Published by Genzyme at http://supplyupdate.genzyme.com (8/07/09)

Alternate Therapies For Gaucher Patients

We recognize that access to treatment is a top concern for physicians, other health care providers, patients, and their families during this temporary period of Cerezyme® (imiglucerase for injection) supply shortage. It is our top priority as well. Therefore, we want to provide some information about alternative therapies that may be helpful for patients who would like to explore other options during this temporary period.

Genzyme is recruiting patients for two clinical trials of an investigational oral Gaucher treatment, GENZ-112638. One trial is for patients who have never been on treatment with Cerezyme®, or have been off Cerezyme® for at least 12 months. The second trial is a switch study for patients who are receiving Cerezyme®. Information about these trials is available on www.clinicaltrials.gov.

Additionally, in the United States, the FDA has a mechanism called a "treatment IND" (IND stands for Investigational New Drug) to allow companies to make experimental drugs available before they are approved. This is only done in certain exceptional situations because the FDA has an obligation to protect patients' health. It is expected that the FDA will reach out to companies with drugs in clinical development to ensure sufficient drug supply during a shortage of approved drug.

Genzyme submitted a treatment IND for our investigational oral therapy, GENZ-112638, to FDA late in July. We expect to receive a response by August 31st. If the treatment IND is approved by the FDA and the relevant IRBs, patients not eligible for the above-mentioned clinical trials will be able to receive GENZ-112638 if they meet the eligibility criteria included in the treatment IND protocol.

Other companies also have investigational drugs for Gaucher disease at various stages of development. As a result of the Cerezyme® supply shortage, the FDA has asked these companies, Shire and Protalix, to submit treatment INDs for velaglucerase and prGCD, respectively. On August 3rd, Shire announced that the FDA had approved their treatment IND. According to their public statements, Protalix submitted a treatment IND to FDA in July.

For more information on the FDA's treatment IND process, please refer to their website: http://www.fda.gov/ScienceResearch/SpecialTopics/RunningClinicalTrials
/GuidancesInformationSheetsandNotices/ucm117839.htm

If you are a patient and want more information on enrolling in these programs, please contact your physician.

If you are a physician and would like more information, please contact the companies directly.

Genzyme: Genzyme Medical Information in your local country. Click this link to access a list of global locations.

Shire (One Path): 1-866-888-0660; http://www.clinicaltrials.gov/ct2/show/NCT00954460?term=velaglucerase&rank=1

Protalix (Israel): +972-4-9028100 or email info@protalix.com or patient-info@protalix.com