Fabry Disease Pipeline Overview: Over 18 Companies Driving Innovation in Targeted Therapies and Next-Gen Treatment Approaches | DelveInsight

The therapeutic landscape for Fabry disease, a rare genetic lysosomal storage disorder caused by deficient alpha-galactosidase A enzyme activity, is undergoing significant transformation. Biopharmaceutical developers are moving beyond enzyme replacement therapies (ERTs) toward novel modalities that address underlying disease mechanisms, including gene therapies, substrate reduction therapies, and chaperone-based treatments. Despite existing therapies, unmet needs persist in improving efficacy, reducing infusion burdens, and targeting multi-organ involvement.

Companies such as Amicus Therapeutics, Sanofi Genzyme, Takeda, Protalix, and Idorsia are pioneering innovative drug candidates that aim to enhance treatment outcomes by stabilizing enzyme function, reducing substrate accumulation, or offering one-time curative potential through gene therapy.

DelveInsight's "Fabry Disease - Pipeline Insight, 2025" delivers an in-depth analysis of the global R&D landscape, profiling clinical and preclinical drug candidates with diverse mechanisms of action. The report examines emerging approaches, including gene editing, next-generation ERTs, oral substrate reduction agents, and pharmacological chaperones. It also highlights advancements in delivery technologies, biomarker-driven clinical trials, regulatory pathways, and strategic partnerships shaping the future of Fabry disease treatment. As Fabry disease continues to challenge patients and healthcare systems, these innovations offer promising avenues for improved disease management and patient quality of life.

Interested in learning more about the current treatment landscape and the key drivers shaping the Fabry disease pipeline? Click here: https://www.delveinsight.com/report-store/fabry-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Key Takeaways from the Fabry Disease Pipeline Report
• DelveInsight's Fabry disease pipeline analysis depicts a strong space with 18+ active players working to develop 18+ pipeline drugs for Fabry disease treatment.
• The leading Fabry disease companies include Idorsia Pharmaceuticals, Protalix, Sanofi Genzyme, Sangamo Therapeutics, 4D Molecular Therapeutics, Resverlogix Corp, AVROBIO, Freeline Therapeutics, Ozmosis Research, CellGenTech, uniQure, Codexis, Canbridge, Eleva GmbH, MP6 Therapeutics, Amicus Therapeutics, Sigilon Therapeutics, and others are evaluating their lead assets to improve the Fabry disease treatment landscape.
• Key Fabry disease pipeline therapies in various stages of development include Pegunigalsidase alfa, Lucerastat, Venglustat, 4D-310, RVX000222, AVR-RD-01, FLT190, Lentivirus Alpha-gal A transduced stem cells, AAV gene therapies, CDX-6311, CAN104, CAN201, Enzyme replacement therapies, M052, M053, SIG-007, and others.
• In May 2025, Roche announced the launch of its Elecsys PRO-C3 test, a new diagnostic solution designed to assess the severity of Fabry Disease in patients with metabolic dysfunction-associated steatotic liver disease (MASLD). Developed in collaboration with Nordic Bioscience, the test provides clinicians with a simple and efficient way to identify patients with Fabry Disease of varying severity, facilitating timely intervention and management of the disease.
• In November 2024, Novo Nordisk announced positive results for semaglutide in treating metabolic dysfunction-associated steatohepatitis. In the Phase III Essence trial, a once-weekly 2.4 mg dose of semaglutide improved Fabry Disease and resolved steatohepatitis without worsening Fabry Disease in MASH patients with stage 2 or stage 3 fibrosis, meeting the primary endpoints.
• In October 2024, the FDA granted Breakthrough Therapy designation to survodutide (BI 456906), a dual glucagon/GLP-1 receptor agonist, for treating adults with non-cirrhotic metabolic dysfunction-associated steatohepatitis (MASH) and moderate to advanced fibrosis (stages 2 or 3). This follows promising Phase II results. Boehringer Ingelheim also launched two Phase III studies: LIVERAGE, for MASH patients with fibrosis, and LIVERAGE-Cirrhosis, for those with MASH and cirrhosis.

