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.

OnePathSM is a comprehensive and individualized product support system dedicated to helping patients, families, and healthcare providers. The array of services offered by OnePathSM provides assistance for eligible US patients in accessing therapy.

OnePath; The way to care

If you are a physician and interested in finding out more about how OnePathSM can help you and your patients, please contact our OnePath services at 1-866-888-0660.

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Physicians

Letter to Physicians

June 22, 2010

Dear Healthcare Professional:

I would like to take this opportunity to inform you of an important update related to our enzyme replacement therapy, VPRIVTM (velaglucerase alfa for injection). You may have heard that, because of 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 your patients currently on VPRIV will continue to have uninterrupted access to treatment at your 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 to 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 also developing an emergency access supply which will be made available for the most critical new cases. We will provide details on this as soon as they are available. Patients will be informed of these changes in the coming days. We will reiterate to patients that any individual treatment decisions will remain with you as their treating physician.

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 thank you for your continued support and assistance to Shire and the Gaucher patient community.

Please See Full Prescribing Information

Indication

VPRIV™ (velaglucerase alfa for injection) is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy (ERT) for pediatric and adult patients with type 1 Gaucher disease.

Important Safety Information

  • The most serious adverse reactions in patients treated with VPRIV were hypersensitivity reactions. Appropriate medical support should be available when VPRIV is administered. If a severe reaction occurs, medical standards for emergency treatment are to be followed.
  • Treatment with VPRIV should be used with caution in patients who have exhibited symptoms of hypersensitivity to the active ingredient, drug product excipients, or to other enzyme replacement therapies.
  • 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, asthenia/fatigue, 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.
  • Management of infusion-related reactions should be based on severity of the reaction, such as slowing the infusion rate, treatment with medications such as antihistamines, antipyretics and/or corticosteroids, and/or stopping and resuming treatment with increased infusion time.
  • Other commonly observed adverse reactions in ≥10% of ERT-naïve and imiglucerase-switched patients were: headache, dizziness, abdominal pain, nausea, back pain, joint pain, upper respiratory tract infection, activated PTT prolonged, infusion-related reactions, pyrexia, and asthenia/fatigue.
  • All adult adverse reactions to VPRIV are considered relevant to pediatric patients (ages 4 to 17 years). Adverse reactions more common in pediatric patients (>10% difference) included upper respiratory tract infection, rash, aPTT prolonged, and pyrexia. The safety of VPRIV has not been established in pediatric patients younger than 4 years of age.
  • As with all therapeutic proteins, there is a potential for immunogenicity. In clinical studies, 1 of 54 treatment-naïve patients treated with VPRIV (who received a 45 Units/kg dose) developed IgG class antibodies (neutralizing in an in vitro assay). It is unknown if the presence of IgG antibodies to VPRIV is associated with a higher risk of infusion reactions. Patients with an immune response to other enzyme replacement therapies who are switching to VPRIV should continue to be monitored for antibodies.
  • For more information, please see the full prescribing information or call Shire at 1-866-888-0660. More information about VPRIV can also be obtained by going to www.vpriv.com. To report suspected adverse events contact Shire Human Genetic Therapies, at the OnePathSM phone number 1-866-888-0660 or MedInfoGlobal@Shire.com, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.