Fabry Disease Market Outlook: 6 Upcoming Therapies Set to Influence the Future Market Landscape | DelveInsight
The emerging Fabry disease market landscape holds a diverse range of therapeutic alternatives for treatment, including Venglustat (Sanofi Genzyme), Lucerastat (Idorsia Pharmaceuticals), Isaralgagene civaparvovec (ST-920; Sangamo Therapeutics), AMT-191 (UniQure), AL1211 (AceLink Therapeutics), GT-GLA-S03 (Glafabra Therapeutics), and others in different lines of treatment.
LAS VEGAS, Oct. 1, 2026 /PRNewswire/ -- The primary treatment approaches for Fabry disease include Enzyme Replacement Therapies (ERTs) and pharmacological chaperone therapy. Currently marketed therapies, such as agalsidase beta (FABRAZYME), agalsidase alfa (REPLAGAL), migalastat (GALAFOLD), and pegunigalsidase alfa-Iwxj (ELFABRIO), offer a range of treatment options aimed at addressing the underlying enzyme deficiency, managing disease manifestations, and slowing disease progression.
The Fabry disease pipeline comprises several promising mid- and late-stage candidates, including Substrate Reduction Therapies (SRTs) and gene therapies. Among the notable investigational therapies are venglustat, developed by Sanofi Genzyme, and ST-920, developed by Sangamo Therapeutics, which have demonstrated encouraging efficacy and safety profiles in clinical development. In addition, other pipeline candidates, including lucerastat from Idorsia Pharmaceuticals and AMT-191 from uniQure, are being evaluated and could potentially reach the market during the forecast period.
Emerging treatment modalities, particularly gene therapies, have the potential to transform Fabry disease management by providing more durable therapeutic benefits. At the same time, increasing disease awareness and advances in diagnostic technologies are expected to support earlier detection, timely treatment initiation, and improved patient outcomes.
Aparna Thakur, Project Manager of Forecasting & Analytics at DelveInsight, said that ST-920 (Sangamo Therapeutics) is a potential gene therapy in the development of Fabry disease, with its expected approval by Q2 2026 in the US. Thakur further said that among all emerging therapies, Idorsia Pharmaceuticals' Lucerastat is in the late stage of development, with its expected approval by 2030 in the US.
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Reflecting continued therapeutic innovation and significant unmet medical needs, DelveInsight estimates that the Fabry disease market across the seven major markets, comprising the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan, was valued at approximately USD 1.7 billion in 2025. The market is projected to expand at a significant CAGR during the forecast period through 2036, driven by the adoption of enzyme replacement therapies and pharmacological chaperone therapy, the development of next-generation and longer-acting treatments, increasing disease awareness and diagnosis, and ongoing efforts to address the limitations of existing therapies, including infusion burden, immunogenicity, and the need for more effective disease control.
Below, we highlight promising emerging therapies poised to reshape the future of the Fabry disease market.
Sangamo Therapeutics's Isaralgagene civaparvovec (ST-920)
Alpha-galactosidase replacements
ST-920 is a liver-tropic recombinant adeno-associated virus (rAAV) 2/6 vector designed to deliver the cDNA encoding human alpha-galactosidase A through a single intravenous infusion. The therapy is intended to introduce a functional copy of the GLA gene into liver cells, enabling them to produce functional alpha-galactosidase A and potentially address the underlying enzyme deficiency associated with Fabry disease.
In February 2026, Sangamo Therapeutics presented detailed data from the registrational Phase I/II STAAR study through four platform and poster presentations at the 22nd Annual World Symposium in San Diego, California. As of May 2026, Sangamo Therapeutics was advancing its rolling Biologics License Application (BLA) submission and expected to complete the filing as early as summer 2026. The company was also continuing discussions regarding a potential commercialization partnership for ST-920 in Fabry disease.
In Europe, Sangamo Therapeutics is engaged in discussions with the European Medicines Agency (EMA) regarding the regulatory pathway for ST-920. The therapy has received EMA PRIME eligibility and UK Medicines and Healthcare products Regulatory Agency (MHRA) Innovative Licensing and Access Pathway (ILAP) status, supporting its potential accelerated development and regulatory review.
Idorsia Pharmaceuticals' Lucerastat
Glucosylceramide synthase inhibitor
Lucerastat is an investigational oral substrate reduction therapy being developed for the treatment of Fabry disease, with its mechanism designed to be independent of α-galactosidase A (α-Gal A) activity, GLA mutation status, and prior enzyme replacement therapy (ERT). The therapy inhibits glucosylceramide synthase, thereby decreasing the production of glycosphingolipids, including globotriaosylceramide (Gb3), which accumulates as a result of deficient α-Gal A activity in patients with Fabry disease. In February 2026, Idorsia Pharmaceuticals announced the design of its FDA-approved Phase III registration program to evaluate lucerastat in patients with Fabry disease.
