Drugs List

Therapeutic Indications

Uses

Haemolytic anaemia in sickle cell disease

Treatment of haemolytic anaemia due to sickle cell disease (HbSS) in adults and children aged 12 years and older as monotherapy or in combination with hydroxycarbamide.

Contraindications

Children under 12 years
Breastfeeding
Pregnancy

Precautions and Warnings

Immunosuppression
Patients over 65 years
End stage renal disease
Primary or secondary immunodeficiencies
Severe hepatic impairment

Reduce dose in patients with severe hepatic impairment
Combination therapy: refer to information for concomitant agent
Treatment to be prescribed under the supervision of a specialist
Contains polyethylene glycol
May affect results of some laboratory tests
Discontinue permanently if severe hypersensitivity reactions occur
Advise patient not to take St John's wort concurrently

Safety and efficacy in sickle cell disease subtypes other than genotype HbSS or sickle beta-zero thalassemia are limited.

Pregnancy and Lactation

Pregnancy

Voxelotor is contraindicated during pregnancy.

The manufacturer advises that it is preferable to avoid the use of voxelotor during pregnancy. Animal data do not indicate direct or indirect harmful effects with respect to reproductive toxicity. The high protein binding of voxelotor (99.8% in vitro) should limit the embryo-fetal exposure. At the time of writing there are no or limited data from the use of voxelotor in pregnant women. The potential risk is unknown.

Lactation

Voxelotor is contraindicated during breastfeeding.

The manufacturer does not recommend voxelotor during breastfeeding. Available data in animals have shown excretion of voxelotor in milk. It is unknown whether voxelotor or it metabolites are excreted in human milk. A risk to the newborn or infant cannot be excluded.

Side Effects

Abdominal pain
Diarrhoea
Eosinophilia
Facial swelling
Gastro-intestinal discomfort
Headache
Hypersensitivity reactions
Macular rash
Nausea
Papular eruption
Pruritic rash
Rash
Shortness of breath
Urticaria

Presentation

Oral formulations of voxelotor.

Drugs List

Therapeutic Indications

Uses

Haemolytic anaemia in sickle cell disease

Treatment of haemolytic anaemia due to sickle cell disease (HbSS) in adults and children aged 12 years and older as monotherapy or in combination with hydroxycarbamide.

Dosage

Adults

Children

Patients with Hepatic Impairment

Contraindications

Children under 12 years
Breastfeeding
Pregnancy

Precautions and Warnings

Immunosuppression
Patients over 65 years
End stage renal disease
Primary or secondary immunodeficiencies
Severe hepatic impairment

Reduce dose in patients with severe hepatic impairment
Combination therapy: refer to information for concomitant agent
Treatment to be prescribed under the supervision of a specialist
Contains polyethylene glycol
May affect results of some laboratory tests
Discontinue permanently if severe hypersensitivity reactions occur
Advise patient not to take St John's wort concurrently

Safety and efficacy in sickle cell disease subtypes other than genotype HbSS or sickle beta-zero thalassemia are limited.

Pregnancy and Lactation

Pregnancy

Voxelotor is contraindicated during pregnancy.

The manufacturer advises that it is preferable to avoid the use of voxelotor during pregnancy. Animal data do not indicate direct or indirect harmful effects with respect to reproductive toxicity. The high protein binding of voxelotor (99.8% in vitro) should limit the embryo-fetal exposure. At the time of writing there are no or limited data from the use of voxelotor in pregnant women. The potential risk is unknown.

Lactation

Voxelotor is contraindicated during breastfeeding.

The manufacturer does not recommend voxelotor during breastfeeding. Available data in animals have shown excretion of voxelotor in milk. It is unknown whether voxelotor or it metabolites are excreted in human milk. A risk to the newborn or infant cannot be excluded.

Side Effects

Abdominal pain
Diarrhoea
Eosinophilia
Facial swelling
Gastro-intestinal discomfort
Headache
Hypersensitivity reactions
Macular rash
Nausea
Papular eruption
Pruritic rash
Rash
Shortness of breath
Urticaria

Effects on Laboratory Tests

Voxelotor may interfere with the measurement of haemoglobin subtypes by high-performance liquid chromatography. If precise quantitation of Hb species is required, chromatography should be performed when the patient has not received voxelotor in the preceding 10 days.

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: October 2022

Reference Sources

Summary of Product Characteristics: Oxbryta 500mg film-coated tablets. Global Blood Therapeutics UK Limited. Revised July 2022.

NICE Evidence Services Available at: www.nice.org.uk Last accessed: 04 January 2023