Drugs List

Therapeutic Indications

Uses

Enzyme replacement therapy in patients with Type 1 Gaucher disease

Administration

For intravenous infusion.

Contraindications

Children under 4 years
Hereditary fructose intolerance

Precautions and Warnings

Restricted sodium intake
Breastfeeding
Glucose-galactose malabsorption syndrome
Pregnancy

Sodium content of formulation may be significant
Consider premedication with antihistamine and/or corticosteroid
Treatment to be initiated and supervised by a specialist
Preparation contains sucrose
Do not use if solution is discoloured or particulates are apparent
Record name and batch number of administered product
Resuscitation facilities must be immediately available
Monitor for hypersensitivity reactions during infusion
Monitor for the presence of antibodies
Advise patient to stop therapy & contact Dr if hypersensitivity signs occur
Discontinue if serious allergic or anaphylactic reaction occurs

Home administration under the supervision of a healthcare professional may be considered only for patients who have received at least three infusions and were tolerating them well.

In clinical studies only 1% of the patients develop IgG antibodies, and no patients developed IgE antibodies to velaglucerase alfa. In case of severe infusion related reactions and lack or loss of effect, the patient should be evaluated for the presence of antibodies and the results reported.

The majority of hypersensitivity reactions usually occur up to 12 hours post infusion. The most frequently reported hypersensitivity reactions include nausea, rash, dyspnoea, back pain, chest discomfort, urticaria, arthralgia and headache. Vomiting as a side effect can be serious and severe and often occurs during the infusion and for 24 hours after the infusion.

Pregnancy and Lactation

Pregnancy

Use velaglucerase alfa with caution during pregnancy.

The manufacturer recommends that pregnant women may experience a period of increased disease activity, therefore a risk-benefit assessment for each pregnancy is required. Close monitoring of the pregnancy and clinical manifestations of Gaucher disease is needed for the individualisation of therapy.

At the time of writing there is limited published data regarding the use of velaglucerase alfa during pregnancy. The potential risk is unknown.

Lactation

Use velaglucerase alfa with caution during breastfeeding.

The manufacturer advises that at the time of publication there had been no studies in breastfeeding women. It is unknown whether velaglucerase alfa is excreted into breast milk. The effects on exposed infants are unknown.

Side Effects

Abdominal pain
Allergic dermatitis
Anaphylactic reaction
Anaphylactoid reaction
Antibody formation
Arthralgia
Asthenia
Back pain
Blurred vision
Bone pain
Chest discomfort
Dizziness
Dyspnoea
Fatigue
Flushing
Headache
Hypersensitivity reactions
Hypertension
Hypotension
Increased partial thromboplastin time
Infusion-related symptoms
Nausea
Pruritus
Pyrexia
Rash
Rise in body temperature
Tachycardia
Upper abdominal pain
Urticaria
Vomiting

Presentation

Parenteral formulations of velaglucerase alfa.

Drugs List

Therapeutic Indications

Uses

Enzyme replacement therapy in patients with Type 1 Gaucher disease

Dosage

Dosage should be individualised for each patient.

Patients switching from imiglucerase enzyme replacement therapy for type 1 Gaucher disease to velaglucerase alfa, should use the same dose and frequency.

Adults

Children

Administration

For intravenous infusion.

Contraindications

Children under 4 years
Hereditary fructose intolerance

Precautions and Warnings

Restricted sodium intake
Breastfeeding
Glucose-galactose malabsorption syndrome
Pregnancy

Sodium content of formulation may be significant
Consider premedication with antihistamine and/or corticosteroid
Treatment to be initiated and supervised by a specialist
Preparation contains sucrose
Do not use if solution is discoloured or particulates are apparent
Record name and batch number of administered product
Resuscitation facilities must be immediately available
Monitor for hypersensitivity reactions during infusion
Monitor for the presence of antibodies
Advise patient to stop therapy & contact Dr if hypersensitivity signs occur
Discontinue if serious allergic or anaphylactic reaction occurs

Home administration under the supervision of a healthcare professional may be considered only for patients who have received at least three infusions and were tolerating them well.

In clinical studies only 1% of the patients develop IgG antibodies, and no patients developed IgE antibodies to velaglucerase alfa. In case of severe infusion related reactions and lack or loss of effect, the patient should be evaluated for the presence of antibodies and the results reported.

The majority of hypersensitivity reactions usually occur up to 12 hours post infusion. The most frequently reported hypersensitivity reactions include nausea, rash, dyspnoea, back pain, chest discomfort, urticaria, arthralgia and headache. Vomiting as a side effect can be serious and severe and often occurs during the infusion and for 24 hours after the infusion.

Pregnancy and Lactation

Pregnancy

Use velaglucerase alfa with caution during pregnancy.

The manufacturer recommends that pregnant women may experience a period of increased disease activity, therefore a risk-benefit assessment for each pregnancy is required. Close monitoring of the pregnancy and clinical manifestations of Gaucher disease is needed for the individualisation of therapy.

At the time of writing there is limited published data regarding the use of velaglucerase alfa during pregnancy. The potential risk is unknown.

Lactation

Use velaglucerase alfa with caution during breastfeeding.

The manufacturer advises that at the time of publication there had been no studies in breastfeeding women. It is unknown whether velaglucerase alfa is excreted into breast milk. The effects on exposed infants are unknown.

Side Effects

Abdominal pain
Allergic dermatitis
Anaphylactic reaction
Anaphylactoid reaction
Antibody formation
Arthralgia
Asthenia
Back pain
Blurred vision
Bone pain
Chest discomfort
Dizziness
Dyspnoea
Fatigue
Flushing
Headache
Hypersensitivity reactions
Hypertension
Hypotension
Increased partial thromboplastin time
Infusion-related symptoms
Nausea
Pruritus
Pyrexia
Rash
Rise in body temperature
Tachycardia
Upper abdominal pain
Urticaria
Vomiting

Overdosage

It is strongly recommended that the UK National Poisons Information Service be consulted on cases of suspected or actual overdose where there is doubt over the degree of risk or about appropriate management.

The following number will direct the caller to the relevant local centre (0844) 892 0111

Information may be obtained if you have access to ToxBase the primary clinical toxicology database of the National Poisons Information Service. This is available via password on the internet ( www.toxbase.org ) or if this is unavailable at the backup site ( www.toxbasebackup.org ).

Further Information

Last Full Review Date: November 2019

Reference Sources

Summary of Product Characteristics: VPRIV 400 Units powder for solution for infusion. Shire Pharmaceuticals Ireland Limited. Revised July 2020.

NICE Evidence Services Available at: www.nice.org.uk Last accessed: 09 November 2019.