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IndexedBuilt automatically from public databases on 2026-09-30. Not yet hand-reviewed by PLOBT.How this works →

Zilucoplan

FDA approved · Rx

Complement C5 inhibitor peptide

Also known as: Zilbrysq, zilucoplanum, RA 101495, C5 Inhibitor RA101495, Complement C5 Inhibitor RA101495, WHO 10602

Evidence score
89/ 100
Strong evidence

Strong, human-grade evidence

  • Regulatory standing. FDA-approved prescription medicine
  • Human trials. Backed by controlled human studies
  • Registered human trials. 19 registered studies on ClinicalTrials.gov, most advanced Phase 3
  • 2 cited sources. Includes primary / regulatory sources

Computed with the same four public criteria as every profile. The human-evidence grade on an indexed profile is set automatically and may change when it is hand-reviewed. It is not a safety score, not a purity rating, and not medical advice, and not a claim that this compound treats, cures, or prevents any condition.

What it is

Zilucoplan, sold as Zilbrysq, is a synthetic macrocyclic peptide that binds complement component C5. By blocking C5 cleavage, it is thought to prevent formation of the membrane attack complex, which damages the neuromuscular junction in antibody-driven myasthenia gravis.

What the evidence shows

Automatic summaryHuman trials

Zilucoplan is FDA approved for generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive, supported by Phase 3 trial data among 19 registered human trials. Evidence for uses outside this approved indication is limited.

Key facts

Molecular formula
C172H278N24O55
Molecular weight
3562 g/mol
FDA approval
Confirmed on openFDA

Registered human trials

Live from ClinicalTrials.gov, refreshed daily.

19registered studies
Phase 3most advanced phase
11completed (sample)
4recruiting (sample)

Safety and Efficacy Study of RA101495 in Subjects With Generalized Myasthenia Gravis

Phase 2CompletedNCT03315130

A Study to Assess Zilucoplan Concentration in Breast Milk of Healthy Lactating Women

Phase 1RecruitingNCT06961747

An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis

Phase 3Enrolling By InvitationNCT06435312

Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis

Phase 3CompletedNCT04115293

Published literature

Live from Europe PMC. This is how much has been published, not how much is proven in humans.

89papers referencing Zilucoplan

Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study.

Lancet Neurol2023

Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis: Results of a Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Trial.

JAMA Neurol2020

Pharmacotherapy of Generalized Myasthenia Gravis with Special Emphasis on Newer Biologicals.

Drugs2022

ACCORD: A Multicentre, Seamless, Phase 2 Adaptive Randomisation Platform Study to Assess the Efficacy and Safety of Multiple Candidate Agents for the Treatment of COVID-19 in Hospitalised Patients: A structured summary of a study protocol for a randomised controlled trial.

Trials2020

Sources

Risks, side effects and study doses are only published on hand-reviewed profiles. Want this one reviewed, or spotted something wrong? Tell us.