Drugs / Evinacumab
Regulatory milestones approvals, filings & regulatory actions · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed homozygous familial hypercholesterolemia 4 milestones | |||||
| Filed | US (FDA) | Evkeeza | homozygous familial hypercholesterolemia | 2022-11-30 | “today announced the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics License Application (sBLA) for Evkeeza® (evinacumab-dgnb)” regeneron.com ↗ |
| Filed | EU (EMA) | — | homozygous familial hypercholesterolemia | 2020-11-05 | “An MAA for HoFH has also been submitted in the EU.” regeneron.com ↗ |
| Filed | EU (EMA) | — | homozygous familial hypercholesterolemia | 2020-08-05 | “The Company completed the rolling BLA submission and a Marketing Authorization Application (MAA) was also submitted for HoFH.” regeneron.com ↗ |
| Filed | US (FDA) | — | homozygous familial hypercholesterolemia | 2020-05-05 | “The Company has also initiated a rolling BLA submission for HoFH and plans to submit an MAA in the EU in the second half of 2020.” regeneron.com ↗ “The Company completed the rolling BLA submission and a Marketing Authorization Application (MAA) was also submitted for HoFH.” regeneron.com ↗ “The FDA accepted for priority review the BLA for HoFH, with a target action date of February 11, 2021.” regeneron.com ↗ |
| Approved Indication not stated 1 milestone | |||||
| Approved | EU (EMA) | Evkeeza | — | 2021-06-17 | europa.eu ↗ |
Trials 13
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 13 trials | ||||||
| Phase 1 | NCT03146416 | May 2017 → Jun 2018 | — | Regeneron Pharmaceuticals | Completed | No outcome recorded |
| Phase 1 | NCT02107872 | Apr 2014 → Sep 2015 | metabolic disease | Regeneron Pharmaceuticals | Completed | No outcome recorded |
| Phase 1 | NCT01749878 | Dec 2012 → Jan 2016 | hypertriglyceridemia | Regeneron Pharmaceuticals | Completed | No outcome recorded |
| Phase 25 trials · 1 met primary · 1 missed | ||||||
| Phase 2 | NCT07271186 | Jan 2026 → Sep 2027 expected | diabetic kidney disease | Regeneron Pharmaceuticals | Recruiting | No outcome recorded |
| Phase 2 | NCT04863014 | Jul 2021 → Feb 2023 | hypertriglyceridemia | Regeneron Pharmaceuticals | Terminated | No outcome recorded Stop: Business |
| Phase 2 | NCT03452228 | Jun 2018 → Dec 2019 | hypertriglyceridemia | Regeneron Pharmaceuticals | Completed | Missed primary |
| Phase 2 | NCT03175367 | Nov 2017 → May 2020 | hyperlipidemia | Regeneron Pharmaceuticals | Completed | Met primary |
| Phase 2 | NCT02265952 | Feb 2015 → Nov 2016 | homozygous familial hypercholesterolemia | Regeneron Pharmaceuticals | Completed | No outcome recorded |
| Phase 34 trials · 2 met primary | ||||||
| Phase 3 | NCT05611528 | Feb 2023 → Dec 2024 | homozygous familial hypercholesterolemia | Daniel Gaudet | Completed | No outcome recorded |
| Phase 3 | NCT04233918 | Jun 2020 → Jan 2022 | homozygous familial hypercholesterolemia | Regeneron Pharmaceuticals | Completed | Met primary |
| Phase 3 | NCT03409744 | Mar 2018 → Apr 2023 | homozygous familial hypercholesterolemia | Regeneron Pharmaceuticals | Completed | No outcome recorded |
| Phase 3 | NCT03399786 | Jan 2018 → Jun 2019 | homozygous familial hypercholesterolemia | Regeneron Pharmaceuticals | Completed | Met primary |
| Phase not stated1 trial | ||||||
| — | NCT06500598 | — | homozygous familial hypercholesterolemia, hyperlipoproteinemia type 3, hypertriglyceridemia | Regeneron Pharmaceuticals | Approved for marketing | No outcome recorded |
News releases announcing trial results or a regulatory action · 11
| Date | Issuer | Release |
|---|---|---|
| 2025-09-26 | Regeneron Pharmaceuticals | Regulatory Evkeeza® (evinacumab-dgnb) ANGPTL3 Antibody Approved in the U.S. for Children as Young as 1 Year Old with Ultra-Rare Form of High Cholesterol regeneron.com ↗ |
| 2024-01-04 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Receives Positive Recommendation from NICE in the U.K. for Evkeeza® ▼ (evinacumab) for Adults and Adolescents Aged 12 Years and Older with Homozygous Familial Hypercholesterolemia (HoFH) ultragenyx.com ↗
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending Evkeeza® (evinacumab) to NHS England. |
| 2023-12-18 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Receives European Commission Decision for Evkeeza® (evinacumab) Expanded Indication in Children Aged 5 Years and Older with Homozygous Familial Hypercholesterolemia (HoFH) ultragenyx.com ↗
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the European Commission (EC) has extended the approval of Evkeeza® (evinacumab) as an adjunct to diet and other lipid-lowering therapies to treat children aged 5 to 11 years with homozygous familial hypercholesterolemia (HoFH). |
