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ANNEX I 

 

SUMMARY OF PRODUCT CHARACTERISTICS 



 

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1. NAME OF THE MEDICINAL PRODUCT 

 

VPRIV 400 Units powder for solution for infusion 

 

 

2. QUALITATIVE AND QUANTITATIVE COMPOSITION 

 

Each vial contains 400 Units* of velaglucerase alfa**. 

After reconstitution, one ml of the solution contains 100 Units of velaglucerase alfa. 

 

*An enzyme unit is defined as the amount of enzyme that is required to convert one micromole of 

p-nitrophenyl β-D-glucopyranoside to p-nitrophenol per minute at 37 ºC. 

**produced in an HT-1080 human fibroblast cell line by recombinant DNA technology. 

 

Excipient with known effect 

 

Each vial contains 12.15 mg sodium. 

 

For the full list of excipients, see section 6.1. 

 

 

3. PHARMACEUTICAL FORM 

 

Powder for solution for infusion. 

 

White to off-white powder. 

 

 

4. CLINICAL PARTICULARS 

 

4.1 Therapeutic indications 

 

VPRIV is indicated for long-term enzyme replacement therapy (ERT) in patients with type 1 Gaucher 

disease.  

 

4.2 Posology and method of administration 

 

VPRIV treatment should be supervised by a physician experienced in the management of patients with 

Gaucher disease.  

 

Posology 

 

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. 

 

Special populations 

 
Elderly (≥65 years old) 

Elderly patients may be treated within the same dose range (15 to 60 units/kg) as other adult patients 

(see section 5.1). 



 

3 

 

Renal impairment 

No dosing adjustment is recommended in patients with renal impairment based on current knowledge 

of the pharmacokinetics and pharmacodynamics of velaglucerase alfa (see section 5.2). 

 

Hepatic impairment 

No dosing adjustment is recommended in patients with hepatic impairment based on current 

knowledge of the pharmacokinetics and pharmacodynamics of velaglucerase alfa (see section 5.2). 

 

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 (see section 5.1 for further information).  

 

The safety and efficacy of velaglucerase alfa in children below the age of 4 years have not yet been 

established. No data are available. 

 

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. 

 

Home administration under the supervision of a healthcare professional may be considered only for 

those patients who have received at least three infusions and were tolerating their infusions well. 

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, immediately discontinue the infusion and initiate appropriate medical treatment (see 

section 4.4). 

 

For instructions on reconstitution and dilution of the medicinal product before administration, see 

section 6.6. 

 

4.3 Contraindications 

 

Severe allergic reaction to the active substance or to any of the excipients listed in section 6.1. 

 

4.4 Special warnings and precautions for use 

 

Traceability 

 

In order to improve the traceability of biological medicinal products, the name and batch number of 

the administered medicinal product should be clearly recorded.  

 

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 



 

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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 studies 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 

infusion-related 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. This is equivalent to 0.6% of the WHO 

recommended maximum daily intake of 2 g sodium for an adult. 

 

4.5 Interaction with other medicinal products and other forms of interaction 

 

No interaction studies have been performed. 

 

4.6 Fertility, pregnancy and lactation 

 

Women of childbearing potential 

 

Patients who have Gaucher disease and become pregnant may experience a period of increased disease 

activity during pregnancy and the puerperium. A risk-benefit assessment is required for women with 

Gaucher disease who are considering pregnancy. 

 



 

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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. 

 

Breast-feeding 

 

There is insufficient information on the excretion of velaglucerase alfa or its metabolites in human 

milk. Velalglucerase is a synthetic form of beta-glucocerebrosidase, which is a normal component of 

human milk. Studies with other forms of the enzyme have found very low levels of the enzyme in 

breastmilk. A decision must be made whether to discontinue breast-feeding or to discontinue/abstain 

from VPRIV taking into account the benefit of breast-feeding for the child and the benefit of therapy 

for the woman. 

 

Fertility 

 

Animal studies show no evidence of impaired fertility (see section 5.3). 

 

4.7 Effects on ability to drive and use machines 

 

VPRIV has no or negligible influence on the ability to drive or use machines. 

 

4.8 Undesirable effects 

 

Summary of the safety profile 

  

The most serious adverse reactions in patients in clinical studies were hypersensitivity reactions 

(2.1%). 

 

The most common adverse reactions were infusion-related reactions (39.4%). The most commonly 

observed symptoms of infusion-related reactions were: headache, dizziness, hypotension, 

hypertension, nausea, fatigue/asthenia, and pyrexia/body temperature increased (see section 4.4 for 

further information). The only adverse reaction leading to discontinuation of treatment was an 

infusion-related reaction. 

 

Tabulated list of adverse reactions 

 

Adverse reactions reported in patients with type 1 Gaucher disease are listed in Table 1. Information is 

presented by system organ class and frequency according to MedDRA convention. Frequency is 

defined as very common (1/10), common (1/100 to <1/10), and uncommon (≥1/1,000 to <1/100). 

Within each frequency grouping, adverse reactions are presented in order of decreasing seriousness.  

 

Table 1: Adverse reactions reported with VPRIV in patients with type 1 Gaucher disease  

 

System organ class 

 

Adverse reaction 

 Very common Common Uncommon 

Immune system 

disorders 

 hypersensitivity reactions 

(includes dermatitis allergic 

and 

anaphylactic*/anaphylactoid 

reactions) 

 



 

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Nervous system 

disorders 

headache, dizziness   

Eye disorders   vision blurred* 

Cardiac disorders  tachycardia  

Respiratory, 

thoracic and 

mediastinal 

disorders 

 dyspnoea*  

Vascular disorders  hypertension, hypotension, 

flushing 

 

Gastrointestinal 

disorders 

abdominal 

pain/abdominal pain 

upper, 

nausea vomiting* 

Skin and 

subcutaneous tissue 

disorders 

 rash, urticaria, pruritus*  

Musculoskeletal 

and connective 

tissue disorders 

bone pain, arthralgia, 

back pain 

  

General disorders 

and administration 

site conditions 

infusion-related 

reaction, 

asthenia/fatigue, 

pyrexia/body 

temperature 

increased 

chest discomfort*  

Investigations  activated partial 

thromboplastin time 

prolonged, neutralizing 

antibody positive 

 

*Adverse reactions derived from post-marketing reports 

 

Description of selected adverse reactions 

 

Vomiting 

 

In some cases vomiting can be serious and severe. Vomiting most often occurs during the infusion and 

up to 24 hours after the infusion. 

