VPRIV® (velaglucerase alfa) for injection is a prescription medication indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease.

FAQs

Type 1 Gaucher disease is a rare, inherited, genetic condition. Those affected don’t produce enough of an enzyme called glucocerebrosidase. This enzyme is necessary to help break down a fatty substance called glucocerebroside, preventing it from building up in cells throughout the body, causing them to enlarge. This can affect different organs over time, which is why GD1 is considered a progressive condition.

Learn more about type 1 Gaucher here.

Type 1 Gaucher disease is a rare disease that affects people of all ages and ethnicities, with equal prevalence across genders. However, because GD1 is a genetic disease, it can be more common in certain populations. For example, in the US, there is a higher incidence of GD1 within the Ashkenazi Jewish community.

Learn more about the prevalence of GD1 here.

VPRIV (velaglucerase alfa) is a long-term enzyme replacement therapy (ERT) for patients with Type 1 Gaucher disease. VPRIV is specifically designed to match and replace the natural human enzyme that people with Type 1 Gaucher disease are missing.

Learn more about VPRIV here.

VPRIV is not a new medication; it was approved by the FDA in 2010 and has been treating patients with type 1 Gaucher ever since, giving VPRIV over 10 years of real-world experience.

Learn more about VPRIV here.

VPRIV is made in a human cell line and has the same amino acid sequence as the naturally occurring human enzyme, glucocerebrosidase, that is found in the body. VPRIV is the only ERT made from a human cell line, designed for targeted uptake into cells.

Learn about how VPRIV works here.

VPRIV has demonstrated safety and efficacy; it was studied in the largest clinical study program of an ERT for type 1 Gaucher disease across three clinical studies. The studies included 99 patients aged 4 years and older, in various stages of life.

Learn more about VPRIV safety and efficacy here.

The safety and efficacy of VPRIV have been established for children 4–17 years old with type 1 Gaucher disease. The safety and efficacy of VPRIV have not been established in children younger than four years of age.

The safety and efficacy of VPRIV were studied in 24 pediatric patients in an extension study, and the safety results were found to be similar to those of older participants.

Learn more about treatment for children using VPRIV here.

The clinical data on VPRIV have shown its safety and efficacy in adults who are new to treatment, as well as those who have made a switch from another ERT. VPRIV is indicated for long-term ERT for patients with type 1 Gaucher disease and has established safety and efficacy data in patients who were in various stages of life (aged 4–75 years).

Learn more about clinical trial data for adults using VPRIV here.

In clinical studies of the treatment of Type 1 Gaucher disease using VPRIV, 56 VPRIV-treated patients were 65 years of age or older, including 10 patients who were 75 years of age or older. Older patients were found to experience similar side effects to children and younger adults.

Learn more about treatment of elderly patients using VPRIV here.

Information about VPRIV in pregnant populations can be found here.

Talk to your doctor if you are pregnant, plan to be pregnant, are breastfeeding, or plan to breastfeed and are on, or are considering, VPRIV treatment.

VPRIV infusions typically last 60 minutes, but in some cases you may need to stay longer for post-treatment monitoring.

Learn more about VPRIV infusions here.

If your infusions are well tolerated, you may eventually be able to work with your doctor to arrange for your infusions to be taken at home. Talk to your doctor if this is something that you would want to plan for in your treatment journey. In these cases, a healthcare professional would come to your home to administer the VPRIV infusions for you.

Learn more about home infusions here.

The most commonly reported side effects during clinical studies (in ≥10% of patients) were hypersensitivity reactions, headache, dizziness, abdominal pain, nausea, back pain, joint pain, increased time it takes for blood to clot, tiredness/weakness, and fever. In clinical studies, the overall frequency of side effects was generally higher in the patients not previously treated with ERT than in the patients who switched from imiglucerase to VPRIV.

Hypersensitivity reactions were the most commonly observed side effects in patients treated with VPRIV in clinical studies. The most commonly observed symptoms of hypersensitivity reactions were: headache, dizziness, low blood pressure, high blood pressure, nausea, tiredness/weakness, and fever.

Side effects more commonly seen in pediatric patients compared with adult patients include (>10% difference): rash, increased time it takes for blood to clot, and fever.

Speak to your doctor about any concerns you might have about your treatment with VPRIV.

Please see the Full Prescribing Information or find out more about the risk and safety profile of VPRIV here.

VPRIV does not have any drug–drug interactions listed on its package insert.

Please see the Full Prescribing Information for information about drug–drug interactions.

In a fertility study in male and female rats using a maximum dose the equivalent of 1.8 times the recommended human dose, VPRIV was not found to cause any significant adverse effects on male or female fertility.

