Estimated the 2014 sales for the Gaucher Disease (GD) market at approximately $875m across the 7MM (US, France, Germany, Italy, Spain, UK, and Israel). The US market contributed the majority of these sales, generating an estimated $482m. By the end of the forecast period in 2024, GD sales are expected to increase to $1.16 billion at a Compound Annual Growth Rate (CAGR) of 2.9%. The majority of these sales will continue to come from the US, which will represent approximately 56.2% of the GD market in the 7MM in 2024.
GlobalData forecasts an increase in the number of diagnosed prevalent cases of GD, and therefore, an increase in the treated GD population. Improved diagnosis will be driven by increasing physician education and awareness of GD and its signs and symptoms. Despite the anticipated increase in the number of diagnosed prevalent cases of GD over the forecast period, it is expected that a large portion of the GD population will remain undiagnosed. This is due to continued late diagnosis and misdiagnosis of GD as a result of the heterogeneous manifestations of the disease. The sparsely populated GD pipeline, with no drugs in late-stage development, will further hinder the growth of the GD market.
Highlights
Key Questions Answered
- Although the use of enzyme replacement therapies (ERTs) and substrate reduction therapies (SRTs) has significantly changed the natural history of GD, with a marked decrease in morbidity, especially in Type 1 GD patients, GD market is still marked by the presence of a number of unmet needs in current treatments. What are the main unmet needs in this market? Will the drugs under development fulfil the unmet needs of the GD market?
- The late-stage GD pipeline is very sparsely populated and there no drugs in late-stage development. Nevertheless, the GD market will experience substantial growth. What are the main drivers of this growth during 2014-2024? Which of the marketed drugs will have the highest peak sales at the highest CAGR, and why?
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Key Findings
- GlobalData forecasts an increase in the number of diagnosed prevalent cases of GD, and therefore, an increase in the treated GD population. Improved diagnosis will be driven by increasing physician education and awareness of GD and its signs and symptoms. This is largely attributable to Genzyme's and Shire's continued efforts to raise awareness of rare diseases through organized events and established websites, and by sponsoring patient registries.
- Genzyme's recently approved oral substrate reduction therapy (SRT), Cerdelga (eliglustat), is more effective and safer than the other oral SRT, Zavesca (miglustat), and therefore will be used more commonly as an oral alternative to IV ERT in adult Type 1 GD patients. As Cerdelga is more expensive than the other GD therapies, it is expected to drive the growth of the GD market.
- None of the therapies fulfilled the greatest unmet need in GD treatment — need for the drugs that can treat the neurological manifestations of the disease. One of the main R&D strategies in the GD space is the focus on the development of new SRT formulations and pharmacological chaperones (PCs) that can cross the blood brain barrier and thereby improve the neurological manifestations of GD.
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Scope
- Overview of GD, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.
- Annualized GD therapeutics market revenue, annual cost of therapy and treatment usage pattern data 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 GD 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 GD 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
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. Additionally a list of acquisition targets included in the pipeline product company list.
- Develop business strategies by understanding the trends shaping and driving the GD therapeutics market.
- Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the GD 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 GD therapeutics market from 2014-2024.
- Organize your sales and marketing efforts by identifying the market categories
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