   

Other special populations 

 

Elderly population (≥65 years) 

 

The safety profile of VPRIV in clinical studies involving patients aged 65 years and above was similar 

to that observed in other adult patients. 

 

Paediatric population 

 

The safety profile of VPRIV in clinical studies involving children and adolescents aged 4 to 17 years 

was similar to that observed in adult patients.  

 

Reporting of suspected adverse reactions 

 

Reporting suspected adverse reactions after authorisation of the medicinal product is important. It 

allows continued monitoring of the benefit/risk balance of the medicinal product. Healthcare 

professionals are asked to report any suspected adverse reactions via the national reporting system 

listed in Appendix V. 

 

http://www.ema.europa.eu/docs/en_GB/document_library/Template_or_form/2013/03/WC500139752.doc


 

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4.9 Overdose 

 

There is limited information available regarding overdose with velaglucerase alfa. In the majority of 

the cases reporting overdose, no additional adverse events were observed. However, in the event of 

accidental or intentional overdose, patients should be carefully observed and treatment should be 

symptomatic and supportive. There is no antidote available. The maximum dose of velaglucerase alfa 
in clinical studies was 60 Units/kg (see section 4.4). 

 
5. PHARMACOLOGICAL PROPERTIES 

 

5.1 Pharmacodynamic properties 

 

Pharmacotherapeutic group: Other alimentary tract and metabolism products, enzymes, ATC code: 

A16AB10. 

 

Gaucher disease is an autosomal recessive disorder caused by mutations in the GBA gene which 

results in a deficiency of the lysosomal enzyme beta-glucocerebrosidase. This enzymatic deficiency 

causes an accumulation of glucocerebroside primarily in macrophages, giving rise to foam cells or 

"Gaucher cells". In this lysosomal storage disorder (LSD), clinical features are reflective of the 

distribution of Gaucher cells in the liver, spleen, bone marrow, skeleton, and lungs. The accumulation 

of glucocerebroside in the liver and spleen leads to organomegaly. Bone involvement results in 

skeletal abnormalities and deformities as well as bone pain crises. Deposits in the bone marrow and 

splenic sequestration lead to clinically significant anaemia and thrombocytopenia. 

 

The active substance of VPRIV is velaglucerase alfa, which is produced by gene activation technology 

in a human cell line. Velaglucerase alfa is a glycoprotein. The monomer is approximately 63 kDa, has 

497 amino acids, and the same amino acid sequence as the naturally occurring human enzyme, 

glucocerebrosidase. There are 5 potential N-linked glycosylation sites, four of which are occupied. 

Velaglucerase alfa is manufactured to contain predominantly high-mannose-type glycans to facilitate 

internalisation of the enzyme by the phagocytic target cells via the mannose receptor. 

 

Velaglucerase alfa supplements or replaces beta-glucocerebrosidase, the enzyme that catalyses the 

hydrolysis of glucocerebroside to glucose and ceramide in the lysosome, reducing the amount of 

accumulated glucocerebroside and correcting the pathophysiology of Gaucher disease. Velaglucerase 

alfa increases haemoglobin concentration and platelet counts and reduces liver and spleen volumes in 

patients with type 1 Gaucher disease. 

 

In studies 025EXT and 034, patients were offered home therapy. In study 025EXT, 7 of 10 patients 

received home therapy at least once during 60 months of treatment. In study 034, 25 of 40 patients 

received home therapy at least once during the 12-month study. 

 

Clinical efficacy and safety 

 

Studies in treatment naïve patients 

 

Study 025 was a 9 month, open-label study in 12 adult (≥18 years) patients who were naïve to ERT 

(defined as having not been treated with ERT for at least 12 months prior to study entry). 

Velaglucerase alfa was initially administered in a dose-escalating fashion in the first 3 patients (15, 30, 

60 Units/kg) and the 9 remaining patients began treatment with 60 Units/kg. 

 

Clinically meaningful improvements from baseline were observed in haemoglobin concentration and 

platelet counts as early as 3 months and in liver and spleen volumes at both 6 months and 9 months 

following the initiation of treatment with velaglucerase alfa. 

 

Ten patients who completed Study 025 enrolled in an open-label extension study (025EXT), 8 of 

whom completed the study. After a minimum of 12 months of continuous treatment with 

velaglucerase alfa, all patients qualified to have the dose of velaglucerase alfa reduced in a step-wise 



 

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fashion from 60 to 30 Units/kg after achieving at least 2 of the 4 “Year 1” therapeutic goals of ERT for 

type 1 Gaucher disease. Patients received doses ranging from 30 to 60 Units/kg (median dose 

35 Units/kg) every other week for up to 84 months (7 years). Sustained clinical activity continued to 

be demonstrated during treatment as observed by improvements in haemoglobin concentrations and 

platelet counts and reduced liver and spleen volumes. 

 

By month 57, 8 out of the 8 patients had achieved a reduction of at least 2 points in the lumbar spine 

Bone Marrow Burden (BMB) score as assessed by MRI scan. Improvement from baseline in mean 

lumbar spine and femoral neck bone mineral density (BMD) Z-scores were observed at month 24 (0.4; 

95% CI 0.1, 0.7) and month 33 (0.4; 95% CI 0.2, 0.6), respectively. After seven years of treatment, the 

mean increase from baseline in Z-scores were 0.7 (95% CI 0.4, 1.0) for the lumbar spine and 0.5 (95% 

CI 0.2, 0.7) for the femoral neck. No patients were classified at a more severe WHO classification of 

bone density compared to baseline.   

 

Study 032 was a 12-month, randomised, double-blind, parallel-group efficacy study in 25 patients 

aged 2 years and older who were naïve to ERT (defined as having not been treated with ERT for at 

least 30 months prior to study entry). Patients were required to have Gaucher disease-related anaemia 

and either thrombocytopenia or organomegaly. Patients were randomised to receive velaglucerase alfa 

at a dose of either 45 Units/kg (N=13) or 60 Units/kg (N=12) every other week.  

 

Velaglucerase alfa 60 Units/kg given intravenously every other week demonstrated clinically 

meaningful increases from baseline in mean haemoglobin concentration (+2.4 g/dl) and platelet count 

(+50.9 x 109/l), liver volume was reduced from 1.46 to 1.22 times normal (mean reduction of 17%) 

and spleen volume was reduced from 14.0 to 5.75 times normal (mean reduction of 50%). Meaningful 

increases from baseline were observed in the 45 Units/kg dose group in haemoglobin concentration 

(+2.4 g/dl) and platelet count (+40.9 x 109/l), liver volume was reduced from 1.40 to 1.24 times 

normal (mean reduction of 6%) and spleen volume was reduced from 14.5 to 9.50 times normal (mean 

reduction of 40%). 

