Up-to-date news on the campaign to Gauchers Association. http://www.gaucher.org.uk Shire Receives CHMP Positive Opinion for VPRIV™ (velaglucerase alfa) for the Treatment of Type 1 Gaucher Disease in the European Union Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency adopted a positive opinion on the marketing authorisation for VPRIV™ (velaglucerase alfa), the company’s enzyme replacement therapy (ERT) for the treatment of type 1 Gaucher disease. The CHMP positive opinion will now be forwarded to the European Commission for ratification, and marks a significant milestone in bringing a new Gaucher treatment to market in all EU Member States. "Gaucher disease is a rare and often debilitating condition," said Professor Timothy Cox, Department of Medicine, University of Cambridge based at Addenbrookes Hospital UK. "I am pleased that, given the difficulties in supplying enzyme treatment for our patients over the last year, we will soon have the opportunity to prescribe velaglucerase alfa without regulatory constraints; the treatment represents an important option in this therapeutic arena." In many European countries patients have been receiving VPRIV on an early access named patient basis, developed in partnership with national and regional authorities. These programs, designed specifically to address the continuing supply shortage of the only other commercially marketed ERT, have experienced strong uptake from physicians and patients and have garnered the support of treatment working groups and advocacy organisations. To date, hundreds of patients in the EU have been treated with VPRIV through Shire’s clinical trials or early access mechanisms. “A positive opinion for VPRIV in the EU, earlier than expected, is very good news,  particularly as we have been working with physicians in 22 countries for almost a year to treat patients with type 1 Gaucher disease through early access programs,” said Sylvie Grégoire, President, Shire Human Genetic Therapies.  “We look forward to our ongoing collaboration with these physicians and the Gaucher community at large as we enter the final stages of the approval process in Europe.”  In addition to the CHMP positive opinion, VPRIV has received orphan drug designation from the Committee for Orphan Medical Products. With the accelerated adoption of VPRIV worldwide, and the earlier than anticipated US approval and EU positive opinion, Shire expects continued high demand for the therapy. As a result of this, the company is now implementing a program with physicians and patients to monitor and manage requests from new patients carefully in order to ensure long-term, uninterrupted treatment with VPRIV. Shire’s VPRIV clinical development program represents the largest and most comprehensive clinical data set supporting registration for an ERT for Type 1 Gaucher disease to date. About VPRIV™ VPRIV is made using Shire’s gene-activation technology, in a human cell line. The glucocerebrosidase enzyme produced has the exact human amino acid sequence and has a human glycosylation pattern. VPRIV was approved by the U.S. Food and Drug Administration on February 26, 2010.  For full US prescribing information see http://www.vpriv.com/ Important Safety Information The most serious adverse reactions in patients treated with VPRIV were hypersensitivity reactions.  Infusion-related reactions were the most commonly observed adverse reactions in patients treated with VPRIV in clinical studies. The most commonly observed symptoms of infusion-related reactions were: headache, dizziness, hypotension, hypertension, nausea, fatigue/asthenia, and pyrexia.  Generally the infusion-related reactions were mild and, in treatment-naïve patients, onset occurred mostly during the first 6 months of treatment and tended to occur less frequently with time.  Other commonly observed adverse reactions in >10% of patients were: abdominal pain, back pain, joint pain, upper respiratory tract infection, and activated partial thromboplastin time prolonged.  Adverse reactions more commonly seen in pediatric patients (>10% difference) included upper respiratory tract infection, rash, activated partial thromboplastin time prolonged, and pyrexia. In clinical trials one patient developed neutralizing antibodies. About Gaucher Disease Gaucher disease is an autosomal recessive disorder caused by mutations in the GBA gene which results in a deficiency of the lysosomal enzyme beta-glucocerebrosidase. This enzymatic deficiency causes an accumulation of glucocerebroside, primarily in macrophages.  