Extended indication

Zilbrysq is indicated as an add-on to standard therapy for the treatment of generalised myasthenia g

Therapeutic value

Possible benefit in ease of use

Registration phase

Positive CHMP opinion


Active substance



Neurological disorders

Reason of inclusion

New medicine (specialité)

Main indication

Muscular diseases other

Extended 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.

Proprietary name




Portfolio holder


Route of administration


Therapeutical formulation


Budgetting framework

Extramural (GVS)

Centre of expertise

LUMC / MUCM+ / AUMC (loc. AMC)

Additional remarks
Mechanism of Action:  zilucoplan is a synthetic macrocyclic peptide inhibitor of the terminal complement protein C5, with potential anti-inflammatory and cell protective activities. Upon subcutaneous administration, complement inhibitor zilucoplan binds to a unique site in terminal complement protein C5, which blocks C5 cleavage into C5a and C5b and prevents the C5b-dependent assembly of the membrane-attack complex (MAC). Zilucoplan also inhibits the interaction between C5b and C6, thereby further blocking MAC assembly.


Registration route

Centralised (EMA)

Type of trajectory

Normal trajectory



Submission date

September 2022

Expected Registration

December 2023

Orphan drug


Registration phase

Positive CHMP opinion

Additional remarks
Positieve CHMP-opinie in september 2023.

Therapeutic value

Current treatment options

Cholinesterase inhibitors, corticosteroids, Intravenous, Immunosuppressants, Immunoglobulins, bloodplasma transfer, Rituximab, Eculizumab

Therapeutic value

Possible benefit in ease of use


De fase 2 studie is afgerond. In vergelijking met placebo laat het geneesmiddel positieve resultaten zien (1). Een fase 3 studie zou verder uitsluitsel moeten geven over de therapeutische waarde van dit geneesmiddel ten opzichte van andere beschikbare geneesmiddelen (2). Op basis van de beschikbare data wordt op dit moment verwacht dat de nieuwe complementremmers ongeveer gelijk zullen presteren aan eculizumab.

Frequency of administration

1 times a day

Dosage per administration


Howard JF Jr, Nowak RJ, Wolfe GI, et al. 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 Neurol. 2020;77(5):582-592. doi:10.1001/jamaneurol.2019.5125, (NCT03315130, fase 2) (1), Studie-opzet fase 3-studie (NCT04115293) (2); NCT03971422)

Expected patient volume per year

Patient volume

150 - 300

Market share is generally not included unless otherwise stated.

Lanet Neurol. 2022. Juvenile myasthenia gravis: a nationwide study in Norway suggested an average annual incidence of 1·6 cases per 1 million people and a prevalence of 3·6–13·8 cases per 1 million people, with more girls affected than boys (1).
Additional remarks
In Nederland zijn geschat 3.000 patiënten met myasthenia gravis (167 per miljoen), de helft hiervan gebruikt immunosuppressiva (rond de1.500) en bij 10 tot 20% hiervan wordt wegens problemen met chronische stabiliteit derdelijns medicatie overwogen waaronder complementremmers (150 tot 300) (1).

Expected cost per patient per year

Additional remarks
Fabrikant geeft aan dat de prijs nog niet bekend is.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.