 

Study 039 was a 9-month, randomised, double-blind, non-inferiority, active-comparator (imiglucerase) 

controlled, parallel-group efficacy study in 34 patients aged 2 years and older who were naïve to ERT 

(defined as having not been treated with ERT for at least 12 months prior to study entry). Patients 

were required to have Gaucher disease-related anaemia and either thrombocytopenia or organomegaly. 

Patients received either 60 Units/kg of velaglucerase alfa (N=17) or 60 Units/kg of imiglucerase 

(N=17) every other week. 

 

The mean absolute increase from baseline in haemoglobin concentrations was 1.624 g/dl (±0.223 SE) 

following 9 months of treatment with velaglucerase alfa. This increase in haemoglobin concentration 

was demonstrated to be clinically and statistically non-inferior to imiglucerase (mean treatment 

difference of change from baseline to 9 months [velaglucerase alfa – imiglucerase]: 0.135 g/dl). There 

were no statistically significant differences between velaglucerase alfa and imiglucerase in changes in 

platelet counts and liver and spleen volumes after 9 months of velaglucerase alfa treatment, and in the 

time to first haemoglobin response (defined as 1 g/dl increase from baseline). 

 

Study in patients switching from imiglucerase treatment to VPRIV 

 

Study 034 was a 12-month, open-label safety study in 40 patients aged 2 years and older who had been 

receiving treatment with imiglucerase at doses ranging from 15 to 60 Units/kg for a minimum of 

30 consecutive months. Patients were required to have a stable dose of imiglucerase for at least 

6 months prior to study enrolment. Treatment with velaglucerase alfa was administered as the same 

number of units and regimen as their imiglucerase dose. Haemoglobin concentration and platelet 

counts were evaluated as changes from baseline, which was defined as the end of the patient’s 

treatment with imiglucerase. 

 

In patients who switched from imiglucerase to velaglucerase alfa, haemoglobin concentrations and 

platelet counts were sustained at therapeutic levels through 12 months of treatment.  



 

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Study 058 was an open-label clinical safety study in 211 patients including 205 patients previously 

treated with imiglucerase 6 treatment-naïve patients and 57 patients aged 65 years or older (56/57 had 

switched from imiglucerase to velaglucerase alfa). Patients transferring from imiglucerase were 

administered velaglucerase alfa infusions every other week at the same number of units as 

imiglucerase within the range of 15 to 60 Units/kg. Patients transferring from a dose of <15 Units/kg 

imiglucerase were administered 15 Units/kg of velaglucerase alfa.  

 

Patients previously treated with imiglucerase received a median of 8 velaglucerase alfa infusions with 

median duration of treatment of 15.1 weeks. The safety profile in these patients was similar to that 

observed in other clinical studies. Only 1 out of 163 patients assessed developed anti-velaglucerase 

alfa antibodies during the study. 

 

The mean haemoglobin concentration and platelet count of patients previously treated with 

imiglucerase were maintained throughout the study and remained within the reference intervals. 

 

Extension study 044 

 

A total of 95 patients (73 adult and 22 paediatric) who participated in studies 032, 034, and 039 

enrolled in the open label extension study and were treated with velaglucerase alfa. 57 patients were 

treatment naïve. All patients received at least 2 years of ERT and were followed for a mean of 4.5 

years (min. 2.3 years, max 5.8 years). 

 

In this study, haemoglobin concentration, platelet count, liver volume and spleen volume were 

assessed in treatment-naïve patients after 24 months of treatment. The results are presented in Table 2. 

 

Table 2: Results at 24 months - change from baseline – study 044 ITT population 

Clinical parameters Overall velaglucerase 

alfa group (N=39) 

 

 

- 

Mean change from 

baseline (95% CI) 

Patients treated 

with 

imiglucerase 

for 9 months 

and then 

velaglucerase 

alfa for 15 

months (N=16) 

- 

Mean change 

from baseline 

(95% CI) 

Patients who switched 

from long-term 

imiglucerase 

treatment to 

velaglucerase alfa 

(N=38) 

- 

Mean change from 

baseline (95% CI) 

Haemoglobin 

concentration (g/dL) 

2.75 

(2.28, 3.22)  

2.00 

(1.25, 2.75) 

-0.05 

(-0.34, 0.25) 

Platelet count (x109/L) 87.85 

(72.69, 103.00) 

160.94 

(117.22, 204.66) 

9.03 

(-2.60, 20.66) 

Normalised liver volume* 

(%BW) 

-1.21 

(-1.50, -0.91) 

-1.69 

(-2.16, -1.21) 

-0.03 

(-0.10, 0.05) 

Normalised spleen 

volume* 

(%BW)§ 

-2.66 

(-3.50, -1.82) 

-3.63 

(-7.25, - 0.02) 

-0.11 

(-0.19, -0.03) 

§ Excludes patients with splenectomy. N=30, 6 and 34 for the 3 above groups. 

*Liver and spleen volume are normalised as a percentage of body weight. Normal spleen is 

defined as 0.2% of body weight; normal liver as 2.5% of body weight 

Note: Imputation was applied to intermittent missing data. 

 

In this study, BMD was assessed using dual x-ray absorptiometry of the lumbar spine and femoral 

neck. Among 31 treatment-naïve adult patients treated with velaglucerase alfa, the mean lumbar spine 

BMD Z-score at baseline was -1.820 (95% CI: -2.21, -1.43) and increased by 0.62 (95% CI: 0.39, 

0.84) from baseline following 24 months of treatment with velaglucerase alfa. Similar results were 

seen in treatment-naïve patients who received 9 months of imiglucerase followed by velaglucerase alfa 



 

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for 15 months. In patients who switched from long-term imiglucerase to velaglucerase alfa, lumbar 

spine BMD was maintained at 24 months. In contrast, no significant change in femoral neck BMD was 

observed. 

 

In the paediatric population (ages 4 to 17 years studied), increases in the mean height Z-score were 

seen through 60 months of treatment in the overall treatment-naïve population, suggesting a beneficial 

treatment effect with velaglucerase alfa on linear growth. Similar treatment effects were seen through 

48 months in the paediatric population who received 9 months of imiglucerase followed by 

velaglucerase alfa. Paediatric subjects who switched from long-term imiglucerase to velaglucerase alfa 

in study 034 had greater mean height Z-scores at baseline and their mean height Z-scores remained 

stable over time. 