In this lysosomal storage disorder, clinical features are reflective of the distribution of Gaucher cells in the liver, spleen, bone marrow, and other organs. The accumulation of glucocerebroside in the liver and spleen leads to organomegaly. Presence of Gaucher cells in the bone marrow and spleen lead to clinically significant anemia and thrombocytopenia. Gaucher disease is the most prevalent of the lysosomal storage disorders. Gaucher disease has classically been categorized into 3 clinical types.  Type 1 Gaucher disease is characterized by variability in signs, symptoms, severity, and progression.  Type 1 is the most common and is distinguished from type 2 and type 3 by the lack of early neurological symptoms. http://www.gaucher.org.uk/news.php?id=75 This program has been set up for the benefit of patients with type I and III Gaucher disease at high-risk for the development of progressive disease or complications and without access to alternative treatments in Europe, the Middle-East and Eurasia during the period of Cerezyme shortage.     Although the program had stopped in January 2010, it has been reinstated now due to continued shortage of Cerezyme supplies. The program is conducted by a Board of Advisors consisting of an independent group of physicians, all members of the European Working Group for Gaucher Disease (EWGGD). The Board of Advisors manages the medical and clinical aspects of the program.  Genzyme supports the logistical part of the program: Genzyme provides the product and manages the distribution of product based on decisions of the Board of Advisors. CETP is not a charitable program. It will run until the stakeholders (EWGGD and Genzyme) conclude that its continuation is no longer necessary. The program is coordinated by the Academic Medical Centre (AMC), Amsterdam, The Netherlands. In order to enable AMC to do this, Genzyme provides financial support to AMC. Background: A global shortage of imiglucerase occurred as a result of viral contamination of the production facility in June 2009. While striving to improve operations at the facility, inventory levels of Cerezyme remain low. As a consequence of a recent disruption to operations of the facility, the current reduced allocations for Cerezyme are prolonged. In September 2009, the European Working Group for Gaucher Disease created an Emergency Treatment Program to allocate Cerezyme to patients at high risk for the development of progressive disease or complications. The program ran till end of December 2009. Due to the prolonged reduced allocations and based on a recommendation of the European Medicines Agency (EMA), the CETP has been reinstated. A limited amount of product allocated to the regions mentioned above will be reserved for this program. The reserved enzyme that is not used within a month’s time, will immediately flow back to the general pool. This will be assessed by Genzyme on a monthly basis in consultation with the coordinator, to allow optimal use of allocations for these regions. If there is insufficient enzyme within the allocation designated to the institution/country to treat patients at high risk for the development of progressive disease or complications, the treating physician may apply for additional Cerezyme, for that patient, to the CETP. Included countries: Albania, Algeria, Austria, Azerbajdian, Belarus, Belgium, Bosnia-Herzogovina, Bulgaria, Croatia, Czech Republic, Denmark, Estonia, Finland, France, Germany, Greece, Hungary, Iceland, Iran*, Jordan*, Ireland, Israel, Italy, Kazakhstan, Kosovo, Kuwait, Lebanon*, Latvia, Lithuania, Luxembourg, Macedonia, Middle East, Montenegro, Morocco, Netherlands, Norway, Oman, Poland, Portugal, Qatar, Romania, Russia, Saudi-Arabia*, Serbia, Slovakia, Slovenia, Spain, Sweden, Switzerland, Tunisia, Turkey, UAE*, UK, Ukraine. The estimated shipment duration is in general 5 working days, except for countries marked with * (about 1 month) and countries in italic (uncertain shipment duration because of import procedures). Patients at high risk for the development of progressive disease or complications: The following criteria were developed by EWGGD: A. Infants, children, adolescents     B. Adult patients (either type I or III) with:    - Exacerbation of disease while on dose reduction/dose interruption     - platelet count < 20.000/ µl - thrombocytopenia and bleeding - symptomatic anemia - severe co-morbidity requiring imiglucerase treatment, such as: 1.    need for chemotherapy 2.    condition that puts a patient at risk for bleeding, e.g. cirrhosis, major surgery, that cannot be postponed for 3-6 months 3.    