Alirocumab approval now includes some pediatric patients with lipid disorder
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Key takeaways:
- The FDA extended its approval of alirocumab to treat a genetic lipid disorder in patients as young as 8 years.
- Approval was based on a recent phase 3 trial.
Regeneron Pharmaceuticals announced the FDA extended its approval of alirocumab as an adjunct to diet and other LDL-lowering therapies to include patients aged 8 years and older with heterozygous familial hypercholesterolemia.
The approval was based on the results of the phase 3, randomized multicenter heterozygous familial hypercholesterolemia (HeFH) pediatric trial evaluating alirocumab (Praluent, Sanofi/Regeneron) in patients aged 8 to 17 years with HeFH and LDL of 130 mg/dL or more already treated with LDL-lowering therapies.
Compared with placebo, participants who received alirocumab every 4 weeks had 31% lower LDL levels at 24 weeks (97.5% CI, 45 to 17.9; P < .0001), according to a company press release.
No new adverse reactions were identified in this trial, and the safety profile was consistent with the safety profile observed in adults with HeFH treated with alirocumab, with the most common adverse reactions that occur more often with alirocumab than placebo being injection site reactions, influenza and diarrhea, according to the release.
“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, MD, chief medical officer of the Family Heart Foundation, said in the release.
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