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Opportunity Analyzer: Fabry Disease - Opportunity Analysis and Forecast to 2024

Opportunity Analyzer: Fabry Disease - Opportunity Analysis and Forecast to 2024

  • August 2015
  • 133 pages
  • ID: 3358865
In this report:
By the end of the forecast period in 2024, Fabry disease sales are expected to grow to $##.## billion at a Compound Annual Growth Rate (CAGR) of ##.##% over the ##-year period.
The US is the most lucrative Fabry disease market, and is forecast to generate $##m in 2024, while sales in the ##EU and Japan are forecast to reach $##m and $##m, respectively, during the same year.

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Opportunity Analyzer: Fabry Disease - Opportunity Analysis and Forecast to 2024

Brief

GlobalData estimates the 2014 sales for the Fabry disease market at approximately $682m across the 7MM. The US contributed the majority of these sales, generating an estimated $295m. By the end of the forecast period in 2024, Fabry disease sales are anticipated to grow to ~$1.3B at a Compound Annual Growth Rate of 6.30 percent over the 10-year period. The majority of sales in the 7MM in 2024 will come from the U.S., which will represent 44.20 percent of the market. GlobalData expects an increase in the number of newly diagnosed cases of Fabry disease, and consequently in the number of treatable Fabry patients, as a result of growing awareness of Fabry disease among physicians. ERT is now well established in the treatment of Fabry disease; however, there still remains concern about its efficacy, tissue penetrance, and intravenous administration. As a result, alternative approaches are being investigated to advance additional treatments for Fabry disease, which focus on three principal areas of research: chaperone therapies, substrate reduction therapies, and combinations of their use with ERT.

Key Findings

Key questions & answers

- At present, patient registries demonstrate a long delay between onset of initial symptoms and a diagnosis, which can span between 10 to 20 years. This is due the condition being very rare, the lack of awareness of the disease among physicians, and the different range of symptoms that a patient may have when initially presenting with the disease. What are the principal unmet needs in this industry? Will the drugs under development fulfil the unmet needs in this sector?

- Since the approval of Fabrazyme and Replagal in the EU in 2001, no other drugs have been approved for the treatment of Fabry disease. Will the pipeline drugs in development change the treatment landscape for Fabry disease and attain high sales revenues during 2014-2024?

- Leading opinion leaders interviewed by GlobalData believe the biggest opportunity lies with combination therapies, to improve drug delivery and increase drugs' efficacy,. How will these changes impact the growth of the future market?

Main highlights

- One of the principal drivers for the Fabry disease market increase will be an increase in the number of newly diagnosed cases of Fabry disease, and consequently in the number of treatable Fabry patients, as a result of rising awareness of Fabry disease among physicians.

- The first pharmacological chaperone for the treatment of Fabry disease, Amicus Therapeutics' migalastat, will launch in the U.S. and 5EU (France (FR), Germany (DE), Italy (IT), Spain (ES), and UK) in 2017, followed by its launch in Japan (JP) in 2020. Migalastat patient share is not only anticipated to be captured from existing patients receiving enzyme replacement therapy, but also treatment-naïve patients who have not previously been considered suitable for ERT.

- There is a lack of suitable treatments available for young children with Fabry disease. ERT requires intravenous infusions every two weeks; hence, until orally available treatment options become available, including Amicus' migalastat, it is expected that expansion of the Fabry disease market to include younger children will not occur.

Overview

- Overview of Fabry disease, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.

- Annualized Fabry disease therapeutics market revenue, annual cost of therapy and treatment usage pattern data from from 2014 and estimate for ten years to 2024.

- Key topics covered include tactical competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the Fabry disease therapeutics market.

- Pipeline analysis: exhaustive data split across distinct phases, emerging novel trends under development, and in-depth analysis of late-stage pipeline drugs.

- Analysis of the current and future market competition in the overall Fabry disease therapeutics market. Insightful review of the top industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

Great reasons to purchase

- 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 strong pipeline. Additionally a list of acquisition targets included in the pipeline product company list.

- Develop strategic management by understanding the trends shaping and driving the Fabry disease therapeutics market.

- Drive revenues by understanding the main trends, innovative products and technologies, market segments, and firms likely to impact the Fabry disease therapeutics market in future.

- Specified effective sales and marketing strategies by understanding the challenging landscape and by analysing the performance of different competitors.

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

- Track drug sales in the 7MM Fabry disease therapeutics market from 2014-2024.

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

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