Vpriv 400

/ Takeda

VPRIV treatment should be supervised by a physician experienced in the management of patients with Gaucher disease.
The recommended dose is 60 Units/kg administered every other week.
Dose adjustments can be made on an individual basis based on achievement and maintenance of therapeutic goals. Clinical studies have evaluated doses ranging from 15 to 60 Units/kg every other week. Doses higher than 60 Units/kg have not been studied.
Patients currently treated with imiglucerase enzyme replacement therapy for type 1 Gaucher disease may be switched to VPRIV, using the same dose and frequency.
Renal or hepatic impairment: No dosing adjustment is recommended in patients with renal or hepatic impairment based on current knowledge of the pharmacokinetics and pharmacodynamics of velaglucerase alfa.
Elderly (≥65 years old): Elderly patients may be treated within the same dose range (15 to 60 U/kg) as other adult patients.
Paediatric population: Twenty of the 94 patients (21%) who received velaglucerase alfa during clinical studies were in the paediatric and adolescent age range (4 to 17 years). The safety and efficacy profiles were similar between paediatric and adult patients.
Method of administration: For intravenous infusion use only. To be administered as a 60-minute intravenous infusion. Must be administered through a 0.22 µm filter.

VPRIV is a hydrolytic glucocerebroside – specific enzyme indicated for the long term replacement therapy (ERT) for pediatric and adult patients with type 1 Gaucher disease.

Severe allergic reaction to the active substance or to any of the excipients.

Hypersensitivity: Hypersensitivity reactions, including symptoms consistent with anaphylaxis, have been reported in patients in clinical studies and in post-marketing experience. The majority of hypersensitivity reactions usually occur up to 12 hours post infusion. The most frequently reported symptoms of hypersensitivity include nausea, rash dyspnoea, back pain, chest discomfort (including chest tightness), urticaria, arthralgia, and headache.
Infusion- related-reactions: An infusion-related reaction is defined as any adverse drug reaction occurring within 24 hours after the initiation of velaglucerase alfa infusion. Infusion-related reactions (IRR) were the most commonly observed adverse reactions in patients treated in clinical studies. An IRR often appears as a hypersensitivity reaction. The most frequently reported symptoms of hypersensitivity include nausea, rash, dyspnoea, back pain, chest discomfort (including chest tightness), urticaria, arthralgia, and headache. Symptoms consistent with anaphylaxis have been reported in patients in clinical studies and in post-marketing experience. Apart from symptoms associated with hypersensitivity reactions IRRs might show as fatigue, dizziness, pyrexia, blood pressure increase, pruritus, or vision blurred.
In treatment-naïve patients, the majority of infusion-related reactions occurred during the first 6 months of treatment.
Prevention and Management of infusion related reactions including hypersensitivity reactions: The management of infusion-related reactions should be based on the severity of the reaction, and include slowing the infusion rate, treatment with medicinal products such as antihistamines, antipyretics and/or corticosteroids, and/or stopping and resuming treatment with increased infusion time.
Due to the risk for hypersensitivity reactions including anaphylaxis appropriate medical support, including adequately trained personnel in emergency measures, should be readily available when velaglucerase alfa is administered. If anaphylactic or other acute reactions occur, in the clinic or home setting, immediately discontinue the infusion and initiate appropriate medical treatment. For patients developing anaphylaxis in a home setting it should be considered to continue treatment in a clinical setting.
Treatment should be approached with caution in patients who have exhibited symptoms of hypersensitivity to velaglucerase alfa or other enzyme replacement therapy.
Pre-treatment with antihistamines and/or corticosteroids may prevent subsequent reactions in those cases where symptomatic treatment was required.
Immunogenicity: Antibodies may play a role in treatment-related reactions found with the use of velaglucerase alfa. To further evaluate the relationship, in cases of severe infusion-related reactions and in cases of lack or loss of effect patients should be tested for the presence of antibodies and the results reported to the company.
In the clinical trials, one of 94 (1%) patients developed IgG-class antibodies to velaglucerase alfa. In this one event, the antibodies were determined to be neutralising in an in vitro assay. No infusionrelated reactions were reported for this patient. No patients developed IgE antibodies to velaglucerase alfa.
Sodium: This medicinal product contains 12.15 mg sodium per vial. To be taken into consideration by patients on a controlled sodium diet.

The most serious adverse reactions in patients in clinical trials were hypersensitivity reactions.
The most common adverse reactions were infusion-related reactions. The most commonly observed symptoms of infusion-related reactions were: headache, dizziness, hypotension, hypertension, nausea, fatigue/asthenia, and pyrexia/body temperature increased.
The only adverse reaction leading to discontinuation of treatment was an infusion-related reaction.
See prescribing information for full details.

No interaction studies have been performed.

Pregnancy: There are no or limited amount of data from the use of velaglucerase alfa in pregnant women.
Animal studies do not indicate direct or indirect harmful effects with respect to pregnancy, embryonal/foetal development, parturition or postnatal development. Close monitoring of the pregnancy and clinical manifestations of Gaucher disease is necessary for the individualisation of therapy . Caution should be exercised when prescribing to pregnant women.
Lactation: There are no data from studies in breast-feeding women. It is not known whether velaglucerase alfa is excreted in human milk. Caution should be exercised when prescribing to a breast-feeding woman.

There is no experience with overdose of velaglucerase alfa. The maximum dose of velaglucerase alfa in clinical studies was 60 Units/kg.

Storage: Store in a refrigerator (2°C – 8°C). Do not freeze.