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 Registered


Active substance Zilucoplan
Domain 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 ZILBRYSQ
Manufacturer UCB
Portfolio holder UCB
Mechanism of action Complement inhibitor
Route of administration Subcutaneous
Therapeutical formulation Injection
Budgetting framework Extramural (GVS)
Centre of expertise LUMC / MUCM+ / AUMC (loc. AMC)/ Radboud UMC / Erasmus MC
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 Yes
Registration phase Registered
Additional remarks Positieve CHMP-opinie in september 2023. Zorginstituut Nederland beoordeelt of zilucoplan (Zilbrysq®) vergoed kan worden. Start beoordeling dossier in mei 2024. Publicatie advies is nog niet bekend.

Therapeutic value

Current treatment options Cholinesterase inhibitors, corticosteroids, Intravenous, Immunosuppressants, Immunoglobulins, bloodplasma transfer, Rituximab, Eculizumab, (ravulizumab)
Therapeutic value Possible benefit in ease of use
Substantiation 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 em/of ravulizumab.
Duration of treatment Average day / days
Frequency of administration 1 times a day
Dosage per administration Afhankelijk lichaamsgewicht. De aanbevolen dosis komt overeen met ongeveer 0,3 mg/kg.
References 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) Howard JF Jr, Bresch S, Genge A, et al. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study. Lancet Neurol 2023; 22: 395–406.

Expected patient volume per year

Patient volume

150 - 300

Market share is generally not included unless otherwise stated.

References 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

References Fabrikant
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

Indication extension No

Other information

There is currently no futher information available.