AMT-191 Market Summary

Key Factors Driving AMT-191 Growth

1. Strong increases in α-Gal A enzyme activity

AMT-191 has demonstrated substantial increases in the deficient enzyme. In data reported in June 2026, involving 11 patients with follow-up ranging from three months to more than 18 months, α-Gal A activity increased in a dose-dependent manner. At the highest dose, activity ranged from 58.7 to 143.6 times the mean normal level. These findings support continued clinical development, although enzyme activity alone does not prove long-term clinical benefit.

2. Potential to reduce dependence on enzyme replacement therapy

Conventional Fabry disease treatment often involves lifelong intravenous ERT, generally administered every two weeks. AMT-191 aims to provide sustained enzyme production after a single infusion. By the June 2026 data update, all 11 treated patients had discontinued ERT under the study protocol. If longer-term studies confirm sustained disease control without ERT, this could become a major differentiator and adoption driver.

3. One-time gene therapy approach

AMT-191 uses an AAV5 vector with a liver-specific promoter to deliver a functional copy of the GLA gene. The approach is designed to enable the liver to produce α-Gal A continuously, potentially avoiding repeated enzyme infusions. This could reduce treatment burden and support long-term management, but the durability of benefit and need for additional treatment remain uncertain.

4. Stable disease-associated biomarker levels

In the February 2026 update, plasma lyso-Gb3—a biomarker associated with Fabry disease—remained stable after treatment across all dose cohorts, regardless of whether patients continued ERT. The June 2026 update reported continued stability. These findings provide supportive evidence that the observed enzyme expression was not accompanied by worsening of this biomarker during the reported follow-up, although longer observation and clinical outcome data are needed.

5. Rare-disease regulatory designations

AMT-191 received FDA Orphan Drug Designation in September 2024 and Fast Track designation in October 2024. These designations may facilitate regulatory communication and development for a rare condition with substantial unmet medical need. They do not guarantee approval, and the candidate must still demonstrate an acceptable benefit–risk profile.

AMT-191 Recent Developments

In February 2026, uniQure reported dose-dependent increases in α-Gal A activity in treated patients, with sustained elevations observed during follow-up; six of the 11 treated patients had discontinued ERT at the reported data cutoff. However, the company also reported liver-enzyme elevations that led to a pause in additional dosing in the mid- and high-dose cohorts pending further evaluation. In June 2026, updated preliminary data continued to show increased α-Gal A activity across dose levels, supporting further investigation of the therapy while safety monitoring remains essential.

“AMT-191 Sales Forecast, and Market Size Analysis – 2034” report provides comprehensive insights of AMT-191 for potential indication like Fabry’s disease in the 7MM. A detailed picture of AMT-191’s existing usage in anticipated entry and performance in potential indications in the 7MM, i.e., the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan for the study period 2020 –2034 is provided in this report along with a detailed description of the AMT-191 for potential indications. The AMT-191 market report provides insights about AMT-191’s sales forecast, mechanism of action (MoA), dosage and administration, as well as research and development including regulatory milestones, along with other developmental activities. Further, it also consists of historical and current AMT-191 performance, future market assessments inclusive of the AMT-191 market forecast analysis for potential indications in the 7MM, SWOT, analysts’ views, comprehensive overview of market competitors, and brief about other emerging therapies in respective indications. It also provides analysis of AMT-191 sales forecasts, along with factors driving its market.

AMT-191 Drug Summary

AMT-191 is an investigational gene therapy developed by uniQure for the treatment of Fabry disease, a rare, inherited X-linked lysosomal storage disorder caused by mutations in the GLA gene, resulting in deficient alpha-galactosidase A (α-Gal A) enzyme activity and the progressive accumulation of glycosphingolipids, including globotriaosylceramide (Gb3) and globotriaosylsphingosine (lyso-Gb3), in multiple organs. AMT-191 uses a recombinant adeno-associated virus serotype 5 (AAV5) vector containing a functional copy of the human GLA gene under the control of a proprietary, liver-specific promoter. Following a single intravenous infusion, the therapy is designed to deliver the genetic material to liver cells, enabling them to produce and release functional α-Gal A enzyme into circulation, which may facilitate substrate clearance and help reduce disease-related damage to the kidneys, heart and other affected tissues. By potentially providing sustained enzyme production, AMT-191 aims to reduce or eliminate the need for lifelong, regularly administered enzyme replacement therapy (ERT). The candidate is being evaluated in an open-label Phase I/II clinical trial to assess its safety, tolerability, enzyme activity and preliminary efficacy in adult patients with classic Fabry disease. The report provides AMT-191’s sales, growth barriers and drivers, post usage and approvals in multiple indications.

