Gaucher’s Disease Therapeutics Market Forecast to Display High Growth until 2018

Wednesday 22 February 2012, Amsterdam

The report is an essential source of information on the global Gaucher’s disease therapeutics market, and provides analysis of a number of key areas. The report identifies the key trends shaping and driving the global Gaucher’s disease therapeutics market and provides insights into the current competitive landscape and emerging companies expected to significantly alter the market positioning of current market leaders. Most importantly, the report provides valuable insights into the pipeline products within the global Gaucher’s disease sector. This report is built using data and information sourced from proprietary databases, primary and secondary research and in-house analysis by a team of industry experts.

Gaucher’s Disease Therapeutics Market Forecast to Display High Growth until 2018

The estimates that the global Gaucher’s disease therapeutics market was worth $851.2m in 2010, after increasing at a Compound Annual Growth Rate (CAGR) of 3.8% during 2005 - 2010. The market is expected to record a CAGR of 7.4% from 2010 - 2018, to value $1,512.2m by 2018. Enzyme Reduction Therapy (ERT) and Substrate Replacement Therapy (SRT) are the two currently approved classes for the treatment of Gaucher’s disease. ERT includes Cerezyme (imiglucerase) from Genzyme (a Sanofi company) and Vpriv (velaglucerase alfa) from Shire Pharmaceuticals. Zavesca (miglustat) is an orally administered SRT drug approved in Europe and the US in November 2002 and July 2003 respectively. Cerezyme gained orphan drug designation was approved in the US in May 1994 Vpriv has not been launched in Japan, meaning that there is only one drug available in the country for the treatment of Gaucher’s disease. Genzyme Corporation has been the market leader in the Gaucher’s disease therapeutics market since 1991 and Cerezyme has enjoyed a monopoly for the last two decades. Supply shortages in June 2009 due to viral contamination at the Allston plant in the US allowed Shire’s drug Vpriv to gain a share of the market share in Gaucher’s disease therapeutics market. The production of Cerezyme has not been fully resumed until now.

The analysis found that the significant growth recorded in Gaucher’s disease therapeutics market during 2005-2010 can be primarily attributed to increased sales of Cerezyme as well as the increased annual cost of therapy and increasing disease awareness among physicians and patients. The Gaucher’s disease therapeutics market is expected to grow at a high rate during the forecast period due to the expected launch of novel molecules Uplyso (taliglucerase alfa) eliglustat tartrate and ISU302. These late stage pipeline molecules have improved safety and efficacy profiles, and are more cost effective than current therapeutic options, meaning that accessibility and affordability will increase. These factors are expected to drive the future market growth.

See figure: Gaucher’s Disease Therapeutics Market, Global, Revenue and Forecasts ($m), 2005 - 2018

Significant Unmet Need in Gaucher’s Disease Therapeutics Market

The analysis suggests that the Gaucher’s disease therapeutics market has significant unmet need, not only in terms of the limited number of therapeutic options available for type 1 Gaucher’s disease, but also a lack of effective treatment options for severe brain damage cases that can occur in type 2 and 3 Gaucher’s patients. Although ERT is effective in the treatment of type 1 Gaucher’s disease, there is insufficient evidence that ERT or SRT improve neurological symptoms in type 2 and 3 Gaucher’s patients. The lack of an early diagnosis may lead to further disease progression and neurological manifestations, ultimately worsening patient quality of life. The annual cost of therapy with Cerezyme exceeds $300,000 and so it is likely that many patients do not have access to medication. As well as early diagnosis, patient adherence to treatment has also been found to present a major unmet need. For example, a serious complication of Gaucher’s disease is bone disease. If left untreated, it can result in pain, permanent damage to the bone and disability. There is significant potential for the introduction of novel therapies with improved safety and efficacy profiles in the Gaucher’s disease therapeutics market. The expected launch of late stage pipeline molecules such as Uplyso, eliglustat tartrate and ISU302 with better safety and efficacy profiles than existing approved drugs will fulfill the unmet need.

Strong Pipeline for the Gaucher’s Disease Therapeutics Market

The analysis shows that the Gaucher’s disease therapeutics pipeline is strong with nine molecules in various Phases of clinical development. One molecule is currently filed with the US Food and Drug Administration (FDA) and European Medicines Agency (EMEA). Two molecules are in Phase III of clinical development, one molecule is in Phase II of clinical development and five molecules are in the preclinical stage. There are no molecules in Phase I. Recently, late stage pipeline molecule Uplyso has been granted Temporary Authorization (ATU) for use by the French Health Products and Safety Agency for the treatment of Gaucher’s patients. The ATU is a special regulatory mechanism which enables the use of non approved products in France when there is a genuine public health need. Eliglustat tartrate is in late Phase III of clinical development and will be the second SRT drug for the treatment of Gaucher’s disease if approved. Another molecule, ISU302, is also in Phase III and will be the first bio-similar in the Gaucher’s disease therapeutics market if approved.

Overall, the analysis suggests that the pipeline for the Gaucher’s disease therapeutics market is strong and that the two promising pipeline candidates have the potential to compete with the currently approved products in addressing unmet market need.