Drug

Zilucoplan

Approved · EMA

Late-stage development

Also known as Zilbrysq, N2-ACETYL-L-LYSYL-L-VALYL-L-.ALPHA.-GLUTAMYL-L-ARGINYL-L-PHENYLALANYL-L-.ALPHA.-ASPARTYL-N-METHYL-L-.ALPHA.-ASPARTYL-3-METHYL-L-VALYL-L-TYROSYL-3-(1H-PYRROLO(2,3-B)PYRIDIN-3-YL)-L-ALANYL-L-.ALPHA.-GLUTAMYL-L-TYROSYL-L-PROLYL-(2S)-2-CYCLOHEXYLGLYCYL-N6-(3, POLY(OXY-1,2-ETHANEDIYL), .ALPHA.-(2-(((4S)-4-CARBOXY-1-OXO-4-(1-OXOHEXADECYL)BUTYL)AMINO)ETHYL)-.OMEGA.-HYDROXY-, 15-ETHER WITH N-ACETYL-L-LYSYL-L-VALYL-L-.ALPHA.-GLUTAMYL-L-ARGINYL-L-PHENYLALANYL-L-.ALPHA.-ASPARTYL-N-METHYL-L-.ALPHA.-ASPARTYL-3-METHYL-.

RxNorm2672489UNIIYG391PK0CC

Active clinical trials

Regulatory events

Latest activity

beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Multicenter Open-Label, Uncontrolled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, Tolerability, and Activity of Zilucoplan in Pediatric Study Participants From 2 to Less Than 18 Years of Age With Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis

Clinical trial2026-07-06Results expected Q4 2026 · ClinicalTrials.gov

An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Activity of Zilucoplan in Pediatric Study Participants With Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis

Clinical trial2026-07-06Results expected Q4 2027 · ClinicalTrials.gov

A Phase 3, Multicenter, Open-Label Extension Study of Zilucoplan in Subjects With Generalized Myasthenia Gravis

Clinical trial2026-06-29Primary completion · ClinicalTrials.gov

HEALEY ALS Platform Trial

Clinical trial2026-06-04Results expected Q3 2027 · ClinicalTrials.gov

A Multicenter, Open-Label, Outpatient Study to Evaluate the Safe And Effective Use of a Zilucoplan Auto-Injector Combination Product for Subcutaneous Self-Administration by Study Participants With Generalized Myasthenia Gravis

Clinical trial2026-03-27Results posted · ClinicalTrials.gov

An Open-Label, Repeated-Dose Assessment of Zilucoplan Concentration in Breast Milk of Healthy Lactating Women

Clinical trial2026-03-24Results expected Q1 2027 · ClinicalTrials.gov

An Open-Label, Single Center, Randomized, 2-Way Crossover, Single-Dose, Bioequivalence Study of Zilucoplan Injected Subcutaneously Either by a Prefilled Syringe or an Auto-Injector in Healthy Adult Participants

Clinical trial2026-01-06Results posted · ClinicalTrials.gov

A Phase 3b, Multicenter, Open-Label, Single-Arm Study to Evaluate the Safety, Tolerability, and Efficacy of Zilucoplan in Participants With Generalized Myasthenia Gravis Switching From Intravenous Complement Component 5 Inhibitors to Subcutaneous Zilucoplan

Clinical trial2025-09-03Results posted · ClinicalTrials.gov

A Phase 3, Multicenter, Randomized, Double Blind, Placebo-Controlled Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis

Clinical trial2025-07-16Results posted · ClinicalTrials.gov

Approval: Zilbrysq (EMA)

Regulatory2023-12-01EMA

HEALEY ALS Platform Trial - Regimen A Zilucoplan

Clinical trial2023-07-25Results posted · ClinicalTrials.gov

A Phase 2 Multicenter, Open-Label, Uncontrolled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Have an Inadequate Response to Eculizumab

Clinical trial2022-07-27Results posted · ClinicalTrials.gov

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name

Zilucoplan

Aliases & brands

ZilbrysqN2-ACETYL-L-LYSYL-L-VALYL-L-.ALPHA.-GLUTAMYL-L-ARGINYL-L-PHENYLALANYL-L-.ALPHA.-ASPARTYL-N-METHYL-L-.ALPHA.-ASPARTYL-3-METHYL-L-VALYL-L-TYROSYL-3-(1H-PYRROLO(2,3-B)PYRIDIN-3-YL)-L-ALANYL-L-.ALPHA.-GLUTAMYL-L-TYROSYL-L-PROLYL-(2S)-2-CYCLOHEXYLGLYCYL-N6-(3POLY(OXY-1,2-ETHANEDIYL), .ALPHA.-(2-(((4S)-4-CARBOXY-1-OXO-4-(1-OXOHEXADECYL)BUTYL)AMINO)ETHYL)-.OMEGA.-HYDROXY-, 15-ETHER WITH N-ACETYL-L-LYSYL-L-VALYL-L-.ALPHA.-GLUTAMYL-L-ARGINYL-L-PHENYLALANYL-L-.ALPHA.-ASPARTYL-N-METHYL-L-.ALPHA.-ASPARTYL-3-METHYL-

RxNorm CUI

2672489

UNII

YG391PK0CC

Regulatory jurisdictions

ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval

2023-12-01

Latest approval

2023-12-01

Authorities

EMA

Total events

emaEuropean Medicines Agency· 1 event

2023-12-01Approval

Approval: Zilbrysq (EMA)

Indication: Zilbrysq is indicated as an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR)… Show full indication

Zilbrysq is indicated as an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.