FDA approves add-on therapy for patients with genetic form o
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FDA has approved Evkeeza (evinacumab-dgnb) injection as an add-on treatment for patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH), a genetic condition that causes severely high cholesterol.

HoFH is a rare, life-threatening condition that occurs in approximately 1 in 250,000 individuals. Patients with HoFH have two mutations in a small group of genes that controls the way the body clears cholesterol. As a result, patients have extremely high circulating levels of low-density lipoprotein cholesterol (LDL-C), known as “bad cholesterol.” These cholesterol levels can range as high as 500-1,000 mg/dL, more than four times the normal levels.

Patients with HoFH can develop premature cardiovascular disease, including heart attack and heart disease, when they are teenagers or in their 20s, and many do not improve with other cholesterol-lowering drugs. Without aggressive treatment, patients may die before age 30.

Evkeeza binds to the angiopoietin-like protein 3 (ANGPTL3). ANGPTL3 slows the function of certain enzymes that break down fats in the body. Evkeeza blocks ANGPTL3, allowing faster break down of fats that lead to high cholesterol.

The effectiveness and safety of Evkeeza were evaluated in a double-blind, randomized, placebo-controlled, 24-week trial enrolling 65 patients with HoFH. In the trial, 43 patients received 15 mg/kg of Evkeeza every four weeks and 22 patients received the placebo. Patients were taking other lipid-lowering therapies as well.

The primary measure of effectiveness was the percent change in LDL-C from the beginning of treatment to week 24. At week 24, patients receiving Evkeeza had an average 47% decrease in LDL-C while patients on the placebo had an average 2% increase.

The safety and effectiveness of Evkeeza 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 Evkeeza are nasopharyngitis (cold), influenza-like illness, dizziness, rhinorrhea (runny nose), and nausea. Serious hypersensitivity (allergic) reactions have occurred in the Evkeeza clinical trials. Evkeeza may cause harm to a fetus (unborn baby), so prescribers should advise patients about these risks and consider pregnancy testing for patients who may become pregnant before starting treatment.

Evkeeza 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: https://www.fda.gov/drugs/drug-safety-and-availability/fda-approves-add-therapy-patients-genetic-form-severely-high-cholesterol
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