 

These treatment effects on haemoglobin, platelet count, organ volumes, bone mineral density and 

height were maintained through the end of the study. 

 

Paediatric population 

 

Use in the age group 4 to 17 is supported by evidence from controlled studies in adults and paediatric 

[20 of 94 (21%)] patients. The safety and efficacy profiles were similar between paediatric and adult 

patients. The studies allowed the inclusion of patients 2 years and older and the safety and efficacy 

profiles are expected to be similar down to the age of 2 years. However, no data are available for 

children under the age of 4 years. The effect on height was assessed in the study 044 (see section 5.1, 

extension study 044). 

 

Phase I/II study HGT-GCB-068 was conducted to explore the efficacy and safety of velaglucerase alfa 

ERT in treatment naïve children and adolescents with type 3 Gaucher disease. This was a multicentre, 

open-label study in which 60 U/kg of velaglucerase alfa was administered by intravenous infusion 

every other week (EOW) over 12 months in 6 patients (2 to 17 years of age at enrolment) with a 

confirmed diagnosis of type 3 Gaucher disease. 

 

In this small, exploratory study, the non-neurological efficacy findings and the safety profile of 

intravenous velaglucerase alfa in type 3 Gaucher patients were consistent with those observed in 

patients with type 1 Gaucher disease. There was no indication of significant improvements of the 

neurological manifestations of type 3 Gaucher disease except for one patient in this study. 

 

The European Medicines Agency has waived the obligation to submit the results of studies with 

VPRIV in all subsets of the paediatric population in type 2 Gaucher disease (see section 4.2 for 

information on paediatric use).  

 

5.2 Pharmacokinetic properties 

 

There were no apparent pharmacokinetic differences between male and female patients with type 1 

Gaucher disease. None of the subjects in the pharmacokinetic studies were positive for 

anti-velaglucerase alfa antibodies on the days of pharmacokinetic evaluation. Therefore, it was not 

possible to evaluate the effect of antibody response on the pharmacokinetic profile of velaglucerase 

alfa. 

 

Absorption 

 

Velaglucerase alfa serum concentrations rose rapidly for the first 20 minutes of the 60-minute infusion 

before levelling off, and Cmax was typically attained between 40 and 60 minutes after the start of the 

infusion. After the end of the infusion, velaglucerase alfa serum concentrations fell rapidly in a 

monophasic or biphasic fashion with a mean t1/2 ranging from 5 to 12 minutes at doses of 15, 30, 45, 

and 60 Units/kg. 

 

Distribution 

 



 

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Velaglucerase alfa exhibited an approximately linear (i.e. first-order) pharmacokinetic profile, and 

Cmax and AUC increased approximately proportional to the dose over the dose range 15 to 60 Units/kg. 

The steady state volume of distribution was approximately 10% of the body weight. The high 

clearance of velaglucerase alfa from serum (mean 6.7 to 7.6 ml/min/kg) is consistent with the rapid 

uptake of velaglucerase alfa into macrophages via mannose receptors. 

 

Elimination 

 

The range of velaglucerase alfa clearance in paediatric patients (N=7, age range 4 to 17 years) was 

contained within the range of clearance values in adult patients (N=15, age range 19 to 62 years). 

 

5.3 Preclinical safety data 

 

Non-clinical data reveal no special hazard for humans based on conventional studies of safety 

pharmacology, repeated dose toxicity, and toxicity to reproduction and development (see section 4.6). 

 

 

6. PHARMACEUTICAL PARTICULARS 

 

6.1 List of excipients 

 

Sucrose  

Sodium citrate dihydrate (E331) 

Citric acid monohydrate (E330) 

Polysorbate 20 

 

6.2 Incompatibilities 

 

In the absence of compatibility studies, this medicinal product must not be mixed with other medicinal 

products. 

 

6.3 Shelf life 

 

3 years. 

 

Reconstituted and diluted solution for infusion: 

 

Chemical and physical in-use stability has been demonstrated for 24 hours at 2 C to 8 C under 

protection from light. 

 

From a microbiological point of view, the medicinal product should be used immediately. If not used 

immediately, in-use storage times and conditions prior to use are the responsibility of the user and 

must not exceed 24 hours at 2 C to 8 C. 

 

6.4 Special precautions for storage 

 

Store in a refrigerator (2 C - 8 C). 

Do not freeze.  

Keep the vial in the outer carton in order to protect from light. 

 

For storage conditions after reconstitution and dilution of the medicinal product, see section 6.3. 

 

6.5 Nature and contents of container 

 

20 ml vial (type I glass) with a stopper (fluoro-resin coated butyl rubber), one-piece seal, and flip-off 

cap. 

 



 

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Pack sizes of 1, 5 and 25 vials. Each vial contains 400 Units powder for solution for infusion.  

 

Not all pack sizes may be marketed. 

 

6.6 Special precautions for disposal and other handling 

 

VPRIV requires reconstitution and dilution, and is intended for intravenous infusion only. It is for 

single use only and is administered through a 0.22 µm filter.  

 

Aseptic technique must be used. 

 

VPRIV has to be prepared as follows: 

1. The number of vials to be reconstituted is determined based on the individual patient’s weight 

and the prescribed dose.  

2. The required vials are removed from the refrigerator. Each 400 Units vial is reconstituted with 

4.3 ml of sterile water for injections.  

3. Upon reconstitution, vials should be mixed gently. Vials should not be shaken. Each vial will 

contain an extractable volume of 4.0 ml (100 Units/ml). 

4. Prior to further dilution, the solution in the vials should be visually inspected; the solution 

should be clear to slightly opalescent and colourless; the solution should not be used if it is 

discoloured or if foreign particulate matter is present. 

5. The calculated volume of medicinal product is withdrawn from the appropriate number of vials 

and the total volume required is diluted in 100 ml of sodium chloride 9 mg/ml (0.9%) solution 

for infusion. The diluted solution should be mixed gently. It should not be shaken. The infusion 

should be initiated within 24 hours from the time of reconstitution. 

 

Any unused medicinal product or waste material should be disposed of in accordance with local 

requirements. 

