OpportunityAnalyzer: Fabry Disease – Opportunity Analysis and Forecast to 2024

Date: 2015-11

“OpportunityAnalyzer: Fabry Disease – Opportunity Analysis and Forecast to 2024


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 expected to grow to $1.25 billion at a Compound Annual Growth Rate (CAGR) of 6.3% over the 10-year period. The majority of sales in the 7MM in 2024 will come from the US, which will represent 44.2% 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 increasing 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 new treatments for Fabry disease, which focus on three main areas of research: chaperone therapies, substrate reduction therapies (SRTs), and combinations of their use with ERT.


Key Questions Answered

– 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 diverse range of symptoms that a patient may have when initially presenting with the disease. What are the main unmet needs in this market? Will the drugs under development fulfil the unmet needs in this market?
– 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?
– Key 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?

Key Findings

– One of the main drivers for the Fabry disease market growth 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 increasing awareness of Fabry disease among physicians.
– The first pharmacological chaperone for the treatment of Fabry disease, Amicus Therapeutics’ migalastat, will launch in the US and 5EU (France, Germany, Italy, Spain, and UK) in 2017, followed by its launch in Japan in 2020. Migalastat patient share is not only expected to be captured from existing patients receiving enzyme replacement therapy (ERT), 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, such as Amicus’ migalastat, it is expected that expansion of the Fabry disease market to include younger children will not occur.


– 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 forecast for ten years to 2024.
– Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the Fabry disease therapeutics market.
– Pipeline analysis: comprehensive data split across different phases, emerging novel trends under development, and detailed analysis of late-stage pipeline drugs.
– Analysis of the current and future market competition in the global Fabry disease therapeutics market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

Reasons to buy

– 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. Additionally a list of acquisition targets included in the pipeline product company list.
– Develop business strategies by understanding the trends shaping and driving the Fabry disease therapeutics market.
– Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the Fabry disease therapeutics market in 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.
– 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 strategic partnerships.”

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