Get Started on VPRIV
In order to begin treatment with VPRIV, you should work with your physician to complete, sign and submit a Start Form, available for download.
If you have questions about the form, please contact us
Enzyme replacement therapy
Learn more about treatment with ERT.
Enzyme replacement therapy is an important treatment choice in type 1 Gaucher disease management.
LEARN MOREDetails About VPRIV
Read more about type 1 Gaucher disease management with VPRIV.
VPRIV has been proven to be an effective treatment option for patients with type 1 Gaucher disease.
LEARN MOREWhat is Type 1 Gaucher Disease?
Overview of Type 1 Gaucher Disease
Named for the French physician Philippe Charles Ernest Gaucher, who described the disease in 1882, Gaucher (go-SHAY) disease is a rare inherited disorder that affects specific cells and organs in the body. It belongs to a group of disorders called lysosomal storage disorders (LSDs).
In Gaucher disease, an enzyme called glucocerebrosidase (gloo-ko-ser-uh-bro-SI-dase) is either absent or faulty. This enzyme's job is to break down a fatty substance called glucocerebroside (gloo-ko-SER-uh-bro-side) that is normally used or eliminated by the body.
When this enzyme doesn’t work properly, glucocerebroside builds up in the cells, where it causes a large accumulation in the organs, tissues, and bone marrow—particularly in the spleen, liver, bones, and central nervous system (CNS: in types 2 and 3 Gaucher disease). This accumulation makes these organs unable to function properly, causing the symptoms of Gaucher disease.
Type 1 Gaucher disease is one of the most common LSDs and affects males and females equally. Although it can affect any ethnic group, Gaucher disease is the most common genetic disease affecting Ashkenazi (eastern and central European) Jewish people. The estimated prevalence rate of Gaucher disease is:
- 1 in 50,000 to 1 in 100,000 in the general population1,2
- 1 in 855 in the Ashkenazi Jewish population2
Incidence of Type 1 Gaucher Disease1,2
- Type 1 Gaucher disease is a non-neuronopathic (i.e no neurologic symptoms) form of the condition and is also the most common form, affecting about 95% of people with Gaucher disease
- Individuals who are affected can show symptoms at any age
- Some people will not show symptoms until adulthood
- The severity of type 1 symptoms can vary, even among family members
Signs and Symptoms of Type 1 Gaucher Disease
The most common signs and symptoms of type 1 Gaucher disease includeA:
- Enlarged spleen and/or liver
- Anemia
- Low platelet count
- Bone abnormalities
AIn VPRIV clinical trials, specific laboratory measurements (hemoglobin concentration, platelet count, and liver and spleen volume) were evaluated and not general symptoms of type 1 Gaucher disease.
For more information about Type 1 Gaucher disease, including other types of the condition, go to gaucherpatients.com.
References:
1. US National Library of Medicine. Medical Encyclopedia: Gaucher disease. http://www.nlm.nih.gov/medlineplus/print/ency/article/000564.htm.Accessed March 5, 2010.
2. GuggenbuhlP, GrosboisB, ChalèsG. Gaucher disease. Joint Bone Spine. 2008;75(2):116-124.
3. MayoClinic.com. Gaucher’s disease. http://www.mayoclinic.com/print/gauchers-disease/DS00972/METHOD=print&DSECTION=all. Accessed March 5, 2010.
VPRIV is available by prescription only.
Indication
VPRIV 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 side effects seen in patients in clinical trials with VPRIV were allergic reactions. Patients who have experienced allergic reactions to VPRIV or to other enzyme replacement therapy should proceed with caution.
- The most common side effects observed in clinical trials in patients treated with VPRIV were infusion-related and included: headache, dizziness, low blood pressure, high blood pressure, nausea, weakness/fatigue, and fever. Generally, infusion-related reactions were mild and, in newly treated patients, occurred mostly during the first 6 months of treatment and tended to occur less frequently with time.
- Management of infusion-related reactions is based on severity and may include slowing the infusion rate, treatment with medications such as antihistamines, fever-reducing agents and/or corticosteroids, and/or stopping and resuming treatment with increased infusion time. Side effects and any treatment concerns should be discussed with your physician.
- The most commonly reported side effects (occurring in ≥10% of patients) that were considered related to VPRIV included: headache, dizziness, abdominal pain, nausea, back pain, joint pain, upper respiratory tract infection, aPTT prolonged (eg, blood clotting difficulty), infusion-related reactions, fever, and weakness/fatigue.
- All adult side effects of VPRIV are considered relevant to children (ages 4 to 17 years). Side effects more commonly seen in children compared with adult patients included: upper respiratory tract infection, rash, aPTT prolonged, and fever. The safety of VPRIV has not been established in patients younger than 4 years of age.
- As with all therapeutic proteins, there is the potential of developing antibodies. It is unknown if the presence of 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.
- Your doctor may prescribe VPRIV to you if you are pregnant, only if it is clearly necessary.
- Tell your healthcare provider if you experience any side effects. For more information about VPRIV, ask your healthcare provider, read the Full Prescribing Information, visit www.VPRIV.com, or call Shire at 1-866-888-0660.
- You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.
