Starting Treatment

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

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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Details 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.

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Therapy Options

Enzyme Replacement Therapy

The most common treatment for type 1 Gaucher disease is called enzyme replacement therapy (ERT). This kind of therapy is given by an intravenous infusion. It works by replacing or supplementing the enzyme known as glucocerebrosidase (gloo-ko-ser-uh-bro-SI-dase) , which is needed to break down a fatty substance called glucocerebroside (gloo-ko-SER-uh-bro-side) . In people with Gaucher disease, this enzyme does not work properly because it is either absent or faulty.

Substrate Reduction Therapy

Another treatment option for type 1 Gaucher disease is substrate reduction therapy (SRT). SRT is indicated for adult patients with mild to moderate type 1 Gaucher disease for whom ERT is not a therapeutic option (eg, due to constraints such as allergy, hypersensitivity, or poor venous access [ie, difficulty accessing veins]).

SRT is given orally. The goal of SRT is to get the body to produce less glucocerebroside, to a level that allows the enzyme (glucocerebrosidase) to be more effective.

Other Treatment Interventions

If needed, your physician may explore other disease management options, in addition to treatment with either an ERT or SRT, such as:

  • Bisphosphonates for treatment of osteopenia and fracture risk
  • Pain medicine (analgesics) for treatment of bone crisis
  • Antibiotics for treatment of bone infection (called osteomyelitis)
  • Orthopedic procedures, such as hip and knee joint replacement

Be sure to talk with your doctor or other healthcare professionals to learn more about the options available and to decide what is appropriate for you, your child, or family member.

Learn more about how VPRIV is dosed.

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.