Press release
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.
Request a sample and discover the recent breakthroughs happening in the Fabry disease pipeline landscape at https://www.delveinsight.com/report-store/fabry-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr
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
Contact Us:
Jatin Vimal
jvimal@delveinsight.com
+14699457679
Healthcare Consulting
https://www.delveinsight.com/consulting-services
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. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform, PharmDelve.
This release was published on openPR.
Permanent link to this press release:
Copy
Please set a link in the press area of your homepage to this press release on openPR. openPR disclaims liability for any content contained in this release.
You can edit or delete your press release Fabry Disease Pipeline Overview: Over 18 Companies Driving Innovation in Targeted Therapies and Next-Gen Treatment Approaches | DelveInsight here
News-ID: 4019047 • Views: …
More Releases from DelveInsight

Cognitive Impairment Associated with Schizophrenia Market to Expand Significantl …
Cognitive Impairment Associated with Schizophrenia (CIAS) is a core and debilitating feature of schizophrenia, significantly impacting daily functioning, social interactions, and overall quality of life. Despite being a major contributor to long-term disability in affected individuals, CIAS remains a largely unmet medical need, with no FDA-approved treatments specifically targeting cognitive deficits.
DelveInsight's latest report, "Cognitive Impairment Associated with Schizophrenia - Market Insight, Epidemiology, and Market Forecast - 2034," provides a comprehensive…

Cervical Dystonia Market Set for Strong Growth Through 2034, Led by AbbVie, Ipse …
Cervical dystonia, also known as spasmodic torticollis, is a rare but debilitating neurological movement disorder characterized by involuntary contractions of the neck muscles, leading to abnormal head postures and significant pain. The condition significantly impairs quality of life and remains underdiagnosed, particularly in early stages or mild presentations. While the exact cause often remains unknown, cervical dystonia may be idiopathic or secondary to trauma, neurodegenerative conditions, or drug exposure.
DelveInsight's latest…

Atherosclerotic Cardiovascular Disease Market to Witness Robust Expansion Throug …
Atherosclerotic Cardiovascular Disease (ASCVD) remains a leading cause of morbidity and mortality worldwide, posing a substantial burden on healthcare systems despite advancements in prevention and management. Characterized by plaque buildup within arterial walls, ASCVD encompasses a spectrum of conditions including coronary artery disease, peripheral artery disease, and cerebrovascular disease. It significantly increases the risk of heart attacks, strokes, and cardiovascular death, especially among aging populations and individuals with comorbidities such…

Alpha-1 Antitrypsin Deficiency Market Set for Steady Growth Through 2034 - Marke …
Alpha-1 Antitrypsin Deficiency (AATD) is a rare but underdiagnosed genetic disorder that significantly increases the risk of developing chronic obstructive pulmonary disease (COPD), liver disease, and other systemic complications. Caused by mutations in the SERPINA1 gene, AATD results in deficient or dysfunctional alpha-1 antitrypsin (AAT), a key protein that protects tissues from enzyme damage, especially in the lungs.
DelveInsight's latest report, "Alpha-1 Antitrypsin Deficiency - Market Insight, Epidemiology, and Market Forecast…
More Releases for Fabry
Fabry Disease Market Expected to Rise Steadily throughout 2026
Fabry diseaseis also known as Anderson-Fabry disease and alpha-galactosidase A deficiency. It is a rare genetic disorder of lipid metabolism resulting from the deficient activity of the alpha-galactosidase A (a-Gal A) enzyme. The deficiency of the enzyme is caused by the alterations in the genes that instructs the cells to make alpha-galactosidase A (a-Gal A) enzyme. Fabry disease is known to cause variety of systemic symptoms and complications, one of…
Fabry Disease - Pipeline Review, H2 2017
ReportsWorldwide has announced the addition of a new report title Fabry Disease - Pipeline Review, H2 201 to its growing collection of premium market research reports.
Summary
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Fabry Disease - Pipeline Review, H2 2017, provides an overview of the Fabry Disease (Genetic Disorders) pipeline landscape.
Fabry disease is an inherited disorder. Fabry disease results from abnormal deposits of a particular fatty substance (called…
Fabry Disease Market show exponential growth by 2023
Fabry disease is an unusual, life-threatening situation with debilitating condition. Fabry disease is an uncommon X-linked inherited disorder. It is caused by the lack of the enzyme alpha galactosidase-A (a-GAL A). It is initiated by the absence of enzyme required to metabolize the lipids, fat-like substances like waxes, oils, and fatty acids. This disease is also known as alpha-galactosidase-A deficiency. Often there is a complete deficiency of a-Gal results in a severe form…
Fabry Disease Market | Analysis & Key Trends 2023
Fabry disease is an unusual, life-threatening situation with debilitating condition. Fabry disease is an uncommon X-linked inherited disorder. It is caused by the lack of the enzyme alpha galactosidase-A (a-GAL A). It is initiated by the absence of enzyme required to metabolize the lipids, fat-like substances like waxes, oils, and fatty acids. This disease is also known as alpha-galactosidase-A deficiency. Often there is a complete deficiency of a-Gal results in…
Fabry Disease - Pipeline Review, H1 2017
ReportsWorldwide has announced the addition of a new report title Fabry Disease - Pipeline Review, H1 2017 to its growing collection of premium market research reports.
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Fabry Disease - Pipeline Review, H1 2017, provides an overview of the Fabry Disease (Genetic Disorders) pipeline landscape.
Fabry disease is an inherited disorder. Fabry disease results from abnormal deposits of a particular fatty substance (called…
Fabry Disease Market Intelligence Report Offers Growth Prospects
Fabry diseaseis also known as Anderson-Fabry disease and alpha-galactosidase A deficiency. It is a rare genetic disorder of lipid metabolism resulting from the deficient activity of the alpha-galactosidase A (a-Gal A) enzyme. The deficiency of the enzyme is caused by the alterations in the genes that instructs the cells to make alpha-galactosidase A (a-Gal A) enzyme. Fabry disease is known to cause variety of systemic symptoms and complications, one of…