Scope of the AMT-191 Market Report

The report provides insights into:

• A comprehensive product overview including the AMT-191 MoA, description, dosage and administration, research and development activities in potential indication like Fabry’s disease.
• Elaborated details on AMT-191 regulatory milestones and other development activities have been provided in AMT-191 market report.
• The report also highlights AMT-191‘s cost estimates and regional variations, reported and estimated sales performance, research and development activities in potential indications across the United States, Europe, and Japan.
• The AMT-191 market report also covers the patents information, generic entry and impact on cost cut.
• The AMT-191 market report contains current and forecasted AMT-191 sales for potential indications till 2034.
• Comprehensive coverage of the late-stage emerging therapies for respective indications.
• The AMT-191 market report also features the SWOT analysis with analyst views for AMT-191 in potential indications.

Methodology

The AMT-191 market report is built using data and information sourced primarily from internal databases, primary and secondary research and in-house analysis by DelveInsight’s team of industry experts. Information and data from the secondary sources have been obtained from various printable and nonprintable sources like search engines, news websites, global regulatory authorities websites, trade journals, white papers, magazines, books, trade associations, industry associations, industry portals and access to available databases.

AMT-191 Analytical Perspective by DelveInsight

• In-depth AMT-191 Market Assessment

This AMT-191 sales market forecast report provides a detailed market assessment of AMT-191 for potential indication like Fabry’s disease in the seven major markets, i.e., the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan. This segment of the report provides current and forecasted AMT-191 sales data uptil 2034.

• AMT-191 Clinical Assessment

The AMT-191 market report provides the clinical trials information of AMT-191 for potential indications covering trial interventions, trial conditions, trial status, start and completion dates.

AMT-191 Competitive Landscape

The report provides Insights on competitors and marketed products within the domain, along with a summary of emerging products and their respective launch dates, posing significant competition in the market.

AMT-191 Market Potential & Revenue Forecast

• Projected market size for the AMT-191 and its key indications
• Estimated AMT-191 sales potential (AMT-191 peak sales forecasts)
• AMT-191 Pricing strategies and reimbursement landscape

AMT-191 Competitive Intelligence

• Number of competing drugs in development (pipeline analysis)
• AMT-191 Market positioning compared to existing treatments
• AMT-191 Strengths & weaknesses relative to competitors

AMT-191 Regulatory & Commercial Milestones

• AMT-191 Key regulatory approvals & expected launch timelines
• Commercial partnerships, licensing deals, and M&A activity

AMT-191 Clinical Differentiation

• AMT-191 Efficacy & safety advantages over existing drugs
• AMT-191 Unique selling points

AMT-191 Market Report Highlights

• In the coming years, the AMT-191 market scenario is set to change due to strong adoption, increased prescriptions and broader uptake in multiple immunological indications; which would expand the size of the market.
• The AMT-191 companies are developing therapies that focus on novel approaches to treat/improve the disease condition, assess challenges, and seek opportunities that could influence AMT-191’s dominance.
• Other emerging products for Fabry’s disease are expected to give tough market competition to AMT-191 and launch of late-stage emerging therapies in the near future will significantly impact the market.
• A detailed description of regulatory milestones, and developmental activities, provide the current development scenario of AMT-191 in potential indications.
• Analyse AMT-191 cost, pricing trends and market positioning to support strategic decision-making in the immunology landscape.
• Our in-depth analysis of the forecasted AMT-191 sales data uptil 2034 will support the clients in decision-making process regarding their therapeutic portfolio by identifying the overall scenario of AMT-191 in potential indications.

Key Questions

• What is the class of therapy, route of administration and mechanism of action of AMT-191? How strong is AMT-191’s clinical and commercial performance?
• What is AMT-191’s clinical trial status in each individual indications such as Fabry’s disease and study completion date?
• What are the key collaborations, mergers and acquisitions, licensing and other activities related to the AMT-191 Manufacturers?
• What are the key designations that have been granted to AMT-191 for potential indications? How are they going to impact AMT-191’s penetration in various geographies?
• What is the current and forecasted AMT-191 market scenario for potential indications? What are the key assumptions behind the forecast?
• What are the current and forecasted sales of AMT-191 in the seven major countries, including the United States, Europe (Germany, France, Italy, Spain) and the United Kingdom, and Japan?
• What are the other emerging products available and how are these giving competition to AMT-191 for potential indications?
• Which are the late-stage emerging therapies under development for the treatment of potential indications?
• How cost-effective is AMT-191? What is the duration of therapy and what are the geographical variations in cost per patient?