FDA Approves Praluent for Children with Genetic Form of High Cholesterol

The FDA has approved Regeneron’s Praluent (alirocumab) to treat children with a genetic form of high cholesterol. This approval extends Praluent’s indication to treat children age 8 years and older with heterozygous familial hypercholesterolemia (HeFH).

Familial hypercholesterolemia (FH) is an inherited condition caused by mutations in one of several genes that control how the body processes cholesterol. Familial hypercholesterolemia can come in two forms: HeFH, which develops when one mutated gene is inherited from one parent; and homozygous familial hypercholesterolemia (HoFH), which develops when a mutated gene is inherited from both parents. Praluent is approved to treat both children and adults with HeFH and adults with HoFH.

Developed by Regeneron and Sanofi, Praluent inhibits the binding of PCSK9 to the LDL receptor and thereby increases the number of available low-density lipoprotein (LDL) receptors on the surface of liver cells to clear LDL and lower LDL cholesterol levels in the blood.

The wholesale acquisition cost of Praluent is $506.48 for two single-dose pens. Commercially insured patients may pay as little as a $50 copay each month up to $3,500 annually. Uninsured patients or insured patients with no pharmacy coverage may be eligible to receive Praluent at no cost through a Patient Assistance Program.

“Many children with heterozygous familial hypercholesterolemia (HeFH) are able to substantially improve their LDL-C (bad cholesterol) with currently available therapies. But for those children whose LDL-C remains dangerously high, this approval is an important milestone as it gives these children and their families an additional option to help reduce and manage their LDL-C levels much earlier in their lives,” Mary P. McGowan, M.D., chief medical officer of the Family Heart Foundation, said in a press release.

The approval is based on a phase 3 trial that enrolled patients aged 8 to 17 with HeFH, who had LDL-C levels of 130mg/dL or greater and were already being treated with lipid-lowering medications. In the trial, 101 patients were treated with Praluent and 52 patients were given placebo every two or four weeks in. Patients who received Praluent every four weeks had 31% lower LDL-C than placebo at 24 weeks. Improvements in additional key lipid parameters were also observed.

No new adverse reactions were identified in this trial. Across Praluent trials in patients with primary hyperlipidemia, the most common adverse reactions have been injection site reactions, and influenza and diarrhea.

Results from the trial were recently published in the Journal of the American Medical Association Pediatrics.