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

Diagnosis and Disease Management

The importance of early diagnosis of type 1 Gaucher disease cannot be understated; as a rare disease, GD1 can often go undiagnosed for longer than it should, and may be manageable with appropriate treatment.

Patients with GD1 can experience diagnostic delays of up to 10 years; reasons for this can include a lack of awareness of GD1 due to its rarity and the fact that common symptoms of GD1 can mimic other, better-known conditions and lead to misdiagnoses.

While experiences of GD1 can vary greatly among patients, being aware of the key Signs and Symptoms is the best starting point to help you and others learn more about type 1 Gaucher disease as early as possible.

If you or your loved one is concerned about type 1 Gaucher disease, speak to your doctor and request a test. A simple test may be readily available, and your doctor can request this on your behalf to help confirm or rule out a GD1 diagnosis.

Enzyme activity test:

Sometimes referred to as a “dried blood spot” test, this is a simple blood test that measures the levels of the enzyme, glucocerebrosidase. Low levels of this enzyme may indicate a need for genetic testing—which would then confirm a diagnosis of GD1.

Genetic testing:

Genetic testing helps to identify the specific genetic mutations that result in type 1 Gaucher disease and confirm a diagnosis. Genetic testing may also be used to discover any carriers in the family: carriers do not experience the symptoms of GD1 themselves but may pass on the condition to any children they may have.

Click here for more information on the prevalence of GD1 and how it is inherited.

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Suggestions for Someone Newly Diagnosed with Type 1 Gaucher

Katie, a GD1 patient

VPRIV patient

Disease monitoring

Type 1 Gaucher disease is a lifelong condition; while treatment may help with symptom management, it will not cure the underlying cause. Consistent monitoring and management of the disease after diagnosis are important to help patients work with their doctors to determine their treatment goals. Throughout your disease journey, you should be monitored regularly for various key signs and symptoms, including red blood cell and platelet counts, which are key markers of disease progression. You should also have regular physical examinations and quality-of-life assessments, to ensure your journey with GD1 is on the right track.

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