 

 

7. MARKETING AUTHORISATION HOLDER 

 

Shire Pharmaceuticals Ireland Limited 

Block 2 & 3 Miesian Plaza 

50 – 58 Baggot Street Lower 

Dublin 2 

Ireland 

 

 

8. MARKETING AUTHORISATION NUMBER(S) 

 

EU/1/10/646/002 

EU/1/10/646/005 

EU/1/10/646/006 

 

 

9. DATE OF FIRST AUTHORISATION/RENEWAL OF THE AUTHORISATION 

 

Date of first authorisation: 26 August 2010 

Date of latest renewal:  

 

 

10. DATE OF REVISION OF THE TEXT 

 

Detailed information on this medicinal product is available on the website of the European Medicines 

Agency http://www.ema.europa.eu 

  

http://www.ema.europa.eu/


 

13 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

ANNEX II 

 

A. MANUFACTURERS OF THE BIOLOGICAL ACTIVE 

SUBSTANCE AND MANUFACTURER RESPONSIBLE FOR 

BATCH RELEASE 

 

B. CONDITIONS OR RESTRICTIONS REGARDING SUPPLY 

AND USE 

 

C. OTHER CONDITIONS AND REQUIREMENTS OF THE 

MARKETING AUTHORISATION 

 

D. CONDITIONS OR RESTRICTIONS WITH REGARD TO 

THE SAFE AND EFFECTIVE USE OF THE MEDICINAL 

PRODUCT 

 



 

14 

A. MANUFACTURERS OF THE BIOLOGICAL ACTIVE SUBSTANCE AND 

MANUFACTURER RESPONSIBLE FOR BATCH RELEASE 

 

Name and address of the manufacturers of the biological active substance 

 

Cell Bank storage and Drug Substance Manufacture  

Shire Human Genetic Therapies, Inc 

205 Alewife Brook Parkway, Cambridge, Massachusetts 02138 

USA 

 

Drug Substance Manufacture 

Shire Human Genetic Therapies, Inc 

400 Shire Way, Lexington, Massachusetts 02421 

USA 

 

Name and address of the manufacturer responsible for batch release 

 

Shire Pharmaceuticals Ireland Limited 

Block 2 & 3 Miesian Plaza 

50 – 58 Baggot Street Lower 

Dublin 2 

Ireland 

 

 

B. CONDITIONS OR RESTRICTIONS REGARDING SUPPLY AND USE 

 

Medicinal product subject to restricted medical prescription (see Annex I: Summary of Product 

Characteristics, section 4.2). 

 

 

C. OTHER CONDITIONS AND REQUIREMENTS OF THE MARKETING 

AUTHORISATION 

 

• Periodic safety update reports (PSURs)  
 

The requirements for submission of PSURs for this medicinal product are set out in the list of Union 

reference dates (EURD list) provided for under Article 107c(7) of Directive 2001/83/EC and any 

subsequent updates published on the European medicines web-portal.  

 

 

D. CONDITIONS OR RESTRICTIONS WITH REGARD TO THE SAFE AND 

EFFECTIVE USE OF THE MEDICINAL PRODUCT  

 

• Risk management plan (RMP) 
 

The marketing authorization holder (MAH) shall perform the required pharmacovigilance 

activities and interventions detailed in the agreed RMP presented in Module 1.8.2 of the 

marketing authorisation and any agreed subsequent updates of the RMP. 

 

An updated RMP should be submitted: 

• At the request of the European Medicines Agency.  

• Whenever the risk management system is modified, especially as the result of new information 
being received that may lead to a significant change to the benefit/risk profile or as the result of 

an important (pharmacovigilance or risk minimisation) milestone being reached. 

 

If the dates of submission of a PSUR and the update of a RMP coincide, they can be submitted at the 

same time. 



 

15 

 

• Additional risk minimisation measures 
 

The MAH must agree on the content and format of the educational materials for use of VPRIV in 

home infusion, including communication media, distribution modalities and any other aspects of the 

programme, with the National Competent Authority. 

 

The educational materials for the use of VPRIV in home infusion are aimed at providing guidance on 

how to manage risk of infusion-related reactions including allergic-type hypersensitivity reactions in a 

home setting. 

 

The MAH shall ensure that in each Member State where VPRIV is marketed, all healthcare 

professionals and patients/carers who are expected to prescribe, dispense or use VPRIV have access 

to/are provided with the following educational package: 

• Manual for patients with Gaucher Disease who receive home infusion; 

• Guide for Healthcare Professionals Treating Patients with Gaucher Disease; 

• Infusion diary; 

• Emergency plan provided by the prescriber including who to call and what to do in the case of a 
serious hypersensitivity reaction. 

 

The manual for patients with Gaucher Disease who receive home infusion should contain the 

following key elements: 

• About the disease, treatment and home infusion; 

• Organisation: Home environment/Caregiver/Treating physician; 

• Description on how to prepare and administer the infusion including safety information; 

• Annex: Package Leaflet, Infusion diary, Patient Reconstitution Guide, Emergency plan. 
 

The Guide for Healthcare Professionals Treating Patients with Gaucher Disease should contain the 

following key elements: 

• Patient evaluation and selection; 

• Requirements for home infusion (home condition, patient assessment, emergency treatment); 

• Organisation of home infusion/preparation of infusion; 

• Safety information; 

• Annex: SmPC; Healthcare Professional Reconstitution Guide, Infusion diary 
  



 

16 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

ANNEX III 

 

LABELLING AND PACKAGE LEAFLET 

  



 

17 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

A. LABELLING 

  



 

18 

PARTICULARS TO APPEAR ON THE OUTER PACKAGING  

 

OUTER CARTON – 400 UNITS (1 vial pack) 

 

 

1. NAME OF THE MEDICINAL PRODUCT 

 

VPRIV 400 Units powder for solution for infusion 

velaglucerase alfa  

 

 

2. STATEMENT OF ACTIVE SUBSTANCE(S) 

 

Each vial contains 400 Units of velaglucerase alfa. 

After reconstitution, one ml of the solution contains 100 Units of velaglucerase alfa. 

 

 

3. LIST OF EXCIPIENTS 

 

Also contains: 

Sucrose 

Sodium citrate dihydrate 

Citric acid monohydrate 

Polysorbate 20 

See leaflet for further information. 

 

 

4. PHARMACEUTICAL FORM AND CONTENTS 

 

Powder for solution for infusion 

1 vial 

 

 

5. METHOD AND ROUTE(S) OF ADMINISTRATION 

 

Single use only. 

Read the package leaflet before use. 

Intravenous use. 

 

 

6. SPECIAL WARNING THAT THE MEDICINAL PRODUCT MUST BE STORED OUT 

OF THE SIGHT AND REACH OF CHILDREN 

 

Keep out of the sight and reach of children. 

