Heterozygous familial hypercholesterolaemia (HeFH)

Active Ingredient: Alirocumab

Indication for Alirocumab

Population group: only children (1 year - 12 years old) , adolescents (12 years - 18 years old)
Therapeutic intent: Curative procedure

Alirocumab is indicated in paediatric patients 8 years of age and older with heterozygous familial hypercholesterolaemia (HeFH) as an adjunct to diet:

  • in combination with a statin or statin with other lipid lowering therapies in patients unable to reach LDL-C goals with the maximum tolerated dose of a statin or,
  • alone or in combination with other lipid-lowering therapies in patients who are statin-intolerant, or for whom a statin is contraindicated.

For this indication, competent medicine agencies globally authorize below treatments:

For patients weighting <50 kg 150 mg once every 4 weeks or 75 mg once every 2 weeks and for patients weighting ≥50 kg 300 mg once every 4 weeks or 150 mg once every 2 weeks

For:

Dosage regimens

Regimen A, in case that patient age in years is ≥ 8 and patient weight is < 50 kg

Subcutaneous, 150 milligrams alirocumab, once every 4 weeks.

Regimen B, in case that patient age in years is ≥ 8 and patient weight is < 50 kg

Subcutaneous, 75 milligrams alirocumab, once every 2 weeks.

Regimen C, in case that patient age in years is ≥ 8 and patient weight is ≥ 50 kg

Subcutaneous, 300 milligrams alirocumab, once every 4 weeks.

Regimen D, in case that patient age in years is ≥ 8 and patient weight is ≥ 50 kg

Subcutaneous, 150 milligrams alirocumab, once every 2 weeks.

Detailed description

HeFH in paediatric patients 8 years of age and older

Body weight of patientsRecommended doseRecommended dose if additional
LDL-C reduction is needed*
Less than 50 kg150 mg once every 4 weeks75 mg once every 2 weeks
50 kg or more300 mg once every 4 weeks150 mg once every 2 weeks

* Lipid levels can be assessed 8 weeks after treatment initiation or titration and dose adjusted accordingly.

Missed dose

If a dose is missed, the dose should be administered as soon as possible and thereafter, dosing should be resumed on the original schedule.

Dosage considerations

Alirocumab is injected as a subcutaneous injection into the thigh, abdomen or upper arm.

It is recommended to rotate the injection site with each injection.

Alirocumab should not be injected into areas of active skin disease or injury such as sunburns, skin rashes, inflammation, or skin infections.

Alirocumab must not be co-administered with other injectable medicinal products at the same injection site.

Active ingredient

Alirocumab

Alirocumab is a fully human IgG1 monoclonal antibody that binds with high affinity and specificity to proprotein convertase subtilisin kexin type 9 (PCSK9). PCSK9 binds to the low-density lipoprotein receptors (LDLR) on the surface of hepatocytes to promote LDLR degradation within the liver. LDLR is the primary receptor that clears circulating LDL, therefore the decrease in LDLR levels by PCSK9 results in higher blood levels of LDL-C. By inhibiting the binding of PCSK9 to LDLR, alirocumab increases the number of LDLRs available to clear LDL, thereby lowering LDL-C levels.

Read more about Alirocumab

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