FDA Approves Evolocumab for Pediatric Familial Hypercholesterolemia Indications


The approval is based on 24- and 80-week findings showing significant benefit in LDL-C reduction with the subcutaneous injection drug.


The US Food and Drug Administration (FDA) has approved evolocumab (Repatha) injection as an add-on therapy with diet or other treatment for patients aged ≥10 years with heterozygous familial hypercholesterolemia (HeFH) and homozygous FH (HoFH).

The new approval expands the indicated use of evolocumab, which was previously approved as an add-on therapy for reducing LDL-C in adults with HeFH and patients with HoFH.

The approval granted to Amgen was based on findings from a pair of pediatric clinical trials. The first, involving patients aged 10-17 years with HeFH, assessed 420 mg monthly subcutaneous injection for 24 weeks; patients reported a mean 38% reduction in LDL-C versus placebo.

In the second trial, patients aged 11-17 years old with HoFH received the same evolocumab dose regimen for 80 weeks, and reported a mean 14% reduction in LDL-C compared to baseline.

Evolocumab previously received orphan drug designation from the FDA in 2013 to treat HoFH. It is not indicated for use in patients with serious hypersensitivity, either by clinical diagnosis or emerging signs or symptoms.

Approximately 1 in 250,000 individuals are diagnosed with HoFH, while HeFH is prevalent in 1 in every 250 individuals. The conditions are rare and life-threatening, characterized by gene mutations that influence the body’s clearance of cholesterol including LDL-C.

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