Evinacumab

Overview

Evinacumab is a recombinant human IgG4 monoclonal antibody targeted against angiopoietin-like protein 3 (ANGPTL3) and the first drug of its kind. The ANGPTL family of proteins serve a number of physiologic functions - including involvement in the regulation of lipid metabolism - which have made them desirable therapeutic targets in recent years. Loss-of-function mutations in ANGPTL3 have been noted to result in hypolipidemia and subsequent reductions in cardiovascular risk, whereas increases in function appear to be associated with cardiovascular risk, and it was these observations that provided a rationale for the development of a therapy targeted against ANGPTL3. In February 2021, evinacumab became the first-and-only inhibitor of ANGPTL3 to receive FDA approval after it was granted approval for the adjunctive treatment of homozygous familial hypercholesterolemia (HoFH) under the brand name "Evkeeza". Evinacumab is novel in its mechanism of action compared with other lipid-lowering therapies and therefore provides a unique and synergistic therapeutic option in the treatment of HoFH.

Indication

Evinacumab is indicated, as an adjunct with other lipid-lowering therapies, for the treatment of familial homozygous hypercholesterolemia (HoFH) in patients 5 years of age and older.

Associated Conditions

Homozygous Familial Hypercholesterolaemia (HoFH)

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
Compassionate Use of Evinacumab	2024/07/15	NCT06500598	N/A	AVAILABLE	Regeneron Pharmaceuticals
Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia	2022/11/10	NCT05611528	Phase 3	Completed	Daniel Gaudet
Efficacy and Safety of Evinacumab in Adult Patients With Severe Hypertriglyceridemia for the Prevention of Recurrent Acute Pancreatitis	2021/04/28	NCT04863014	Phase 2	Terminated	Regeneron Pharmaceuticals
Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia	2020/01/18	NCT04233918	Phase 3	Completed	Regeneron Pharmaceuticals
Safety and Efficacy Following Repeat-Dose of Evinacumab (Anti-ANGPTL3) in Patients With Severe Hypertriglyceridemia (sHTG) at Risk for Acute Pancreatitis	2018/03/02	NCT03452228	Phase 2	Completed	Regeneron Pharmaceuticals
Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia	2018/01/24	NCT03409744	Phase 3	Completed	Regeneron Pharmaceuticals
Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia	2018/01/16	NCT03399786	Phase 3	Completed	Regeneron Pharmaceuticals
Study of Evinacumab (REGN1500) in Participants With Persistent Hypercholesterolemia	2017/06/05	NCT03175367	Phase 2	Completed	Regeneron Pharmaceuticals
Study of Evinacumab (REGN1500) in Caucasian and in Japanese Healthy Volunteers	2017/05/10	NCT03146416	Phase 1	Completed	Regeneron Pharmaceuticals
Study to Assess the Safety and Tolerability of Multiple Ascending Doses of REGN1500	2014/04/08	NCT02107872	Phase 1	Completed	Regeneron Pharmaceuticals

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
Evkeeza	Regeneron Pharmaceuticals, Inc.	61755-010	INTRAVENOUS	150 mg in 1 mL	3/30/2023
Evkeeza	Regeneron Pharmaceuticals, Inc.	61755-013	INTRAVENOUS	150 mg in 1 mL	3/30/2023

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
Evkeeza	Ultragenyx Germany GmbH,Rahel-Hirsch-Str. 10,10557 Berlin,Germany	Authorised	6/17/2021
Evkeeza	Ultragenyx Germany GmbH,Rahel-Hirsch-Str. 10,10557 Berlin,Germany	Authorised	6/17/2021

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
No TGA approvals found for this drug.

Health Canada Drug Approvals

Approved Product	Company	DIN	Dosage Form	Strength	Market Date
EVKEEZA	ULTRAGENYX PHARMACEUTICAL INC	02541769	Solution - Intravenous	150 MG / ML	11/21/2023

CIMA AEMPS Drug Approvals

Approved Product	Company	Registration Number	Pharmaceutical Form	Prescription Type	Status
EVKEEZA 150 MG/ML CONCENTRADO PARA SOLUCION PARA PERFUSION	Ultragenyx Germany Gmbh	1211551001	CONCENTRADO PARA SOLUCIÓN PARA PERFUSIÓN	Uso Hospitalario	Commercialized

Philippines FDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Philippines FDA approvals found for this drug.

Saudi SFDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Saudi SFDA approvals found for this drug.

Malaysia NPRA Drug Approvals

Approved Product	Company	Registration Number	Dosage Form	Strength	Approval Date
No Malaysia NPRA approvals found for this drug.

UK EMC Drug Information

Medicine Name	MA Holder	MA Number	Pharmaceutical Form	Active Ingredient	Authorization Date
No UK EMC drug information found for this drug.

Related News

Health Canada Extends Evkeeza Approval to Infants as Young as 6 Months for Rare Cholesterol Disorder

2 months ago

Health Canada has extended approval of Evkeeza (evinacumab) to treat children as young as 6 months old with homozygous familial hypercholesterolemia (HoFH), a devastating inherited cholesterol disorder.

CRISPR Therapeutics' Casgevy Shows Commercial Progress as Gene Therapy Pipeline Advances

4 months ago

CRISPR Therapeutics and Vertex report encouraging progress with Casgevy, their approved gene therapy for sickle cell disease and beta-thalassemia, with 65 treatment centers now activated globally.

Evkeeza Approved in EU for HoFH Treatment in Children as Young as 6 Months

8 months ago

• The European Commission has approved Evkeeza (evinacumab) for treating children aged six months and older with homozygous familial hypercholesterolemia (HoFH). • Evkeeza is the first medicine approved in the EU for HoFH treatment in children as young as six months, addressing a critical unmet need. • Efficacy in young patients was predicted through model-based extrapolation, showing similar or greater LDL-C reduction compared to adults. • Evkeeza is now reimbursed and commercially available in multiple countries, with early access schemes in additional regions.

Ultragenyx Reports Positive Q3 2024 Results and Clinical Updates

10 months ago

• Ultragenyx's Q3 2024 total revenue increased by 42% year-over-year to $139 million, driven by Crysvita and Dojolvi sales growth, reaffirming its 2024 revenue guidance of $530-$550 million. • The FDA granted Breakthrough Therapy Designation for setrusumab (UX143) in osteogenesis imperfecta, based on promising Phase 2 clinical trial results showing a significant reduction in fracture rate. • Phase 3 follow-up data for DTX401 in glycogen storage disease type Ia (GSDIa) demonstrated a substantial 62% mean reduction in cornstarch intake among crossover patients. • Ultragenyx anticipates filing a J-NDA for Dojolvi in Japan by mid-2025 and a BLA submission for DTX401 in mid-2025, marking significant progress in expanding treatment options for rare diseases.

AHA Advocates for Expanded Lipoprotein Apheresis Use in High-Risk Cardiovascular Patients

a year ago

The American Heart Association's new scientific statement highlights lipoprotein apheresis as an underutilized treatment that can reduce LDL-C and Lp(a) levels by 65-85% in a single session for familial hypercholesterolemia patients.

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