New Medicines

EvkeezaHomozygous familial hypercholesterolaemia, in adults and children aged ≥ 12 years


New molecular entity

Development and Regulatory status

Approved (Licensed)
Approved (Licensed)
Jan 22Regeneron and Ultragenyx announce a collaboration agreement for Ultragenyx to clinically develop, commercialise and distribute Evkeeza in countries outside of the US [15].
Jun 21Approved in EU [14]
Apr 21Recommended for EU approval by CHMP under exceptional circumstances - the full indication is "as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adult and adolescent patients aged 12 years and older with homozygous familial hypercholesterolaemia (HoFH).” Evkeeza will be available as concentrate for solution for infusion (150 mg/ml) [13]. Under exceptional circumstances indicates that an authorisation may be granted subject to certain specific obligations, to be reviewed annually. This happens when the information available is less than would usually be required, due to the rarity of the condition it is intended for, limited scientific knowledge in the area concerned, or ethical considerations involved in the collection of such data.
Feb 21Evinacumab is administered based on weight (15 mg/kg) once a month via intravenous infusion. The average Wholesale Acquisition Cost (WAC) per patient in the U.S. will vary based on weight, and is expected to be approximately $450,000 per year on average [12].
Feb 21FDA has approved evinacumab as an adjunct to other low-density lipoprotein cholesterol (LDL-C) lowering therapies to treat adult and children 12 years and older with homozygous familial hypercholesterolemia [11].
Sep 20Filed in EU via centralised procedure and accepted for accelerated assessment [10].
Aug 20Evicanumab has been given priority review as an adjunct to other lipid-lowering therapies in pts with homozygous familial hypercholesterolemia (HoFH) by the FDA with a PDUFA date of February 2021. [8]
Aug 20Regeneron plans to submit PIII data to regulatory authorities, starting with the FDA in 2020 [7].
Sep 18Has orphan drug status in US [5].
Sep 18Has breakthrough therapy status in US [4].


Fully human angiopoietin-like 3 (ANGPTL3) blocking antibody. Angiopoietin-like 3 (ANGPTL3) is a secreted protein expressed in the liver. It acts to increase plasma levels of triglycerides, LDL cholesterol, and high-density lipoprotein (HDL) cholesterol.
Heterozygous familial hypercholesterolaemia is one of the most common familial conditions, with a prevalence of about 1 in 500. Homozygous familial hypercholesterolaemia (HoFH) is rare [1]. Prevalence of HoFH is 1 per 1,000,000 people [2].
Homozygous familial hypercholesterolaemia, in adults and children aged ≥ 12 years
Intravenous infusion

Further information


Trial or other data

May 22Company announces positive results for evinacumab in children aged 5 to 11 with homozygous familial hypercholesterolemia (NCT04233918, n=14). The trial met its primary endpoint, showing children who added evinacumab to other lipid-lowering therapies reduced their low-density lipoprotein-cholesterol (LDL-C) by 48% at week 24 on average. Reductions in levels of all lipid endpoint parameters assessed, which were generally observed within the first 8 weeks of treatment. Evinacumab was generally well tolerated. Most common adverse events were throat pain, upper abdominal pain, diarrhea, headache and nasopharyngitis. Two severe adverse events occurred, both of which were considered unrelated to treatment [17]
Aug 20ELIPSE HoFH (NCT03399786; n=65) reported IV infusion of evinacumab (15 mg/kg) every 4 weeks lowered LDL cholesterol from baseline significantly compared to placebo at week 24 (relative reduction 47.1% vs. increase of 1.9%; between-group least-squares mean absolute difference, –3.4 mmol/L, p<0.001) [9].
Aug 19 PIII trial evaluating evinacumab in patients (aged 12 to 75) with HoFH has released top line results, demonstrating a 49% reduction from baseline of LDL-C with evinacumab compared to placebo. 47% of patients treated with evinacumab achieved target LDL-C of <100mg/dl, compared to 23% of placebo patients [6].
Feb 18PIII trial to evaluate long-term safety and tolerability of evinacumab in patients with HoFH starts (NCT03409744). This open-label trial is enrolling approximately 66 patients in US, Canada & Netherlands, and is expected to complete Apr 22 [3].
Jan 18PIII trial to evaluate efficacy and safety of evinacumab in patients with HoFH starts (NCT03399786). The trial is enrolling approximately 57 patients in sites including the US & EU (but not UK). Collection of primary outcome data (percent change in calculated LDL-C from baseline to week 24) is expected to complete Apr 20 [3].

Evidence based evaluations