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Peptide

Zilucoplan

Daily self-injected peptide that blocks complement. Used in adult generalized myasthenia gravis with antibodies against the acetylcholine receptor.
Regulatory status
Approved (FDA / EMA)
Family
Peptide complement C5 inhibitor
Scientific evidence
High
Number of findings
1
Where it acts
Immunity and defense · Bones and muscles
Also known as
zilucoplan, Zilbrysq, RA101495

Regulatory status

FDA (US): Approved (2023, Zilbrysq) for generalized myasthenia gravis in adults with anti-acetylcholine receptor antibodies.

EMA (EU): Approved (2023, Zilbrysq) as an add-on to standard treatment in generalized myasthenia gravis in adults with anti-acetylcholine receptor antibodies.

Dose

One subcutaneous injection daily according to weight, equivalent to about 0.3 mg/kg (RAISE trial; FDA/EMA Zilbrysq label).

Safety

Risk of serious meningococcal infections from blocking complement: vaccination beforehand is mandatory (FDA boxed warning and monitoring program). Injection-site reactions (bruising), respiratory infections, diarrhea. Only under specialized neurology care.

Interactions

Do not combine with other complement inhibitors. Intravenous immunoglobulins and plasma exchange may interfere with its effect.

Effect by goal · level of evidence

Symptom improvement in myasthenia gravis

improves High RCT (AA) in people
−2.1 MG-ADL points vs placebo

RAISE trial with 174 patients over 12 weeks: the MG-ADL daily activities scale improved 2.09 points more than with placebo. Serious adverse effects and serious infections were similar between groups.

View the study (RCT) →

In depth

Where it acts

Immunity and defense, Bones and muscles

Autoimmunity

How it works

In myasthenia, antibodies activate complement, which damages the junction between nerve and muscle. Zilucoplan binds to the C5 protein and prevents it from forming the complex that attacks the membrane. This protects the motor endplate and improves strength.

What the evidence says

In people

  • Symptom improvement in myasthenia gravis. RAISE trial with 174 patients over 12 weeks: the MG-ADL daily activities scale improved 2.09 points more than with placebo. Serious adverse effects and serious infections were similar between groups. PubMed 37059508

References

  1. PubMed 37059508

Information, not medical advice. Prescription or unapproved peptides should only be used under medical supervision.

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