutics, Sanofi Genzyme, Sangamo Therapeutics, Protalix Biotherapeutics, AVROBIO, Sanofi Genzyme, Sangamo Therapeutics, Freeline Therapeutics, 4D Molecular Therapeutics, Idorsia Pharmaceuticals, Greenovation Biotech GMBH, ICON plc, Chiesi Farmaceutici S.p.A., Amicus Therapeutics, Shire, and others, are developing therapies for the Fabry Disease treatment

• Emerging Fabry Disease therapies in the different phases of clinical trials are- ELFABRIO (PRX-102), Venglustat, Isaralgagene civaparvovec (ST-920), PRX-102, AVR-RD-01, Venglustat, ST-920, FLT190, 4D-310, Lucerastat, Moss-aGal, PRX-102, pegunigalsidase alfa, migalastat, Agalsidase alfa, and others are expected to have a significant impact on the Fabry Disease market in the coming years.

• In November 2026, PTC Therapeutics – ST-920: The rolling Biologics License Application (BLA) submission for isaralgagene civaparvovec continues to represent a key upcoming regulatory milestone. As of November 6, 2026, the company’s previously communicated timeline for completing the submission remains the fourth quarter of 2026. However, the completion of the FDA submission or regulatory approval has not yet been confirmed in the available sources.

• In October 2026, Idorsia announced the launch of FAB-KLEAR, a pivotal Phase III clinical trial investigating lucerastat, an investigational oral substrate reduction therapy for Fabry disease. The study plans to enroll 16 treatment-naïve or pseudo-naïve adult male patients to evaluate the therapy’s potential to reduce globotriaosylceramide (Gb3) accumulation in the kidneys. The primary endpoint will assess the change in the kidney Gb3 BLISS score from baseline to Month 18. The clinical development program is being conducted in accordance with the US FDA’s regulatory strategy, with a potential regulatory submission anticipated as early as 2029.

• In September 2026, PTC Therapeutics finalized its acquisition of isaralgagene civaparvovec (ST-920), an investigational one-time AAV-based gene therapy for Fabry disease, from Sangamo Therapeutics. The liver-directed gene therapy is designed to enable patients to produce functional alpha-galactosidase A (α-Gal A) enzyme. Following the acquisition, PTC Therapeutics stated that it planned to complete the rolling Biologics License Application (BLA) submission to the US FDA in the fourth quarter of 2026, potentially bringing the therapy closer to an accelerated approval pathway.

• In June 2026, uniQure presented updated preliminary clinical data from its ongoing AMT-191 study, based on a March 15, 2026, data cutoff. Results from all 11 treated patients demonstrated dose-dependent increases in α-Gal A activity, ranging from 1.0- to 16.2-fold above the mean normal range in the lowest-dose cohort, 14.5- to 229.6-fold in the mid-dose cohort, and 58.7- to 143.6-fold in the highest-dose cohort. All 11 patients had discontinued ERT, while plasma lyso-Gb3 levels remained stable across the dose cohorts. Additional dosing in the mid- and high-dose cohorts remained paused following previously reported liver enzyme elevations.

• In May 2026, Ongoing Clinical Development of ST-920 (Isaralgagene Civaparvovec), Sangamo Therapeutics continued advancing ST-920, one of the leading investigational gene therapies for Fabry disease. The program continued generating long-term follow-up data from the STAAR clinical study, with regulatory and development activities supporting its potential future commercialization.

• In April 2026, Continued Advancement of Gene Therapy Programs, Multiple developers continued to advance liver-directed and AAV-based gene therapy candidates for Fabry disease, with ongoing evaluation of safety, enzyme expression, and biomarker improvements. Investigational programs remained focused on achieving durable α-Gal A production and reducing dependence on lifelong enzyme replacement therapy (ERT).

• In March 2026, GT-GLA-S03 Received FDA Orphan Drug Designation, Glafabra Therapeutics announced that the FDA granted Orphan Drug Designation to GT-GLA-S03, an investigational autologous cell therapy for classic Fabry disease. The therapy is designed as a potentially redosable hematopoietic stem cell–based approach to provide continuous alpha-galactosidase A enzyme production and support long-term disease management.

• In March 2026, Sangamo Therapeutics (Nasdaq: SGMO), a genomic medicine company, announced progress in its rolling Biologics License Application (BLA) submission to the U.S. Food and Drug Administration seeking accelerated approval for Isaralgagene civaparvovec (ST-920), an investigational gene therapy fully owned by the company and being developed for the treatment of adults with Fabry disease.

