Sources of Information and Support
Being diagnosed with a rare disease like type 1 Gaucher disease can be difficult for your patients and their families. Let your patients know that they are not alone. There are many rare disease support groups and advocacy organizations that may serve as a source of information and support for patients with type 1 Gaucher disease.
This group gives your patients access to information about diseases like Gaucher that have a high incidence among those of Ashkenazi Jewish descent. Contact them to connect with relevant organizations. Visit their website now.
Find information on the latest Gaucher disease research, get financial assistance, discuss legislative issues, and find outreach programs. Also participate in live web-based meetings, national conferences, and patient meetings. Visit their website now.
This organization provides information about rare diseases like Gaucher, but also has more specific functions that may be useful for both patients and HCPs. These include referrals to patient organizations, information about research grants and fellowships, advocacy and medication assistance programs, and networked peer-to-peer support for those with Gaucher disease. Visit their website now.
Connect your patients with this organization so they can find genetic counseling in their area for inherited conditions like Gaucher disease. Visit their website now.
Important Safety Information
Hypersensitivity reactions, including anaphylaxis have occurred. The most serious adverse reactions in patients treated with VPRIV (velaglucerase alfa for injection) were hypersensitivity reactions.
Hypersensitivity reactions were the most commonly observed adverse reactions in patients treated with VPRIV in clinical studies. Patients were not routinely pre-medicated prior to infusion of VPRIV. The most commonly observed symptoms of hypersensitivity reactions were: headache, dizziness, hypotension, hypertension, nausea, fatigue/asthenia, and pyrexia. Hypersensitivity reactions in the clinical trials include any event considered related to and occurring within up to 24 hours of VPRIV infusion, including one case of anaphylaxis. Generally the 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.
As with any intravenous protein product, hypersensitivity reactions are possible, therefore appropriate medical support, including personnel adequately trained in cardiopulmonary resuscitative measures and access to emergency measures should be readily available when VPRIV is administered. If anaphylactic or other acute reactions occur, immediately discontinue the infusion of VPRIV and initiate the appropriate medical treatment.
Management of hypersensitivity 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. In cases where patients have exhibited symptoms of hypersensitivity to the active ingredient or excipients in the drug product or to other enzyme replacement therapy, pre-treatment with antihistamines and/or corticosteroids may prevent subsequent reactions.
The most common adverse reactions during clinical studies (in ≥10% of patients) were hypersensitivity reactions, headache, dizziness, abdominal pain, nausea, back pain, joint pain, prolonged activated PTT, fatigue/asthenia, and pyrexia. In clinical studies, the overall frequency of adverse events was generally higher in the population naïve to ERT (enzyme replacement therapy) than in the population switched from imiglucerase to VPRIV.
VPRIV is classified as pregnancy category B.
The safety and efficacy profiles were similar in pediatric (ages 4 to 17) and adult patients. The safety of VPRIV has not been established in patients under 4 years of age. Adverse reactions more commonly seen in pediatric patients compared to adult patients include (>10% difference): rash, aPTT prolonged, and pyrexia.
The adverse reaction profile in elderly patients was consistent with that previously observed across pediatric and adult patients. In general, dose selection for an elderly patient should be approached cautiously, considering comorbid conditions.
As with all therapeutic proteins, there is a potential for immunogenicity. In clinical studies, 1 of 54 enzyme treatment-naïve patients treated with VPRIV 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 to VPRIV.
Please see Full Prescribing Information.
To report suspected adverse reactions, contact Shire Medical Information at 1-866-888-0660, option 2 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
For assistance with medical inquiries about VPRIV, please contact Medical Information at 1-866-888-0660, option 2 or email
VPRIV® (velaglucerase alfa for injection) is indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease.