Up-to-date news on the campaign to Gauchers Association. http://www.gaucher.org.uk HG Shire Announces FDA Approval of VPRIVTM (velaglucerase alfa for injection) for the Treatment of Type 1 Gaucher Disease Click here to see full press release: Press Release http://www.gaucher.org.uk/news.php?id=72 Latest Cerezyme Update - 17th February 2010: Due to several factors including to improve manufacturing quality systems and perform preventative maintenance and upgrades at the Allston plant, it has been announced that current supply of Cerezyme is less than originally anticipated. To more consistently manage the resupply of Cerezyme to patients in approximately 100 countries and reduce the interruptions in shipping that occur in the absence of inventory, Genzyme will work to immediately build a small inventory buffer. This buffer will allow a more predictable schedule for Cerezyme delivery through the remainder of 2010 and help avoid the challenges many physicians and patients have experienced in scheduling infusions. To build this inventory buffer, the company will ship 50 percent of demand for an eight-week period beginning the week of February 22, 2010. Genzyme is working closely with physicians and patient organizations to manage this temporary initiative. We encourage you to discuss any concerns you may have with your doctor at your treating centre. Call us on 01453 549231 if you are worried or have any further questions http://www.gaucher.org.uk/news.php?id=71 A letter to the editor of the Blood Cells, Molecules and Diseases was published on the 18th December 2009 from Professor Timothy Cox, University of Cambridge Department of Medicine: Recommendations for treating patients with Gaucher disease with emerging enzyme products: see publication http://www.gaucher.org.uk/news.php?id=70 Press Release Source: Protalix BioTherapeutics, Inc. On Wednesday January 13, 2010, 7:30 am CARMIEL, Israel, Jan. 13 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE-Amex: PLX), announced today that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMEA), after reviewing all relevant clinical data, has recommended that the European Commission grant orphan drug designation to taliglucerase alfa, the Company's proprietary plant cell expressed recombinant form of glucocerebrosidase for the treatment of Gaucher disease.  The U.S. Food and Drug Administration (FDA) granted orphan drug designation and fast track designation to taliglucerase alfa in 2009.   Orphan drug designation in Europe is granted to medicinal products intended for the diagnosis, prevention and treatment of life-threatening diseases and very serious conditions that affect not more than five in 10,000 people in the European Union.  Orphan drug designation is generally given to medicinal products that treat conditions for which no current therapy exists or are expected to bring a significant benefit to patients over existing therapies.  If granted by the European Commission, orphan drug designation will provide the Company with a centralized procedure for obtaining marketing authorization for taliglucerase alfa, with a single marketing authorization valid throughout all EU Member States.  The Company may also be eligible for a number of additional incentives including protocol assistance, reduction in registration fees and eligibility for grants and initiatives supporting research and development related to the orphan drug designation. "This is an important step in our global commercialization strategy for taliglucerase alfa," said Dr. Einat Brill Almon, the Company's Senior Vice President, Product Development.  "We plan to file a marketing authorization application with the European Medicines Agency for taliglucerase alfa in the upcoming months."   The Company reported positive top-line results from its pivotal phase III clinical trial of taliglucerase alfa in October 2009.  Full results from this study will be presented at the Annual Meeting of the Lysosomal Disease Network: WORLD Symposium 2010, February 10-12, in Miami, Florida.  In addition, the Company completed the filing of a New Drug Application for taliglucerase alfa with the FDA in December 2009.   In November 2009, the Company entered into a license and supply agreement with Pfizer Inc. (NYSE: PFE) pursuant to which the Company granted Pfizer the exclusive worldwide rights to develop and commercialize taliglucerase alfa for the treatment of Gaucher disease, except for Israel.  The Company retained the right to develop and commercialize taliglucerase alfa in Israel. http://www.gaucher.org.uk/news.php?id=69 Blood Cells, Molecules, and Diseases - 18th December 2009: Guidelines for the restart of imiglucerase in patients with Gaucher disease: Recommendations from the European Working Group on Gaucher disease, click here to see the full paper http://www.gaucher.org.uk/news.php?id=68