What is type 1 Gaucher disease (GD1)?
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.
Who is affected by type 1 Gaucher disease?
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.
What is VPRIV?
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.
Is VPRIV a new medication?
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.
How is VPRIV made?
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.
Has VPRIV demonstrated safety and efficacy?
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.
Is VPRIV an appropriate treatment option for my child?
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.
Is VPRIV an appropriate treatment option for me as an adult?
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.
Is VPRIV an appropriate treatment option for me as an older patient?
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.
Where can I find information about VPRIV and pregnancy?
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.
How long will my infusion take?
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.
Can I have my VPRIV infusions at home?
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.
What are the most common side effects of VPRIV?
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.
Does VPRIV have any interactions with other drugs?
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.
What information is available about VPRIV and fertility?
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.
What support services are offered by Takeda?
OnePath® is a free product support program offered by Takeda to those who have opted in.
Through OnePath®, patients can work with a dedicated Patient Support Manager to help answer questions and navigate individual treatment needs. A Patient Access Manager can help to navigate new or existing insurance information, including reimbursement, financial, or other access-related issues.
Click here to learn more about this and other support programs available.
As a caregiver to a patient on VPRIV treatment, how should I prepare for doctor appointments or hospital visits?
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.
What are some ways I can take care of myself while also caring for a loved one during their VPRIV treatment?
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.