Regeneron Pharmaceuticals has announced that its Praluent (alirocumab) injection has received extended approval from the US Food and Drug Administration (FDA) to include paediatric patients with heterozygous familial hypercholesterolaemia (HeFH).
Paediatric patients aged eight years or older with HeFH will be eligible to receive Praluent as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies.
Caused by mutations in one of several genes that control how the body processes cholesterol, familial hypercholesterolaemia (FH) is an inherited condition that can lead to very high levels of LDL-C.
HeFH develops when one mutated gene is inherited from one parent and homozygous FH, the second form of FH, develops when a mutated gene is inherited from both parents.
The decision was based on results from a phase 3 randomised multicentre trial, evaluating patients aged eight to 17 years with HeFH who had LDL-C levels of 130mg or greater and were already receiving treatment with lipid-lowering medications.
Randomised to receive either Praluent or placebo every two or four weeks in two consecutive cohorts, patients who received Praluent every four weeks had 31% lower LDL-C compared to placebo at 24 weeks.
Published in the Journal of the American Medical Association Pediatrics, the study also showed improvements in additional key lipid parameters, with no new adverse reactions identified and the safety profile remaining consistent with the safety profile observed in adults with HeFH.
George Yancopoulos, board co-chair, president and chief scientific officer, Regeneron, commented: “Praluent has made a meaningful impact in the treatment of adults with FH and… will now be able to help appropriate children with the heterozygous form of this disease manage their dangerously high levels of LDL-C.”
Mary McGowan, chief medical officer, Family Heart Foundation, said, for “many children with HeFH… whose LDL-C remains dangerously high, this approval is an important milestone as it gives these children… an additional option to help reduce and manage their LDL-C levels much earlier in their lives”.
Currently approved in 60 countries, including the US, EU, Japan, Canada, Switzerland and Brazil, Praluent works to inhibit the binding of PCSK9 to the LDL receptor, increasing the number of available LDL receptors on the surface of liver cells to clear LDL, which lowers LDL-C in the blood.