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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.

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If you have questions about the form, please contact us

Clinical Trial Program for VPRIV

Clinical Trial

See details on the most extensive Gaucher disease clinical trial program for an enzyme replacement therapy (ERT) to date.

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Understanding Type 1 Gaucher Disease

Learn more about this inherited lysosomal storage disorder.

Gaucher disease is a rare genetic disorder that affects specific cells and organs in the body, such as the spleen, liver, and bones.

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OnePath® Services

Learn about the many support services that OnePath® can offer you

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OnePath® is a comprehensive and personalized support program for eligible patients, families, and healthcare providers needing assistance with approved Shire Human Genetic Therapies, Inc. products.

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Why VPRIV?

VPRIV has been shown to improve hemoglobin (a part of red blood cells that carries oxygen in the blood) concentrations and platelet (cells that help blood to clot) counts, and to reduce the size of an enlarged spleen in people with type 1 Gaucher disease.

In VPRIV clinical trials, specific laboratory measurements were evaluated and not general symptoms of type 1 Gaucher disease.

Study comparing 2 different dosages of VPRIV1,2

percentages

At the end of a 12-month study, VPRIV improved several signs of type 1 Gaucher disease in patients who received
60 Units/kg every other week

  • A 12-month study in 25 patients with type 1 Gaucher disease, and compared the effectiveness and safety of 2 different dosages of VPRIV (60 Units/kg and 45 Units/kg.
  • Patients were randomly selected to receive either dosage of VPRIV every other week until week 51. Each patient received a total of 26 infusions.
  • Although not statistically significant, the study also showed a 17% decrease in liver size in patients who received VPRIV 60 Units/kg every other week.

Study comparing VPRIV and imiglucerase1,2

  • A 9-month study of 34 patients with type 1 Gaucher disease that compared the safety and effectiveness of imiglucerase versus VPRIV. The primary objective was to compare the effects of VPRIV and imiglucerase on hemoglobin concentrations in patients with type 1 Gaucher disease.
  • Patients were randomized to receive either 60 Units/kg imiglucerase or 60 Units/kg VPRIV, every other week.
  • The mean treatment difference in change from baseline to 9 months in hemoglobin concentration was 0.1 g/dL.

change in hemoglobin concentration

  • This change from baseline demonstrated that VPRIV was at least as effective as imiglucerase in improving hemoglobin concentration.
  • Secondary endpoints (platelet counts, spleen volume, and liver volume) improved from baseline in both groups, but the differences between VPRIV and imigucerase were not statistically significant between the two groups after 9 months of treatment.

Study in patients switching from imiglucerase to VPRIV1,2

A range of adult and pediatric patients with type 1 Gaucher disease were safely transitioned from imiglucerase to VPRIV.

  • A 12-month, open-label, multinational study in 40 patients who had been receiving treatment with imiglucerase for at least 30 months (dose range 15 Units/kg to 60 Units/kg) and required to have had a stable biweekly dose for at least 6 months prior to enrollment.
  • Imiglucerase therapy was stopped and treatment with VPRIV was begun, administered every other week at the same number of units as the patient’s previous imiglucerase dose.
  • Clinically stable patients who were switched to VPRIV every other week maintained therapeutic responses from baseline (last treatment with imiglucerase) to 12 months.

mean changes from baseline to month 12

Antibody Profile of VPRIV1

Antibodies in the body are produced as a response of the immune system. This response may cause a treatment to stop working or not work as well.

  • In clinical studies, 1 of 54 patients treated with VPRIV (who were not previously treated for type 1 Gaucher disease) developed antibodies to VPRIV
  • 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
  • Antibody test results depend on the type of test performed
  • The presence of antibodies may be affected by such factors as:
    • Method of testing
    • Sample handling
    • Timing of sample collection
    • Other medications used
    • Underlying disease
  • Comparing the risks of developing antibodies to VPRIV with that of developing antibodies to other products may be misleading
  • Patients with an immune response to other enzyme replacement therapies who are switching to VPRIV should continue to be monitored for antibodies

Be sure to speak with your doctor if you have any questions or concerns about developing antibodies.

 

References:
1. VPRIV [package insert]. Cambridge, MA: Shire Human Genetic Therapies, Inc.; 2010.
2. Data on file. Shire Human Genetic Therapies, Inc.

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.