Please see the Full Prescribing Information for information and talk to your doctor if you have questions about VPRIV and fertility.

After you and your physician choose a treatment path, Takeda Patient Support is here for you with a range of personalized services for your treatment journey — even when you need to travel away from home. Our support specialists are here to address your questions and concerns, and help get you the answers, resources, and tools you need.

Click here to learn more about this and other support programs available.

It can be useful to record any new or any difference in symptoms you see in the person you care for between appointments or hospital visits—this can help to give a clearer picture of what exactly might have changed since the last time you saw your doctor. You can also note any other aspects that may be affecting your loved one’s experience with GD1 and VPRIV treatment, such as life or schedule changes, or sleeping and eating patterns, and work out how to address these with your doctor. Also be sure to let your doctor know if you think you need any extra support or resources.

Read more potential topics to discuss with your doctor here.

While looking after a loved one can be very fulfilling, it can also have a physical and emotional impact on you as a caregiver. It is very important to still take time for yourself; an important way to make this happen is to be honest with those closest to you about your needs and explain ways they may be able to help you, and free up some time for yourself. Staying organized can also be a big help to relieve stresses surrounding appointments and infusion schedules—if you feel more in control and sure that you’re not missing anything important, it can lift a weight off your shoulders.

important safety information <

Life-threatening hypersensitivity reactions, including anaphylaxis may occur with VPRIV treatment. This reaction may occur early in treatment or after many doses. Seek immediate help if you experience wheezing, shortness of breath, trouble breathing, itching, hives, rapid heartbeat, swelling of the tongue or throat.

important safety information <

Life-threatening hypersensitivity reactions, including anaphylaxis may occur with VPRIV treatment. This reaction may occur early in treatment or after many doses. Seek immediate help if you experience wheezing, shortness of breath, trouble breathing, itching, hives, rapid heartbeat, swelling of the tongue or throat. VPRIV should be administered under the supervision of a healthcare professional. Appropriate medical support should be available when VPRIV is administered.

Hypersensitivity reactions were the most commonly observed side effects in patients treated with VPRIV in clinical studies. The most commonly observed symptoms of hypersensitivity reactions were: headache, dizziness, low blood pressure, high blood pressure, nausea, tiredness/weakness, and fever. 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 patients not previously treated, occurred mostly during the first 6 months of treatment and tended to occur less frequently with time. After the drug was approved, additional hypersensitivity reactions of chest discomfort, difficulty breathing, itching and vomiting have been reported. In some cases, vomiting can be serious and require hospitalization and/or stopping the medication.

If anaphylactic or other acute reactions occur, seek immediate medical care. Your healthcare provider will immediately discontinue the infusion of VPRIV and initiate the appropriate medical treatment. A hypersensitivity reaction should be treated based on the severity of the reaction. Your healthcare provider may manage a reaction by slowing the infusion rate or treating with medicine such as antihistamines, fever-reducing agents and/or corticosteroids or possibly stopping the medication and then restarting with a longer infusion time. For patients who have had symptoms of hypersensitivity reaction to enzyme replacement therapy, the doctor may consider treating the patient with antihistamines and/or corticosteroids before an infusion to help prevent such a reaction from happening.

The most commonly reported side effects during clinical studies (in ≥10% of patients) were hypersensitivity reactions, headache, dizziness, abdominal pain, nausea, back pain, joint pain, increased time it takes for blood to clot, tiredness/weakness, and fever. In clinical studies, the overall frequency of side effects was generally higher in the patients not previously treated with ERT than in the patients who switched from imiglucerase to VPRIV.

Talk to your doctor if you are pregnant, plan to be pregnant, are breastfeeding, or plan to breastfeed.

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. Side effects more commonly seen in pediatric patients compared to adult patients include (>10% difference): rash, increased time it takes for blood to clot, and fever.

The side effect profile in elderly patients was generally similar to that seen in pediatric and other adult patients. In general, dose selection for an elderly patient should be approached cautiously, considering other existing medical conditions.

As with all therapeutic proteins, there is a potential for developing antibodies to VPRIV. In clinical studies, 1 of 54 (2%) patients who had not previously been treated with ERT, who were then treated with VPRIV, developed antibodies. One additional patient developed antibodies to VPRIV during an extension study. It is unknown if having 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.

For additional safety information, please click here for Full Prescribing Information including WARNING for Risk of Anaphylaxis, and discuss with your doctor
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.

For more information, contact Takeda at 1-877-TAKEDA-7 (1-877-825-3327), or by email at medinfous@takeda.com