TitleFDA Approves Add-On Therapy for Patients With Homozygous Familial Hypercholesterolaemia
The US Food and Drug Administration (FDA) has approved evinacumab-dgnb (Evkeeza) injection as an add-on treatment for patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH).
The effectiveness and safety of evinacumab were evaluated in a double-blind, randomised, placebo-controlled, 24-week trial enrolling 65 patients with HoFH. In the trial, 43 patients received evinacumab 15 mg/kg every 4 weeks and 22 patients received placebo. Patients were taking other lipid-lowering therapies as well.
The primary outcome was the percent change in low-density lipoprotein cholesterol (LDL-C) from the beginning of treatment to week 24. At week 24, patients receiving evinacumab had an average 47% decrease in LDL-C while patients on the placebo had an average 2% increase.
The safety and effectiveness of evinacumab have not been established among patients with other causes of high cholesterol, including heterozygous familial hypercholesterolemia. The drug’s effect on cardiovascular morbidity and mortality has not been determined.
Common side effects of evinacumab are nasopharyngitis, influenza-like illness, dizziness, rhinorrhea, and nausea. Serious hypersensitivity reactions have occurred in the evinacumab clinical trials. Evinacumab may cause harm to a fetus, so prescribers should advise patients about these risks and consider pregnancy testing for patients who may become pregnant before starting treatment.
Evinacumab received orphan drug designation, which provides incentives to assist and encourage drug development for rare diseases, breakthrough therapy designation and priority review designation for this indication.
SOURCE: US Food and Drug Administration