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

FITTING VPRIV INTO YOUR LIFE

Living with GD1 sometimes means factoring in some extra planning and thinking ahead, and might mean you need some extra support an guidance as you journey through life.

The people in these hypothetical patient profiles have experienced growing up and living with GD1, navigating through various stages that you might be facing in your own life. Their experiences might help you navigate your own, similar circumstances.

BACK TO ALL PROFILES

LIBBY – STARTING SCHOOL

MATEO – GOING TO COLLEGE ALISON – TREATMENT CHOICE NATASHA – PREGNANCY DONNA – TAKING OTHER MEDICATION MICHAEL – FINDING SUPPORT JOHN – RETIREMENT AND TRAVELING
Pediatric Female VPRIV Patient

*Patient profiles for illustrative purposes only and do not depict actual patients

Pediatric Female VPRIV Patient

LIBBY
– STARTING SCHOOL

Male VPRIV Patient
MATEO
– GOING TO COLLEGE
Female VPRIV Patient
ALISON
– TREATMENT CHOICE
Female VPRIV Patient
NATASHA
– PREGNANCY
Female VPRIV Patient
DONNA
– TAKING OTHER MEDICATION
Male VPRIV Patient
MICHAEL
– FINDING SUPPORT
Older Male VPRIV Patient
JOHN
– RETIREMENT AND TRAVELLING

*Patient profiles for illustrative purposes only and do not depict actual patients

KEY TOPICS

  • Newborn Screening
  • Symptom Monitoring
  • Starting School
  • Growing-up with Type 1 Gaucher

LIBBY’S STORY*

When Libby’s parents decided to start a family, a relative told them about genetic screening as part of family planning, given their Ashkenazi Jewish ancestry. People with this ancestry have a one in 17 chance of being a carrier for GD1. With the help of a genetic counselor, Libby’s parents discovered they were both GD1 carriers. When Libby was born and underwent newborn screening, she had positive results for GD1. Their genetic counselor discussed next steps with them.

Libby was asymptomatic at first. Her first GD1 symptom, frequent nosebleeds, appeared around age four. Unsure about starting treatment, Libby’s parents took a “watch and wait” approach with their doctor. Libby was monitored every six to 12 months for changes to her spleen, liver, bone marrow, and blood. Eighteen months later, her nosebleeds were becoming more frequent, and tests showed abnormal platelet counts. Libby’s parents, along with her doctor, made the decision to start her on VPRIV infusions.

As Libby was starting school around this time, her parents met with the school principal to explain her condition and needs, such as the need for an infusion once every two weeks, and that she may experience symptoms such as fatigue. Libby’s parents provided the school nurse with resources to help explain GD1 and VPRIV.

Now, aged eight, Libby is growing up and starting to understand more about her disease. With her parents, she has been learning about GD1 online, and has watched some patient story videos to hear from others. Libby’s parents are looking for community events about rare disease and GD1 so Libby can connect with others who have similar experiences.

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