 

 

7. OTHER SPECIAL WARNING(S), IF NECESSARY 

 

 

8. EXPIRY DATE 

 

EXP  

 



 

19 

9. SPECIAL STORAGE CONDITIONS 

 

Store in a refrigerator.  

Do not freeze. 

Keep the vial in the outer carton in order to protect from light. 

 

 

10. SPECIAL PRECAUTIONS FOR DISPOSAL OF UNUSED MEDICINAL PRODUCTS 

OR WASTE MATERIALS DERIVED FROM SUCH MEDICINAL PRODUCTS, IF 

APPROPRIATE 

 

 

11. NAME AND ADDRESS OF THE MARKETING AUTHORISATION HOLDER 

 

Shire Pharmaceuticals Ireland Limited 

Block 2 & 3 Miesian Plaza 

50 – 58 Baggot Street Lower 

Dublin 2 

Ireland 

 

 

12. MARKETING AUTHORISATION NUMBER(S)  

 

EU/1/10/646/002 

 

 

13. BATCH NUMBER 

 

Lot 

 

 

14. GENERAL CLASSIFICATION FOR SUPPLY 

 

  

15. INSTRUCTIONS ON USE 

 

 

16. INFORMATION IN BRAILLE 

 

Justification for not including Braille accepted 

 

 

17. UNIQUE IDENTIFIER – 2D BARCODE 

 

2D barcode carrying the unique identifier included 

 

 

18. UNIQUE IDENTIFIER – HUMAN READABLE DATA 

 

PC 

SN 

NN 



 

20 

PARTICULARS TO APPEAR ON THE OUTER PACKAGING  

 

OUTER CARTON – 400 UNITS (5 vial pack) 

 

 

1. NAME OF THE MEDICINAL PRODUCT 

 

VPRIV 400 Units powder for solution for infusion 

velaglucerase alfa  

 

 

2. STATEMENT OF ACTIVE SUBSTANCE(S) 

 

Each vial contains 400 Units of velaglucerase alfa.  

After reconstitution, one ml of the solution contains 100 Units of velaglucerase alfa. 

 

 

3. LIST OF EXCIPIENTS 

 

Also contains: 

Sucrose 

Sodium citrate dihydrate 

Citric acid monohydrate 

Polysorbate 20 

See leaflet for further information. 

 

 

4. PHARMACEUTICAL FORM AND CONTENTS 

 

Powder for solution for infusion 

5 vials 

 

 

5. METHOD AND ROUTE(S) OF ADMINISTRATION 

 

Single use only. 

Read the package leaflet before use. 

Intravenous use. 

 

 

6. SPECIAL WARNING THAT THE MEDICINAL PRODUCT MUST BE STORED OUT 

OF THE SIGHT AND REACH OF CHILDREN 

 

Keep out of the sight and reach of children. 

 

 

7. OTHER SPECIAL WARNING(S), IF NECESSARY 

 

 

8. EXPIRY DATE 

 

EXP  

 



 

21 

9. SPECIAL STORAGE CONDITIONS 

 

Store in a refrigerator.  

Do not freeze. 

Keep the vial in the outer carton in order to protect from light. 

 

 

10. SPECIAL PRECAUTIONS FOR DISPOSAL OF UNUSED MEDICINAL PRODUCTS 

OR WASTE MATERIALS DERIVED FROM SUCH MEDICINAL PRODUCTS, IF 

APPROPRIATE 

 

 

11. NAME AND ADDRESS OF THE MARKETING AUTHORISATION HOLDER 

 

Shire Pharmaceuticals Ireland Limited 

Block 2 & 3 Miesian Plaza 

50 – 58 Baggot Street Lower 

Dublin 2 

Ireland 

 

 

12. MARKETING AUTHORISATION NUMBER(S)  

 

EU/1/10/646/005 

 

 

13. BATCH NUMBER 

 

Lot 

 

 

14. GENERAL CLASSIFICATION FOR SUPPLY 

 

  

15. INSTRUCTIONS ON USE 

 

 

16. INFORMATION IN BRAILLE 

 

Justification for not including Braille accepted 

 

 

17. UNIQUE IDENTIFIER – 2D BARCODE 

 

2D barcode carrying the unique identifier included 

 

 

18. UNIQUE IDENTIFIER – HUMAN READABLE DATA 

 

PC 

SN 

NN 



 

22 

PARTICULARS TO APPEAR ON THE OUTER PACKAGING 

 

OUTER CARTON – 400 UNITS (25 vial pack) 

 

 

1. NAME OF THE MEDICINAL PRODUCT 

 

VPRIV 400 Units powder for solution for infusion 

velaglucerase alfa  

 

 

2. STATEMENT OF ACTIVE SUBSTANCE(S) 

 

Each vial contains 400 Units of velaglucerase alfa.  

After reconstitution, one ml of the solution contains 100 Units of velaglucerase alfa. 

 

 

3. LIST OF EXCIPIENTS 

 

Also contains: 

Sucrose 

Sodium citrate dihydrate 

Citric acid monohydrate 

Polysorbate 20 

See leaflet for further information. 

 

 

4. PHARMACEUTICAL FORM AND CONTENTS 

 

Powder for solution for infusion 

25 vials 

 

 

5. METHOD AND ROUTE(S) OF ADMINISTRATION 

 

Single use only. 

Read the package leaflet before use. 

Intravenous use. 

 

 

6. SPECIAL WARNING THAT THE MEDICINAL PRODUCT MUST BE STORED OUT 

OF THE SIGHT AND REACH OF CHILDREN 

 

Keep out of the sight and reach of children. 

 

 

7. OTHER SPECIAL WARNING(S), IF NECESSARY 

 

 

8. EXPIRY DATE 

 

EXP  

 



 

23 

9. SPECIAL STORAGE CONDITIONS 

 

Store in a refrigerator. 

Do not freeze. 

Keep the vial in the outer carton in order to protect from light. 

