What is Gaucher Disease?
Gaucher disease is a rare, inherited disorder that affects specific cells and organs in the body. The areas most affected include the spleen, liver, and bones, as well as the central nervous system. A fatty substance known as glucocerebroside, begins to accumulate inside the cells in these areas when a specific enzyme, named glucocerebrosidase, is unable to break down glucocerebroside. In people with Gaucher disease, Glucocerebrosidase is either missing, is produced by the body in only small amounts, or is unable to function properly.
There are approximately 7,000 diagnosed Gaucher patients worldwide. Based on that incidence, the estimated total world population is likely between 10,000 and 15,000 patients.
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Patients
Letter to Patients
June 22, 2010
Dear Patient Community:
Due to the accelerated approval for VPRIVTM (velaglucerase alfa for injection) and early access programs we initiated for patients during the continuing imiglucerase supply shortage, as well as the extremely high demand for VPRIV that has followed, we must now closely monitor future demand, inventory and capacity in order to set appropriate expectations for additional patients that may be interested in VPRIV in the future.
The purpose of this letter is to emphasize that for patients who are currently on VPRIV or already in the process to begin VPRIV therapy (i.e. Shire has received a OnePathSM start form), nothing has changed. We can tell you that we are committed to ensuring patients currently on VPRIV will continue to have uninterrupted access to treatment at their prescribed dose. We also want to assure you we have sufficient inventory to meet the expected demand of all patients for whom a start form has been submitted and received by Shire by June 21, 2010.
To ensure continued access to all patients mentioned above, we need to carefully manage additional requests for VPRIV. Therefore, for additional patients who submit a start form after June 21, 2010, Shire has established a waiting list process. Patients will be added to the list according to the date Shire receives their start form. This waitlist will enable patients to begin the process of accessing treatment as soon as additional product becomes available.
We are working to optimize our existing manufacturing facilities to increase supply, and are expanding manufacturing facilities by building a new production site in Lexington, MA (USA).
Finally, we would like to reassure you that this process has nothing to do with product quality or manufacturing delays or concerns. Rather, it is centered on an accelerated launch timeline, finite capacity, and extremely high demand.
If you should have further questions, please contact OnePathSM. OnePathSM, our comprehensive U.S. product support center, is available from 8:30am to 8:00pm (ET) at 1-866-888-0660 and can assist with any questions you may have as well.
We look forward to serving you now and in the future. Thank you for your continued support.