Sickle Cell Anemia Therapeutics - Pipeline Assessment and Market Forecasts to 2018

Dec. 15, 2011 - PRLog -- GlobalData valued the global (to include the US, the UK, France, Germany, Italy, Spain and Japan) Sickle
Cell Anemia therapeutics market at $33m in 2010 and forecasts it to grow at a Compound Annual Growth Rate (CAGR) of 18.9% to reach $130m in 2018. There are no approved branded drugs in the sickle cell anemia market. The Phase II pipeline has two potential novel molecules GMI-1070 (orphan drug and fast track status by US Food and Drug Administration (FDA)) and HQK-1001 (orphan drug in the US and Europe).These drugs are expected to be launched in the forecast period. GMI-1070 is expected to replace the use of hydroxyurea in adolescents and adults in the near future. However, the market is expected to have slow growth until 2018.

Current competition in the sickle cell anemia therapeutics market is weak. The current market is weak due lack of approved drugs in the market. GlobalData anticipates that the only approved drug hydroxyurea (for treatment of adult patients) despite being generic would remain the leader in the sickle cell anemia treatment drugs market alongwith Penicillin V (off-label drug) used for the treatment of pediatric patients. Novel drugs in Phase II of the pipeline, if launched, could depict improved efficacy, safety and patient compliance than the currently available options.

There is currently no promising molecule under Phase III clinical development in the sickle cell anemia pipeline. However, in the early stage of development (Phase II) there are first-in-class (FIC) molecules such as Varespladib sodium (A-001) from Anthera Pharmaceuticals and GMI-1070 from Glyco Mimetics Inc/Pfizer which have been granted orphan drug designation and fast track status by the US FDA. HQK-
1001 of HemaQuest Pharmaceuticals in Phase II has also been granted orphan drug status in the US and
Europe. The launch of these orphan drugs can provide treatment options for treating the complications of sickle cell anemia. There are no molecules which target the disease directly. Hence, there is a requirement for novel molecules with enhanced efficacy, safety and patient compliance which could offer a cure for patients with sickle cell anemia.

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GlobalData revealed that the current competition in the sickle cell anemia therapeutics market is weak, and the available treatment options are not adequately meeting the market demand in terms of curability. Hydroxyurea in the sickle cell anemia therapeutics market displays moderate efficacy, a low safety profile and variable patient compliance. The chart below shows the efficacy and safety profiles of hydroxyurea used for the treatment of adults. The unavailability of approved drugs with improved efficacy and safety profiles increases the overall unmet need in the market.

GlobalData, the industry analysis specialist, has released its new report, “Sickle Cell Anemia Therapeutics - Pipeline Assessment and Market Forecasts to 2018”. The report is an essential source of information and analysis on the global sickle cell anemia market. The report identifies the key trends shaping and driving the global sickle cell anemia market. It also provides insights on the prevalent competitive landscape and the emerging players expected to significantly alter the positioning of current market leaders. Most importantly, the report provides valuable insights on the pipeline products within the global sickle cell anemia sector. This report is built using data and information sourced from proprietary databases, primary and secondary research and in-house analysis by GlobalData’s team of industry experts.

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