 

 

10. SPECIAL PRECAUTIONS FOR DISPOSAL OF UNUSED MEDICINAL PRODUCTS 

OR WASTE MATERIALS DERIVED FROM SUCH MEDICINAL PRODUCTS, IF 

APPROPRIATE 

 

 

11. NAME AND ADDRESS OF THE MARKETING AUTHORISATION HOLDER 

 

Shire Pharmaceuticals Ireland Limited 

Block 2 & 3 Miesian Plaza 

50 – 58 Baggot Street Lower 

Dublin 2 

Ireland 

 

 

12. MARKETING AUTHORISATION NUMBER(S)  

 

EU/1/10/646/006 

 

 

13. BATCH NUMBER 

 

Lot 

 

 

14. GENERAL CLASSIFICATION FOR SUPPLY 

 

  

15. INSTRUCTIONS ON USE 

 

 

16. INFORMATION IN BRAILLE 

 

Justification for not including Braille accepted 

 

 

17. UNIQUE IDENTIFIER – 2D BARCODE 

 

2D barcode carrying the unique identifier included 

 

 

18. UNIQUE IDENTIFIER – HUMAN READABLE DATA 

 

PC 

SN 

NN 



 

24 

MINIMUM PARTICULARS TO APPEAR ON SMALL IMMEDIATE PACKAGING UNITS 

 

VIAL – 400 UNITS 

 

 

1. NAME OF THE MEDICINAL PRODUCT AND ROUTE(S) OF ADMINISTRATION 

 

VPRIV 400 Units powder for solution for infusion 

velaglucerase alfa 

Intravenous use  

 

 

2. METHOD OF ADMINISTRATION 

 

Read the package leaflet before use. 

 

 

3. EXPIRY DATE 

 

EXP  

 

 

4. BATCH NUMBER 

 

Lot  

 

 

5. CONTENTS BY WEIGHT, BY VOLUME OR BY UNIT 

 

 

6. OTHER 

 

 

  



 

25 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

B. PACKAGE LEAFLET 

 



 

26 

Package leaflet: Information for the user 

 

VPRIV 400 Units powder for solution for infusion 

velaglucerase alfa  

  

Read all of this leaflet carefully before you start using this medicine because it contains 

important information for you. 

 

- Keep this leaflet. You may need to read it again. 
- If you have any further questions, ask your doctor. 
- If you get any side effects, talk to your doctor. This includes any possible side effects not listed 

in this leaflet (see section 4).  

 

What is in this leaflet  

 

1. What VPRIV is and what it is used for 

2. What you need to know before VPRIV is used 

3. How VPRIV is used 

4. Possible side effects 

5. How to store VPRIV 
6. Contents of the pack and other information 

 

 

1. What VPRIV is and what it is used for 

 

VPRIV is a long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease. 

 

Gaucher disease is a genetic disorder caused by a missing or defective enzyme named 

glucocerebrosidase. When this enzyme is missing or does not work properly, a substance called 

glucocerebroside builds up inside cells in the body. The build-up of this material causes the signs and 

symptoms found in Gaucher disease. 

 

VPRIV contains a substance called velaglucerase alfa which is designed to replace the missing or 

defective enzyme, glucocerebrosidase, in patients with Gaucher disease. 

 

 

2. What you need to know before VPRIV is used 

 

Do not use VPRIV  

- if you are severely allergic to velaglucerase alfa or any of the other ingredients of this medicine 
(listed in section 6). 

 

Warnings and precautions 

Talk to your doctor before VPRIV is used 

- If you are treated with VPRIV, you may experience side effects during or following the infusion 
(see section 4, possible side effects). These are called infusion related reactions and might 

appear as a hypersensitivity reaction with symptoms like nausea, rash, difficulty in breathing, 

back pain, chest discomfort (chest tightness), hives, joint pain or headache. 

- Apart from symptoms of hypersensitivity reactions infusion-related reactions might show as 
dizziness, high blood pressure, tiredness, fever, itching, or blurry vision. 

If you experience any of the symptoms, you must tell your doctor immediately. 

- You may be given additional medicines to treat or help prevent future reactions. These 
medicines may include antihistamines, antipyretics, and corticosteroids. 

- If the reaction is severe, your doctor will stop the intravenous infusion immediately and start 
giving you appropriate medical treatment. 

- If the reactions are severe and/or there is a loss of effect from this medicine, your doctor will 
perform a blood test to check for antibodies which may affect the outcome of your treatment  



 

27 

- Your doctor or nurse may decide to continue to administer VPRIV even if you experience any 
infusion related-reaction. Your condition will be closely monitored. 

 

Tell your doctor if you have previously experienced an infusion-related reaction with other ERT for 

Gaucher disease. 

 

Children 

VPRIV should not be used in children under the age of 4 years because there is no experience of using 

the medicine in this age group. 

 

Other medicines and VPRIV 

Tell your doctor if you are taking, have recently taken or might take any other medicines.  

 

Pregnancy  

Gaucher disease may become more active in a woman during pregnancy and for a few weeks after 

birth. Women with Gaucher disease who are pregnant or considering pregnancy should talk with their 

doctor before this medicine is used. 

 

Breast-feeding 

It is not known whether VPRIV can pass into breast milk. If you are breast-feeding or considering 

breast-feeding, you should talk to your doctor before this medicine is used. Your doctor will then help 

you decide whether to stop breast-feeding, or whether to stop taking VPRIV, considering the benefit 

of breast-feeding to the baby and the benefit of VPRIV to the mother. 

 

Driving and using machines 

VPRIV has no or negligible influence on your ability to drive or use machines. 

 

VPRIV contains sodium 

This medicine contains 12.15 mg sodium (main component of cooking/table salt) in each vial. This is 

equivalent to 0.6% of the recommended maximum daily dietary intake of sodium for an adult. 

  

   

3. How VPRIV is used 

 

This medicine is only to be used under appropriate medical supervision of a doctor who is 

knowledgeable in the treatment of Gaucher disease. It is given by a doctor or nurse by intravenous 

infusion. 

 

Dose 

The recommended dose is 60 Units/kg given every other week.  

 

If you are currently being treated for Gaucher disease with another ERT and your doctor wants to 

change you to VPRIV, you can initially receive VPRIV at the same dose and frequency you had been 

receiving the other ERT.  

 

Use in children and adolescents 

VPRIV may be given to children and adolescents (4 to 17 years of age) at the same dose and 

frequency as in adults.  

 

Use in elderly 

VPRIV may be given to the elderly (aged over 65 years) at the same dose and frequency as in adults. 

 

Response to treatment 

Your doctor will monitor your response to treatment and may change your dose (up or down) over 

time. 

 



 

28 

If you are tolerating your infusions well in the clinic, your doctor or nurse may administer your 

infusions at home. 

 

Administration 

VPRIV is supplied in a vial as a packed powder which is mixed with sterile water and further diluted 

in sodium chloride 9 mg/ml (0.9%) solution for infusion prior to intravenous infusion.  