• In May 2025, All patients treated with Sangamo Therapeutics’ gene therapy candidate ST-920 (isaralgagene civaparvovec) for Fabry disease in a Phase 1/2 trial have now reached the FDA’s required one-year milestone to proceed toward accelerated approval. According to Sangamo’s recent update, a pivotal data readout is anticipated by the end of June. The FDA previously indicated that data from the ongoing Phase 1/2 STAAR trial (NCT04046224) could be sufficient for accelerated approval, potentially eliminating the need for further clinical trials and speeding the therapy’s market entry.

• In March 2025, Sangamo Therapeutics announced its agreement with the FDA on pursuing an accelerated approval pathway for ST-920 in Fabry disease, with a Biologics License Application (BLA) planned for submission in the latter half of 2025.

• In February 2025, Isaralgagene civaparvovec (ST-920) is an experimental gene therapy designed to lower the abnormal buildup of specific fatty substances and potentially slow or stop the progression of Fabry disease. Developed by Sangamo Therapeutics, this therapy is intended to be administered through a single intravenous (IV) infusion. The U.S. Food and Drug Administration (FDA) has granted it Fast Track designation, Orphan Drug status, and Regenerative Medicine Advanced Therapy (RMAT) designation, recognizing its potential to address a serious unmet medical need.

• In February 2025, Amicus Therapeutics shared updates on its migalastat development programs through oral presentations and poster sessions at the 21st Annual WORLD Symposium 2025.

Fabry Disease Overview

Fabry disease is a rare genetic disorder caused by mutations in the GLA gene, which leads to a deficiency or malfunction of the enzyme alpha-galactosidase A. This enzyme is crucial for breaking down a fatty substance called globotriaosylceramide (Gb3 or GL-3) in the body’s cells. When alpha-galactosidase A is deficient or dysfunctional, Gb3 accumulates in various tissues and organs, leading to a wide range of symptoms.

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Emerging Fabry Disease Drugs Under Different Phases of Clinical Development Include:

• 4D 310: 4D Molecular Therapeutics

• GALAFOLD (migalastat): Amicus Therapeutics

• ELFABRIO (PRX-102/pegunigalsidase alfa): CHIESI Farmaceutici and Protalix Biotherapeutics

• Venglustat: Sanofi (Genzyme)

• Isaralgagene civaparvovec (ST-920): Sangamo Therapeutics

• PRX-102: Protalix Biotherapeutics

• AVR-RD-01: AVROBIO

• Venglustat: Sanofi Genzyme

• ST-920: Sangamo Therapeutics

• FLT190: Freeline Therapeutics

• 4D-310: 4D Molecular Therapeutics

• Lucerastat: Idorsia Pharmaceuticals

• Moss-aGal: Greenovation Biotech GMBH

• PRX-102: ICON plc

• pegunigalsidase alfa: Chiesi Farmaceutici S.p.A.

• migalastat: Amicus Therapeutics

• Agalsidase alfa: Shire

Fabry Disease Route of Administration

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

• Intravenous

• Subcutaneous

• Topical

Fabry Disease Molecule Type

Fabry Disease Products have been categorized under various Molecule types, such as

• Monoclonal Antibody

• Peptides

• Polymer

• Small molecule

• Gene therapy

Fabry Disease Pipeline Therapeutics Assessment

• Fabry Disease Assessment by Product Type

• Fabry Disease By Stage and Product Type

• Fabry Disease Assessment by Route of Administration

• Fabry Disease By Stage and Route of Administration

• Fabry Disease Assessment by Molecule Type

• Fabry Disease by Stage and Molecule Type

DelveInsight’s Fabry Disease Report covers around 18+ products under different phases of clinical development like

• Late-stage products (Phase III)

• Mid-stage products (Phase II)

• Early-stage product (Phase I)

• Pre-clinical and Discovery stage candidates

• Discontinued & Inactive candidates

• Route of Administration

Further Fabry Disease product details are provided in the report. Download the Fabry Disease pipeline report to learn more about the emerging Fabry Disease therapies

Some of the key companies in the Fabry Disease Therapeutics Market include:

Key companies developing therapies for Fabry Disease are – Amicus Therapeutics, CHIESI Farmaceutici and Protalix Biotherapeutics, Sanofi (Genzyme), Takeda Pharmaceuticals, Sangamo Therapeutics, UniQure Biopharma, and others.