lung disease caused by Gaucher cell infiltration 4.    new acute bone event during last 12 months C. Pregnant women with symptomatic Gaucher disease     Recommendations by EWGGD for monitoring during imiglucerase reduction or cessation of enzyme replacement therapy and during the recovery period -    clinical examination and history at least every two months -    complete blood count at least every two months -    plasma sample for biomarker analysis such as chitotriosidase at least every two months. -    skeletal manifestations and organ volumes. For the assessment of chitotriosidase, it was recommended to employ local laboratory facilities for early evaluations, using percentage increase from baseline as a possible indication of deterioration. For an analysis of absolute values in the entire group, the stored plasma sample can afterwards be assayed at a central facility to correct for differences between labs. Results of all follow-up studies need to be carefully recorded and all were encouraged to submit these data to the Gaucher Registry of the International Collaborative Gaucher Group (ICGG) and/or national registries. Application process: Click here for application form The treating physician will submit a request form with details on the patient to the AMC (see below for contact details). Approval / response letters to the physicians will be provided by the Program coordinator as soon as possible but no later than 10 days after applications have been received. This program does not guarantee that Cerezyme supply will be available for all emergency cases. The CETP board will review and discuss applications on a weekly basis. If necessary, additional information can be requested. After approval, Genzyme will ship the necessary extra Cerezyme as soon as possible, preferably within 1-2 weeks. For logistical reasons, a prerequisite to the granting of a request is that import of imiglucerase should be feasible within 1 month. CETP board members: Carla E. M. Hollak Department of Endocrinology and Metabolism, Academic Medical Centre, Amsterdam, The Netherlands Ari Zimran Shaare Zedek Medical Centre, Jerusalem, Israel Patrick Deegan Department of Medicine, Addenbrook’s Hospital, University of Cambridge, UK Bruno Bembi Centro di Coordinamento Regionale per le Malattie Rare Ospedale Universitario S. Maria della Misericordia, Udine, Italy Program coordinator and assistant: Marieke Biegstraaten and Lydia Veerhuis, AMC, Amsterdam CETP coordinating centre contact details: Fax: +31 20 691 7682 E-mail: cetp@amc.nl Websites:     www.gaucher.org.uk/index.php www.amc.nl/CETP www.esgld.org   http://www.gaucher.org.uk/news.php?id=74 Cerezyme Genzyme achieved its goal of building a small inventory buffer during the first quarter. The current shipping allocation of 50 percent of demand will be extended, however, due to an interruption in operations at the company’s Allston facility late in the quarter. The interruption resulted from an unexpected city electrical power failure that compounded issues with the plant’s water system, which have been corrected. The facility is fully operational. Genzyme estimates that it will need to continue the 50 percent shipping allocation for 2-3 months. The company will provide a more precise supply update within a month, after determining whether Cerezyme material that was unfinished when the interruption occurred can be finished, the impact of the pending consent decree on product release timelines and a more accurate assessment of global demand. Genzyme will continue to work with minimal levels of inventory for Cerezyme and Fabrazyme until the company’s new Framingham manufacturing facility is approved, which is anticipated to take place in late 2011, and any additional manufacturing delays will likely impact supply of these products. Across the company’s manufacturing operations, programs to expand capacity are on-track. The new Framingham plant is mechanically complete. Pre-operational activities, including cell culture, media preparation, bioreactor validation and staff training, are currently taking place. Engineering and process validation runs are planned for this year. At its Geel, Belgium facility, Genzyme is adding a third bioreactor for the production of Myozyme, and approval is anticipated in mid-2011. The company is also working to transition fill/finish operations out of its Allston facility to its Waterford, Ireland plant and to a contract manufacturer. http://www.gaucher.org.uk/news.php?id=73 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