 

After preparation, your doctor or nurse will give the medicine to you through a drip into a vein (by 

intravenous infusion) over a period of 60 minutes.  

 

If you have any further questions on the use of this medicine, ask your doctor. 

 

 

4. Possible side effects 

 

Like all medicines, this medicine can cause side effects, although not everybody gets them. 

 

Commonly (may affect up to 1 in 10 people), patients experienced a severe allergic reaction, with 

difficulty breathing, chest discomfort (chest tightness), feeling sick (nausea), swelling of the face, lips, 

tongue or throat (anaphylactic/anaphylactoid reactions), common is also an allergic skin reaction such 

as hives, severe rash or itching. If any of these happen tell your doctor immediately. 

 

Most side effects, including the allergic reactions, occurred during the infusion or shortly after. These 

are called infusion related reactions. Other infusion related reactions that occurred very commonly 

(may affect more than 1 in 10 people) include headache, dizziness, fever/body temperature increased, 

back pain, joint pain and tiredness, as well as high blood pressure (commonly reported), and blurry 

vision (uncommonly reported). If any of these happen tell your doctor immediately. 

 

Other side effects include: 

 

Very common side effects (may affect more than 1 in 10 people) are: 

- bone pain 
- weakness/loss of strength 
- stomach ache 
 

Common side effects (may affect up to 1 in 10 people) are: 

- lengthening of the time it takes for a cut to stop bleeding may lead to easy/spontaneous 
bleeding/easy bruising 

- skin flushing 
- rapid heart beat 
- developing antibodies to VPRIV (see section 2) 
- decreased blood pressure  
 

Uncommon side effects (may affect up to 1 in 100 people) are: 

- vomiting 
 

Reporting of side effects 

If you get any side effects, talk to your doctor or pharmacist. This includes any possible side effects 

not listed in this leaflet. You can also report side effects directly via the national reporting system 

listed in Appendix V. By reporting side effects you can help provide more information on the safety of 

this medicine. 

 

 

http://www.ema.europa.eu/docs/en_GB/document_library/Template_or_form/2013/03/WC500139752.doc


 

29 

5. How to store VPRIV 

 

Keep this medicine out of the sight and reach of children. 

 

Do not use this medicine after the expiry date which is stated on the carton and label after ‘EXP’. The 

expiry date refers to the last day of that month. 

 

Store in the refrigerator (2 ºC – 8 ºC). 

Do not freeze. 

Keep the vial in the outer carton in order to protect from light.  

Do not use if the solution is discoloured or if foreign particles are present.  

 

Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to 

throwaway medicines you no longer use. These measures will help to protect the environment. 

 

 

6. Contents of the pack and other information 

 

What VPRIV contains 

- The active substance is velaglucerase alfa. 
 Each vial contains 400 Units of velaglucerase alfa. 

 After reconstitution, one ml of solution contains 100 Units of velaglucerase alfa 

 

- The other ingredients are sucrose, sodium citrate dihydrate, citric acid monohydrate and 
polysorbate 20 (see section 2 “VPRIV contains sodium”). 

 

What VPRIV looks like and contents of the pack 

20 ml glass vial containing a white to off-white powder for solution for infusion.  

 

Packs of 1, 5 or 25 vials. 

Not all pack sizes may be marketed. 

 

Marketing Authorisation Holder and Manufacturer 

Shire Pharmaceuticals Ireland Limited 

Block 2 & 3 Miesian Plaza 

50 – 58 Baggot Street Lower 

Dublin 2 

Ireland 

Tel: + 800 66838470 

E-mail: medinfoEMEA@shire.com 

 

This leaflet was last revised in  

 

Other sources of information 

 

Detailed information on this medicine is available on the European Medicines Agency web site: 

http://www.ema.europa.eu. There are also links to other websites about rare diseases and treatments. 

 

------------------------------------------------------------------------------------------------------------------------ 

 

The following information is intended for healthcare professionals only. 

 

VPRIV is a powder for solution for infusion. It requires reconstitution and dilution and is intended for 

intravenous infusion only. VPRIV is for single–use only and is administered through a 0.22 µm filter. 

Discard any unused solution. VPRIV should not be infused with other medicines in the same infusion 

mailto:medinfoEMEA@shire.com
http://www.ema.europa.eu/


 

30 

as the compatibility in solution with other medicines has not been evaluated. The total volume of 

infusion should be delivered over a period of 60 minutes.  

 

Use aseptic technique. 

Prepare VPRIV as follows: 

1. The number of vials to be reconstituted is determined based on the individual patient’s weight 
and the prescribed dose. 

2. The required vials are removed from the refrigerator. Each vial is reconstituted using sterile 
water for injections: 

Vial size Water for Injections 

400 Units 4.3 ml 

 

3. Upon reconstitution, mix vials gently. Do not shake. 
4. Prior to dilution, visually inspect the solution in the vials; the solution should be clear to slightly 

opalescent and colourless; do not use if the solution is discoloured, or if foreign particulate 

matter is present. 

5. The calculated volume of medicinal product is withdrawn from the appropriate number of vials. 
Some solution will remain in the vial: 

Vial size Extractable volume 

400 Units 4.0 ml 

 

6. The total volume required is diluted in 100 ml of sodium chloride 9 mg/ml (0.9%) solution for 
infusion. Mix gently. Do not shake. The infusion should be initiated within 24 hours from the 

time of reconstitution. 

 

From a microbiological point of view, the medicinal product should be used immediately. If not used 

immediately, in-use storage times and conditions prior to use are the responsibility of the user and 

must not exceed 24 hours at 2 C to 8 C. 

 

Medicines should not be disposed of via waste water or household waste. Any unused medicine or 

waste material should be disposed of in accordance with local requirements. 

 

Keeping a record 

In order to improve the traceability of biological medicinal products, the name and batch number of 

the administered product should be clearly recorded. 

 


	SUMMARY OF PRODUCT CHARACTERISTICS
	A. MANUFACTURERS OF THE BIOLOGICAL ACTIVE SUBSTANCE AND MANUFACTURER RESPONSIBLE FOR BATCH RELEASE
	B. CONDITIONS OR RESTRICTIONS REGARDING SUPPLY AND USE
	C. OTHER CONDITIONS AND REQUIREMENTS OF THE MARKETING AUTHORISATION
	D. CONDITIONS OR RESTRICTIONS WITH REGARD TO THE SAFE AND EFFECTIVE USE OF THE MEDICINAL PRODUCT
	A. LABELLING
	B. PACKAGE LEAFLET