Fabry Disease Market Sales Was about $682m in 2014

Date: 2015-11-04

It 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.

– 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.

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