Kynamro in Pediatric HoFH Patients

Kynamro (mipomersen) is a second-generation antisense therapy for treatment of patients with homozygous familial hypercholesterolemia (HoFH).

In the pivotal trial that led to its approval in 2013,  Raal et al (2010) looked 51 HoFH patients age 12 to 53 yrs, who were given Kynamro (n=37) or placebo (n=14).  

At the National Lipid Association (NLA) Scientific Session, data from the 7 children (12 -16 years) in the pivotal 51-patient trial were presented by Raal and Selvey. 

In this exclusive interview with Rare Disease Report, Sheryl Selvey, PharmD, associate director of Scientific Communications at Genzyme, discusses the data obtained from those children.

In the trial, all 7 patients eventually received Kynamro in the second open-label phase of the trial, but the trial began with 3 patients blinded to receive Kynamro and 4 patients blinded to receive placebo for the first 28 weeks.

As Dr. Selvey explains in this video, the drug was found to be safe and effective in these pediatric patients.


Raal FJ, Santos RD, Blom DJ, et al. Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial. Lancet. 2010;375(9719):998-1006.

Raal SJ, Selvey S. Efficacy and safety of mipomersen in pediatric patients with homozygous familial hypercholesterolemia. Poster presented at National Lipid Association Scientific Sessions; May 1-4, 2014; Orlando, FL. Poster #42.
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