Zilucoplan

DB15636ApprovedInvestigationalSmall moleculeComplement Inactivating Agents

Zilucoplan is a complement inhibitor used to treat generalized myasthenia gravis in anti-acetylcholine receptor (AChR) antibody-positive adults. Zilucoplan is a 15 amino-acid, synthetic macrocyclic peptide.

Mechanism: The complement system is part of the innate immune system and is critical in inflammatory reactions in response to pathogenic bacteria. Activation pathways of the complement system involve the cleavage of the complement protein C5 by C5 convertases to form C5a, a potent anaphylatoxin, and C5b. Cleavage of C5 also recruits C6, C7, C8, and C9. C5b binds to C6 to yield the terminal complement complex C5b9, a hydrophilic pore that spans the cell membrane. C5b9 causes an influx of water and ions, resulting in osmotic lysis of the targeted cell. The terminal complement cascade has been implicated in the pathophysiology of various inflammatory and autoimmune disorders, including gMG. gMG is an autoimmune disorder characterized by pathogenic autoantibodies that bind to AChRs. Accumulated MAC on the postsynaptic plasma membrane of the neuromuscular junction leads to muscle weakness and damage.
The exact mechanism of zilucoplan in gMG has not been fully elucidated. Zilucoplan binds to the complement protein C5 with high affinity to inhibit its cleavage to C5a and C5b, preventing the generation of C5b9.
Curator reviewed · 8 references
Primary indication: Zilucoplan is indicated as the main treatment or add-on treatment to standard therapy for generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody-positive by the FDA and EMA respectively.Curator reviewed · 2 structured indications
Formula / weight: C₁₇₂H₂₇₈N₂₄O₅₅ · 3562.229 g/mol (avg)
First approval: Canada, 2024 · United States, 2024 · European Union, 2024
Also known as: 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-
Code names: RA101495 · WHO 10602
Brand names: Zilbrysq

Resolves to

Compound

UNIIYG391PK0CCCAS1841136-73-9

Clinical / RWD

RxNorm2672489ATCL04AJ06

Structure

InChIKeyJDXCOXKBIGBZSK-PSNKNOTQSA-NSMILES

CCCCCCCCCCCCCCCC(=O)N[C@@H](CCC(=O)NCCOCCOCCOCCOCCOCCOCCOCCOCCOCCOCCOCCOCCOCCOCCOCCOCCOCCOCCOCCOCCOCCOCCOCCOCCC(=O)NCCCC[C@H](NC(=O)[C@@H](NC(=O)[C@@H]1CCCN1C(=O)[C@H](CC1=CC=C(O)C=C1)NC(=O)[C@H](CCC(O)=O)NC(=O)[C@H](CC1=CNC2=NC=CC=C12)NC(=O)[C@H](CC1=CC=C(O)C=C1)NC(=O)[C@@H](NC(=O)[C@H](CC(O)=O)N(C)C(=O)[C@@H]1CC(=O)NCCCC[C@H](NC(C)=O)C(=O)N[C@@H](C(C)C)C(=O)N[C@@H](CCC(O)=O)C(=O)N[C@@H](CCCNC(N)=N)C(=O)N[C@@H](CC2=CC=CC=C2)C(=O)N1)C(C)(C)C)C1CCCCC1)C(O)=O)C(O)=O

Targets

P01031C5

What you can answer from here — as of September 13, 2026

1Protein targetEach mapped to UniProt, with action and pharmacological action

Listed above

362Drug interactionsStructured to mechanism, not free text

15Clinical trialsPhase, status and sponsor resolved per trial

2Structured indicationsCondition, population, route and combination as fields, not prose

3ContraindicationsEach with its own population and attribute set

15Marketed productsAcross 4 countries and 2 labellers

1ATC codeIncluding every combination product, plus 8 drug categories

14+ReferencesStructured and connected to the statements they support

Which drugs share a target with Zilucoplan, and which of those have an active Phase 3 trial?

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