Fabry Disease – Opportunity Assessment and Forecast

Fabry Disease, or FD for short, refers to a rare, inherited X-linked lysosomal storage disease (LSD) whereby mutations in the galactosidase-α (GLA) gene encoding the lysosomal enzyme α-GAL A. Due to a deficiency or absence of GLA, an enzyme substrate, primarily globotriaosylceramide (Gb3) accumulates throughout the blood vessels, other tissues, and organ. Clinical manifestations include, but are not limited to, renal, cardiac and cerebrovascular complications, as well as pain, angiokeratoma, gastrointestinal disturbance, an overall reduce quality of life and early death. Many of the current therapies for FD are unable to alter the disease’s natural course entirely, with α-GAL A activity remaining below optimal over time, resulting in a reduction of symptoms but some continued complications in the long-term.

Chiesi Farmaceutici and Protalix Biotherapeutics’ pegunigalsidase alfa and Idorsia Pharmaceuticals’ lucerastat are the only late-stage treatment modalities in the FD pipeline. There are several gene therapies in the phase I/II development stage and are either adeno-virus variant or lenti-viral variant-based therapies which deliver a transgene that ensure the production of the lysosomal enzyme α-GAL A. These therapies are likely to be a driver of market growth in the later stages of the forecast period, and early clinical data has indicated these therapies are high efficacious and single-dose, with the potentiality of being curative.

Key Questions Answered

– How will the Fabry Disease market landscape in the 2MM (US and Japan) change from 2020-2030?

– What are the most promising late-stage pipeline products for Fabry Disease?

– How do the clinical and commercial attributes of late-stage pipeline products compare with one another, and against existing treatment options?

– What are the remaining unmet needs in Fabry Disease?

– What drivers and barriers will affect Fabry Disease treatment sales in the 2MM over the forecast period?

Scope

Overview of Fabry Disease including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and current management strategies.

Topline Fabry Disease market revenue from 2020-2030. Annual cost of treatment and major pipeline product sales in this forecast period are included.

Key topics covered include current treatment options, unmet needs and opportunities, and the drivers and barriers affecting Fabry Disease treatment sales in the 2MM.

Pipeline analysis: comprehensive data split across different phases, emerging novel trends under development, synopses of innovative early-stage projects, and detailed analysis of late-stage pipeline products.

Analysis of the current and future market competition in the global Fabry Disease market. Insightful review of the key industry drivers, constraints, and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

Key Highlights

During the 10-year forecast period, there is one major pipeline product on track to launch, driving a forecast growth in the 2MM from $820.15M in 2020 to $985.81M in 2030.

The FD market is expected to have several novel entrants over the forecast period. These novel oral, ERT and gene therapies will provide significant opportunity to address the need for long-term highly efficacious therapies.

The uptake of therapies in the FD pipeline by physicians will be driven by clinical data demonstrating efficacy and ease of administration, as well as price. If a gene therapy is able show single-dose curative potential at a justifiable price, it will likely achieve significant uptake toward the end of the forecast.

However, The pricing of novel therapies is likely to be very high ($300,000-1,000,000) depending on the therapy type, so novel therapies are unlikely to immediately receive reimbursement or insurance coverage. The cost-to- benefit ratio and QALY added are significant factors to achieving reimbursement and coverage. So far, this has not yet been determined due to several late-stage trials that have not yet been conducted.

Reasons to buy

The report will enable you to:

Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.

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Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the Fabry Disease in the future.

Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors.

Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.

Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments, and strategic partnerships.

Companies mentioned

Sanofi Genzyme

Shire Pharmaceuticals (Takeda)

Amicus Therapeutics

Chiesi Famaceutici SpA

Idorsia Pharmaceuticals

4D Molecular Therapeutics Inc.

AVROBIO Inc.

Sangamo Therapeutics Inc.

Table of Contents

1 Executive Summary

2 Disease Overview

2.1 Definition of Fabry Disease

2.2 Fabry SWOT Analysis

2.3 Etiology and Pathophysiology

3 Epidemiology

3.1 Diagnosed Prevalent Cases of FD, 2020-2030

3.2 Diagnosed Prevalent Cases of FD by Specific Type, 2020

3.3 Sources and Methodology

3.4 Diagnosis of Fabry Disease – Treatable Population

4 Current Treatment Options

4.1 Treatment Paradigm

4.2 Marketed Products

4.3 Product Profile: Genzyme and Sanofi's Fabrazyme

4.4 Product Profile: Shire's Replagal

4.5 Product Profile: Amicus Therapeutics' Galafold

5 Unmet Needs and Opportunities

5.1 Unmet Needs in FD

5.2 Earlier Disease Diagnosis

5.3 Fabry Disease Treatments With Improved Efficacy

5.4 Lower cost of Fabry Disease treatments

5.5 Widespread Availability of Home-based Infusion to Improve Compliance

5.6 Fabry Disease Treatment With Improved Drug Clearance

6 Pipeline Assessment

6.1 Fabry Disease: Pipeline Overview

6.2 Leading Pipeline Agents

6.3 Product Profile: pegunigalsidase alfa

6.4 Product Profile: lucerastat

6.5 Product Profile: 4D-310

6.6 Product Profile: AVR-RD-01

6.7 Product Profile: ST-920

6.8 Pipeline Products – Review Designations

6.9 Fabry Disease: Clinical Trials Overview

7 R&D Strategies

7.1 Trends in Clinical Trial Design in Fabry Disease

7.2 Trends in Deal-Making in Fabry Disease

8 Market Outlook

8.1 Fabry Disease Market Growth

8.2 Fabry Disease Market Forecast

8.3 Market Drivers and Barriers

9 Appendix

9.1 Primary Research: KOL Information

9.2 About the Authors

10 Contact Us

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