| 2023-03-22 | Regeneron Pharmaceuticals | Regulatory FDA Approves First-in-class Evkeeza® (evinacumab-dgnb) for Young Children with Ultra-rare Form of High Cholesterol regeneron.com ↗
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced the U.S. Food and Drug Administration (FDA) has extended the approval of Evkeeza® (evinacumab-dgnb) as an adjunct to other lipid-lowering therapies to treat children aged 5 to 11 with homozygous familial hypercholesterolemia (HoFH). |
| 2022-11-30 | Regeneron Pharmaceuticals | Regulatory Evkeeza® (evinacumab-dgnb) sBLA for Children with Ultra-rare Inherited Form of High Cholesterol Accepted for FDA Priority Review regeneron.com ↗ |
| 2022-05-21 | Regeneron Pharmaceuticals | Results Evkeeza® (evinacumab) Phase 3 Trial Demonstrates 48% LDL-C Reduction in Children with Ultra-rare Form of High Cholesterol regeneron.com ↗
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced positive results from a Phase 3 trial evaluating Evkeeza® (evinacumab) in children aged 5 to 11 with homozygous familial hypercholesterolemia (HoFH). |
| 2021-02-11 | Regeneron Pharmaceuticals | Regulatory FDA Approves First-in-class Evkeeza™ (evinacumab-dgnb) for Patients with Ultra-rare Inherited Form of High Cholesterol regeneron.com ↗
Evkeeza is the first FDA-approved treatment that binds to and blocks the function of angiopoietin-like 3 (ANGPTL3), a protein that plays a key role in lipid metabolism. |
| 2020-08-19 | Regeneron Pharmaceuticals | Results New England Journal of Medicine Publishes Positive Phase 3 Evinacumab Results in Patients with Severe Inherited Form of High Cholesterol regeneron.com ↗
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the New England Journal of Medicine (NEJM) published positive results from the Phase 3 trial of evinacumab in 65 patients with homozygous familial hypercholesterolemia (HoFH). |
| 2020-08-12 | Regeneron Pharmaceuticals | Regulatory FDA Accepts Evinacumab Biologics License Application for Priority Review as a Treatment for Patients with HoFH, an Ultra-rare Inherited Form of High Cholesterol regeneron.com ↗
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review a Biologics License Application (BLA) for evinacumab as an adjunct to other lipid-lowering therapies in patients with homozygous familial hypercholesterolemia (HoFH). |
| 2020-03-30 | Regeneron Pharmaceuticals | Results Regeneron Announces American College of Cardiology Presentation of Positive Phase 3 Evinacumab Results in Patients with Severe Inherited Form of High Cholesterol regeneron.com ↗
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) announced that detailed Phase 3 results of evinacumab were presented today as a late-breaking presentation at the American College of Cardiology's Annual Scientific Session together with World Congress of Cardiology (ACC.20). |
| 2019-08-14 | Regeneron Pharmaceuticals | Results Regeneron Announces Positive Topline Results from Phase 3 Trial of Evinacumab in Patients with Severe, Inherited Form of High Cholesterol regeneron.com ↗
The trial met its primary endpoint, showing that adding evinacumab to other lipid-lowering therapies decreased LDL cholesterol by 49% on average, compared to lipid-lowering therapies alone. |
All press releases naming this drug 13 releases
Evidence & citations 8 cited values
Every value below carries the sentence it was read from. 16 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | evinacumab | “Evinacumab, an ANGPTL3 inhibitor, reduced triglycerides in healthy human volunteers and in homozygous familial hypercholesterolemic individuals.” PMID 31242752 ↗ Jun 2019 |
| Known as | Evinacumab dgnb | ChEMBL registry synonym — accepted as the source's own label CHEMBL3545191 ↗ |
| Known as | Evkeeza | ChEMBL registry synonym — accepted as the source's own label CHEMBL3545191 ↗ |
| Known as | REGN1500 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT01749878 ↗ |
| Action | Inhibit | “Evinacumab, an ANGPTL3 inhibitor, reduced triglycerides in healthy human volunteers and in homozygous familial hypercholesterolemic individuals.” PMID 31242752 ↗ Jun 2019 |
| Modality | Monoclonal antibody | “We also tested the effects of a human monoclonal antibody, evinacumab, against Angptl3 in dyslipidemic mice and against ANGPTL3 in healthy human volunteers with elevated levels...” PMID 28538136 ↗ May 2017 |
| Route | Subcutaneous | “SAD subjects received evinacumab subcutaneously at 75/150/250 mg” PMID 31242752 ↗ Jun 2019 |
| Target | ANGPTL3 | “a human monoclonal antibody, evinacumab, against Angptl3 in dyslipidemic mice and against ANGPTL3 in healthy human volunteers” PMID 28538136 ↗ May 2017 |