Mucopolysaccharidosis Therapeutics ??? Pipeline Assessment and

1. Mucopolysaccharidosis Therapeutics - PipelineAssessment and Market Forecasts to 2017

2. The Mucopolysaccharidosis (MPS) Therapeutics Market is Forecast to Show Steady Growth Until 2017 GlobalData’s analysis revealed that the global Mucopolysaccharidosis (MPS) market was worth $0.65 billion in 2010. It is forecast to grow at a Compound Annual Growth Rate (CAGR) of 9.6% for the next seven years, to reach $1.24 billion by 2017. This is primarily attributed to the increase in the prevalence of the disease and pipeline molecules targeting the MPS subtypes where there were no approved treatment options available. A weak clinical pipeline coupled with high unmet need in the MPS therapeutics market will lead to the steady growth in the market. Current MPS Market is Weak due to the Presence of Few Companies and a Limited Number of Treatment Options GlobalData’s analysis of the current competitive landscape for MPS drugs revealed that the competition is weak. Although MPS is a rare disease and a niche indication, there are only three FDA (Food and Drug Administration) approved drugs on the market and target only specific types of MPS: Aldurazyme (laronidase) for MPS I, Elaprase (idursulfase) for MPS II and Naglazyme (galsulfase) for MPS VI. No drug therapy is available for MPS III, IV and VII. All of these therapies are enzyme replacement therapies, which replace the deficient enzyme and target the underlying cause of the disease, but do not cure the disease.

3. These therapies halt the progression of the disease as long as the therapy is ongoing. Stem cell transplant is another treatment option currently used only for MPS I, VI and VII. Based on the availability of limited treatment options for specific types of MPS, the current competition in the MPS therapeutic market is characterized as weak. The key companies operating in the MPS market are BioMarin Pharmaceuticals Inc and Genzyme (Sanofi-Aventis) and Shire Plc. There are no approved drugs or therapies for the other subtypes of MPS. The MPS Therapeutics Market has High Unmet Need The global MPS therapeutics market is attractive, with high unmet need. This unmet need in the market is 68%, valued at $0.44 billion. This is due to limited treatment options, high cost of therapy, poor diagnosis, moderate safety and efficacy profile of the marketed drugs and no approved drug treatment for MPS III, IV and VII. The MPS market offers significant opportunities for newer cost effective treatment options and therapies targeting other MPS subtypes. For further details, please click or add the below link to your browser: http://www.globaldata.com/reportstore/Report.aspx?ID=Mucopolysaccharidosis-Therapeutics-Pipeline-Assessment-and-Market-Forecasts-to-2017

4. Weak Clinical Pipeline with only Three Molecules in Clinical Development The MPS pipeline is weak, with only three molecules in clinical development stages. Of these, two are first-in-class molecules, for the treatment of MPS III and MPS IV. These drugs have displayed encouraging safety and efficacy profiles during clinical development. BMN-110 is currently in Phase III, HGT-1410 and HGT-2310 are in Phase II. The three molecules are all enzyme replacement therapies and are targeting the underlying cause of the disease. The first-in-class molecules are BMN-110 (BioMarin Pharmaceuticals Inc) for the treatment of MPS IV and HGT-1410 (Shire Plc) for MPS III. Currently, marketed drugs are not targeting MPS III and IV. HGT-2310 (Shire Plc) is an extension of currently approved drug Elaprase, this product has an advanced delivery mechanism and is targeting MPS II. Future Implications for the MPS Therapeutics Market BioMarin Pharmaceuticals Inc, Genzyme (Sanofi-Aventis) and Shire Plc are likely to continue their strong position in the market. This is largely due to the fact that these companies have first-in-class drugs in the pipeline. The new drugs are targeting MPS III and IV and these companies are already marketing drugs for MPS I, II and VI. The current MPS market has high unmet need and there is a huge opportunity for newer treatment options to enter the market that could register double digit growth.

5. GlobalData, the industry analysis specialist, has released its new report, “Mucopolysaccharidosis Therapeutics - Pipeline Assessment and Market Forecasts to 2017”. The report is an essential source of information and analysis on the global mucopolysaccharidosismarket. The report identifies the key trends shaping and driving the global mucopolysaccharidosismarket. The report also provides insights on the prevalent competitive landscape and the emerging players expected to significantly alter the market positioning of the current market leaders. Most importantly, the report provides valuable insights on the pipeline products within the global mucopolysaccharidosis 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. For further details, please click or add the below link to your browser: http://www.globaldata.com/reportstore/Report.aspx?ID=Mucopolysaccharidosis-Therapeutics-Pipeline-Assessment-and-Market-Forecasts-to-2017 Visit our report store: http://www.globaldata.com For more details contact: pressreleases@globaldata.com

6. For more details contact: pressreleases@globaldata.com North America: +1 646 395 5477 Europe: +44 207 753 4299 +44 1204 543 533 Asia Pacific: +91 40 6616 6782