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Sanofi Genzyme's Venglustat
Glucosylceramide synthase inhibitor
Venglustat is a novel investigational oral glucosylceramide synthase inhibitor (GCSi) designed to cross the blood-brain barrier, enabling it to exert effects within the central nervous system. By inhibiting the abnormal accumulation of glycosphingolipids (GSLs), Venglustat has the potential to slow disease progression in certain disorders by addressing the underlying cellular dysfunction and pathophysiological consequences associated with GSL buildup. GSLs are essential cellular components; however, their abnormal accumulation has been linked to the development and progression of several rare diseases. Venglustat has previously received orphan drug designation in the European Union, the United States, and Japan for its potential use in Gaucher disease type 3 (GD3) and Fabry disease. Additionally, the US FDA has granted Venglustat Fast Track designation for its potential development in both GD3 and Fabry disease.
UniQure's AMT-191
Alpha-galactosidase replacements; Gene transference
AMT-191 is an investigational AAV5-based gene therapy designed to deliver a functional GLA transgene to the liver, enabling the production of GLA protein. In patients with Fabry disease, pathogenic variants in the GLA gene result in deficient GLA enzyme activity, leading to the progressive accumulation of lipids across multiple cell types and ultimately causing a multisystem disorder. AMT-191 offers a novel potential treatment approach based on a single administration.
Preclinical studies have demonstrated that AMT-191, driven by a proprietary liver-specific promoter, can achieve substantial reductions in (lyso)Gb3 levels, along with cross-correction and phenotypic improvement in mouse models. Subsequent biodistribution and toxicology studies in non-human primates (NHPs) further supported the favorable safety profile of AMT-191 and confirmed GLA expression.
In February 2026, uniQure announced updated preliminary safety and exploratory efficacy data from 11 patients enrolled in its Phase I/IIa clinical trial evaluating AMT-191 as an investigational AAV gene therapy for the treatment of Fabry disease.
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AceLink Therapeutics' AL1211
Glucosylceramide synthase inhibitor
AL1211 is a glucosylceramide synthase (GCS) inhibitor that is designed to offer unique and potentially superior properties compared with other GCS inhibitors currently approved or under development. It demonstrates greater potency and a favorable off-target activity profile relative to other GCS inhibitors. AL1211 also exhibits excellent tissue penetration in organs affected by Fabry disease, including the heart and kidneys. Importantly, its low brain penetration is expected to enable effective treatment of peripheral organs while minimizing concerns regarding potential adverse effects in the brain.
AL1211 may provide a more convenient and effective treatment option compared with enzyme replacement therapy (ERT), which requires burdensome intravenous infusions and may have limited benefits because of inadequate tissue penetration.
The company has completed IND-enabling studies supporting the clinical development of AL1211, as well as Phase I clinical trials in Australia and China. Both studies demonstrated consistent and robust pharmacokinetic and pharmacodynamic responses, with increasing AL1211 exposure associated with greater reductions in plasma GL1 and GL3 levels. Importantly, AL1211 was found to be safe and well tolerated in both studies, with no serious adverse events observed at any of the dose levels evaluated.
The company has received regulatory clearance from both the National Medical Products Administration (NMPA) in China and the U.S. Food and Drug Administration (FDA) to initiate Phase II clinical trials of AL1211 in patients with Fabry disease. These Phase II studies are currently underway.
Glafabra Therapeutics' GT-GLA-S03
Alpha-galactosidase replacements; Gene transference
GT-GLA-S03 is being developed to replace the infusion burden associated with conventional enzyme replacement therapy with an outpatient procedure designed to provide durable, steady-state enzyme expression from the patient's own gene-modified cells. The therapy utilizes a reduced-intensity, non-myeloablative, single-agent melphalan conditioning regimen instead of the myeloablative busulfan regimen that limited earlier stem-cell gene therapy programs. In a clinical pilot study conducted by the company's co-founders, four of five patients completed the conditioning regimen as a same-day outpatient procedure.
As an autologous approach that does not use a viral capsid, GT-GLA-S03 is designed to allow re-administration as enzyme expression declines. This approach also avoids the pre-existing anti-capsid antibody exclusions associated with AAV-based gene therapies, which can render approximately 40% of adults ineligible and are generally not repeatable. GT-GLA-S03 is being developed for potential re-administration at intervals of five years or longer rather than as a one-time treatment.
In March 2026, Glafabra Therapeutics announced that the U.S. Food and Drug Administration (FDA) had granted Orphan Drug Designation (ODD) to GT-GLA-S03, the company's lead cell therapy candidate for the treatment of classic Fabry disease.
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Source: Fabry Disease Market Report
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