Evinacumab safe, shows durable long-term LDL lowering in children with HoFH
Click Here to Manage Email Alerts
Key takeaways:
- Evinacumab durably lowered LDL out to 72 weeks in young patients with genetic high cholesterol.
- The treatment was well tolerated with no serious drug-related adverse events.
Among pediatric patients with homozygous familial hypercholesterolemia and elevated LDL despite optimal lipid-lowering therapy, evinacumab durably reduced LDL and other significant lipid parameters out to 72 weeks, a speaker reported.
The long-term results of the 17100 study, an open-label, single-arm trial, were presented at the National Lipid Association Scientific Sessions.
“homozygous FH (HoFH) is a rare genetic disorder caused primarily by loss of function of the LDL receptor gene. HoFH is characterized by severely elevated LDL cholesterol starting in utero and increased risk of early onset of atherosclerotic CVD,” Patrick M. Moriarty, MD, FACC, FACP, professor of medicine and director of clinical pharmacology and the Atherosclerosis/Lipoprotein-Apheresis Center at the University of Kansas Medical Center, said during a presentation. “Despite the value of multiple standard care lipid-lowering therapies, the majority of pediatric patients never reach their guideline-recommended LDL, and their risk of cardiovascular disease is obviously very elevated. ... Here, we report the for the first time the long-term, up to 72 weeks, efficacy and safety of evinacumab.”
The present study was a pooled analysis of two parts of the single-arm 17100 trial of evinacumab-dgnb (Evkeeza, Regeneron) in pediatric patients with HoFH with LDL more than 130 mg/dL despite optimized lipid-lowering therapy.
Twenty patients aged 5 to 11 years were enrolled to receive evinacumab 15 mg/kg every 4 weeks for 48 or 72 weeks with an optional additional 24 weeks of follow-up (mean age, 9 years; 60% girls; 70% white).
At baseline, mean lipid parameters were LDL of 301.9 mg/dL; apolipoprotein B of 185.4 mg/dL; non-HDL of 320.9 mg/dL; total cholesterol of 355.8 mg/dL; and lipoprotein(a) of 68 nmol/L.
At 48 weeks, evinacumab reduced LDL by a mean of 44.6%, which was sustained out to 72 weeks (mean, 40.7%).
Durable reductions in other lipid parameters were also observed at 72 weeks:
- 34% lower ApoB;
- 41.6% lower non-HDL;
- 42.2% lower total cholesterol; and
- 15.7% lower Lp(a).
Moriarty reported two serious treatment-emergent adverse events, with one instance of tonsilitis and another of severe aortic valve stenosis. Neither events were related to the study drug, according to the presentation.
Overall, 100% of participants experienced adverse events, but none resulted in treatment discontinuation.
“In pediatric patients with HoFH aged 5 to 11 years of age and inadequate control of LDL despite optimized lipid-lowering therapy, evinacumab demonstrated a substantial and durable reduction of LDL-C,” Moriarty said during the presentation. “Favorable and durable reductions of ApoB, non-HDL, total cholesterol and Lp(a) were also observed.”
Collapse
Moriarty PM, et al. Oral poster presentations. Presented at: National Lipid Association Scientific Sessions; May 30-June 2, 2024; Las Vegas (hybrid meeting).
Click Here to Manage Email Alerts