Praluent Approved for Pediatric Patients With Heterozygous Familial Hypercholesterolemia – Renal and Urology News

The Food and Drug Administration (FDA) has approved Praluent^® (alirocumab) as an adjunct to diet and other low density lipoprotein cholesterol (LDL-C)-lowering therapies in pediatric patients aged 8 years and older with heterozygous familial hypercholesterolemia (HeFH) to reduce LDL-C.

The use of Praluent, a proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitor, in pediatric patients with HeFH was based on data from a randomized, placebo-controlled, double-blind phase 3 trial (ClinicalTrials.gov Identifier: NCT03510884). Study participants (N=153) were between the ages of 8 and 17 years (mean age, 13 years) and were on a low fat diet and receiving background lipid-lowering medications (eg, statins, ezetimibe). Mean body weight was 53kg and mean LDL-C at baseline was 174mg/dL.

Patients were randomly assigned to receive Praluent dosed every 2 weeks or every 4 weeks or placebo. In the every 2 weeks group (n=49), patients received Praluent at a dose of 40mg for body weight less than 50kg (not an approved dosage) or 75mg for body weight of 50kg or more. In the every 4 weeks group (n=52), patients received Praluent at a dose of 150mg for body weight less than 50kg or 300mg for body weight of 50kg or more. 

Adjustments in dose to 75mg every 2 weeks (for body weight <50kg) or 150mg every 2 weeks (for body weight ≥50kg) occurred at week 12 in patients with LDL-C of greater than or equal to 110mg/dL.

Findings showed a treatment difference of -31.4% (97.5% CI, -45, -17.9; P <.0001) in least squares (LS) mean LDL-C percent change from baseline between the Praluent group receiving treatment every 4 weeks and the placebo groups at 24 weeks (primary endpoint). For apolipoprotein B, non-high density lipoprotein-C, and total cholesterol, the LS mean differences from placebo were -29.7%, -30.1%, and -22.3%, respectively.

The safety of Praluent in pediatric patients was consistent with previous trials in adults with HeFH; no new safety signals were observed in the trial. 

“The approval of Praluent for the treatment of high cholesterol was a historic landmark achievement, as it was the first approved therapy targeting the genetically-validated PCSK9 target for heart disease,” said George D. Yancopoulos, MD, PhD, Board co-Chair, President and Chief Scientific Officer at Regeneron, and a principal inventor of Praluent. “Praluent has made a meaningful impact in the treatment of adults with familial hypercholesterolemia, and we are proud that our innovation will now be able to help appropriate children with the heterozygous form of this disease manage their dangerously high levels of LDL-C.”

The dosage of Praluent for pediatric patients aged 8 years and older with HeFH is based on body weight and can be adjusted based on LDL-C lowering response. The LDL-lowering effect may be measured as early as 4 weeks after initiation.

Praluent is supplied in 2 dosage strengths, 75mg/mL and 150mg/mL, in a single-dose prefilled pen. In children aged 8 to 11 years, Praluent should be given by a caregiver following proper training. In children 12 to 17 years, it is recommended that Praluent be given by or under the supervision of an adult.

This article originally appeared on MPR