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Fabry Disease Overview
Fabry Disease is a rare, inherited genetic disorder caused by mutations in the GLA gene, which leads to a deficiency or malfunction of the enzyme alpha-galactosidase A. This enzyme plays a crucial role in breaking down a fatty substance called globotriaosylceramide (Gb3 or GL-3). Without adequate enzyme activity, Gb3 accumulates in the body's cells, disrupting normal function and leading to widespread symptoms. Fabry Disease can affect multiple organ systems and typically presents with chronic burning or tingling pain in the hands and feet, clusters of dark red skin lesions (angiokeratomas), cloudy corneas that impair vision, ringing in the ears (tinnitus), and progressive hearing loss. Inheritance follows an X-linked dominant pattern, meaning the faulty gene is located on the X chromosome. In severe cases, the enzyme is almost entirely inactive, while milder cases may retain partial enzyme function.

Find out more about Fabry disease medication at https://www.delveinsight.com/report-store/fabry-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Fabry Disease Treatment Analysis: Drug Profile
Pegunigalsidase Alfa: Protalix Biotherapeutics
Pegunigalsidase alfa (PRX-102) is an experimental enzyme replacement therapy developed using plant cell culture technology. It is a chemically modified, stabilized form of recombinant α-Galactosidase A, where the protein subunits are cross-linked with short PEG chains to enhance stability and extend circulation time, with a reported half-life of around 80 hours. Aimed at treating Fabry disease, PRX-102 is intended to address unmet clinical needs by improving the pharmacokinetic profile over existing treatments. In May 2020, Protalix and its partner Chiesi submitted a Biologics License Application (BLA) to the FDA for accelerated approval of PRX-102 (1 mg/kg every other week). The therapy has received Orphan Drug Designation in Europe and Fast Track Designation in the U.S. However, in April 2022, the FDA issued a Complete Response Letter due to issues related to facility inspections during the COVID-19 pandemic. The companies are now engaging with the FDA to determine the path forward and plan to submit a similar application to the European Medicines Agency.

Venglustat: Sanofi
Venglustat is a novel, orally administered investigational therapy developed by Sanofi, targeting glycosphingolipids (GSLs), lipid molecules whose abnormal accumulation contributes to several rare genetic disorders, including Fabry disease. By inhibiting GSL synthesis, Venglustat aims to slow disease progression by reducing cellular dysfunction linked to substrate buildup. The drug is currently in Phase II trials for multiple lysosomal storage disorders, including Fabry, Gaucher, Tay-Sachs, and Sandhoff diseases. In 2015, the FDA granted Venglustat Fast Track Designation for Fabry disease, reflecting its potential as a disease-modifying treatment.

Learn more about the novel and emerging Fabry disease pipeline therapies at https://www.delveinsight.com/report-store/fabry-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Fabry Disease Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.

By Stage
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

By Route of Administration
• Oral
• Parenteral
• Intravitreal
• Subretinal
• Topical

By Molecule Type
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Scope of the Fabry Disease Pipeline Report
• Coverage: Global
• Key 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.
• Key Fabry Disease Pipeline Therapies: Pegunigalsidase alfa, Lucerastat, Venglustat, 4D-310, RVX000222, AVR-RD-01, FLT190, Lentivirus Alpha-gal A transduced stem cells, AAV gene therapies, CDX-6311, CAN104, CAN201, Enzyme replacement therapies, M052, M053, SIG-007, and others.

Dive deep into rich insights for drugs used for Fabry disease treatment, visit: https://www.delveinsight.com/report-store/fabry-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Table of Contents
1. Introduction
2. Executive Summary
3. Fabry Disease Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Fabry Disease Pipeline Therapeutics
6. Fabry Disease Pipeline: Late-Stage Products (Phase III)
7. Fabry Disease Pipeline: Mid-Stage Products (Phase II)
8. Fabry Disease Pipeline: Early Stage Products (Phase I)
9. Therapeutic Assessment
10. Inactive Products
11. Company-University Collaborations (Licensing/Partnering) Analysis
12. Key Companies
13. Key Products
14. Unmet Needs
15. Market Drivers and Barriers
16. Future Perspectives and Conclusion
17. Analyst Views
18. Appendix

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Jatin Vimal
jvimal@delveinsight.com
+14699457679
Healthcare Consulting
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This release was published on openPR.