Fabry Disease Pipeline Analysis:

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 key 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.

The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.

Download Sample PDF Report to know more about Fabry Disease drugs and therapies

Fabry Disease Pipeline Market Drivers

• Advancement of novel therapeutic approaches: The development of next-generation enzyme replacement therapies, pharmacological chaperones, substrate reduction therapies, mRNA-based treatments, and gene therapies is expanding the Fabry disease pipeline.

• High unmet need with existing treatments: Current therapies have limitations related to long-term administration, immunogenicity, tissue penetration, and patient eligibility, creating demand for more durable and effective treatment options.

• Growing focus on gene-based therapies: Gene therapy and gene-editing approaches could potentially provide sustained production of functional α-galactosidase A, supporting continued R&D investment in disease-modifying treatments.

• Improved diagnosis and disease awareness: Greater awareness of rare genetic disorders, genetic testing, and screening initiatives are expected to improve Fabry disease identification and expand the addressable patient population for emerging therapies.

• Expansion of personalized treatment strategies: Mutation-specific approaches, including pharmacological chaperone therapy for amenable GLA mutations, are encouraging more targeted treatment development and precision medicine strategies.

Fabry Disease Pipeline Market Barriers

• High development and treatment costs: Fabry disease therapies, particularly biologics and advanced gene-based treatments, involve substantial development, manufacturing, and treatment costs, potentially limiting accessibility and adoption.

• Small and heterogeneous patient population: Fabry disease is rare and exhibits considerable variation in age of onset, disease severity, organ involvement, and genetic mutations, which can complicate clinical trial recruitment and efficacy assessment.

• Limitations of current treatment approaches: Existing ERTs require repeated administration and may face challenges such as immunogenicity and limited penetration into certain tissues, while chaperone therapy is restricted to patients with amenable mutations.

• Clinical and regulatory uncertainty for advanced therapies: Gene therapy, mRNA, and other novel approaches must demonstrate durable efficacy and acceptable long-term safety, creating challenges for clinical development and regulatory approval.

• Complex competitive and intellectual property landscape: Increasing activity across ERT, chaperone, substrate reduction, mRNA, and gene therapy approaches is creating a complex competitive and IP environment that can influence development and commercialization strategies.

Scope of Fabry Disease Pipeline Drug Insight

• Coverage: Global

• Key Fabry Disease Companies: Chiesi and Protalix Biotherapeutics, Sanofi Genzyme, Sangamo Therapeutics, Protalix Biotherapeutics, AVROBIO, Sanofi Genzyme, Sangamo Therapeutics, Freeline Therapeutics, 4D Molecular Therapeutics, Idorsia Pharmaceuticals, Greenovation Biotech GMBH, ICON plc, Chiesi Farmaceutici S.p.A., Amicus Therapeutics, Shire, and others

• Key Fabry Disease Therapies: ELFABRIO (PRX-102), Venglustat, Isaralgagene civaparvovec (ST-920), PRX-102, AVR-RD-01, Venglustat, ST-920, FLT190, 4D-310, Lucerastat, Moss-aGal, PRX-102, pegunigalsidase alfa, migalastat, Agalsidase alfa, and others

• Fabry Disease Therapeutic Assessment: Fabry Disease current marketed and Fabry Disease emerging therapies

• Fabry Disease Market Dynamics: Fabry Disease market drivers and Fabry Disease market barriers

Request for Sample PDF Report for Fabry Disease Pipeline Assessment and clinical trials

Table of Contents

1. Fabry Disease Report Introduction

2. Fabry Disease Executive Summary

3. Fabry Disease Overview

4. Fabry Disease- Analytical Perspective In-depth Commercial Assessment

5. Fabry Disease Pipeline Therapeutics

6. Fabry Disease Late Stage Products (Phase II/III)

7. Fabry Disease Mid Stage Products (Phase II)

8. Fabry Disease Early Stage Products (Phase I)

9. Fabry Disease Preclinical Stage Products

10. Fabry Disease Therapeutics Assessment

11. Fabry Disease Inactive Products

12. Company-University Collaborations (Licensing/Partnering) Analysis

13. Fabry Disease Key Companies

14. Fabry Disease Key Products

15. Fabry Disease Unmet Needs

16 . Fabry Disease Market Drivers and Barriers

17. Fabry Disease Future Perspectives and Conclusion

18. Fabry Disease Analyst Views

19. Appendix

20. About DelveInsight

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.

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