Extended indication Generalized Myasthenia Gravis (in adult patients who are anti-acetylcholine receptor (AChR) antibo
Therapeutic value Possible equal value
Registration phase Registration application pending


Active substance Zilucoplan
Domain Neurological disorders
Reason of inclusion New medicine (specialité)
Main indication Muscular diseases other
Extended indication Generalized Myasthenia Gravis (in adult patients who are anti-acetylcholine receptor (AChR) antibody positive and require treatment in addition to steroids or non-steroidal immunosuppressants)
Manufacturer UCB
Portfolio holder UCB
Route of administration Subcutaneous
Therapeutical formulation Injection
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 November 2023
Orphan drug Yes
Registration phase Registration application pending

Therapeutic value

Current treatment options Cholinesterase inhibitors, corticosteroids, Immunosuppressants, Intravenous, Immunoglobulins, bloodplasma transfer, Rituximab, Eculizumab
Therapeutic value Possible equal value
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.
Frequency of administration 1 times a day
Dosage per administration tbd
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)

Expected patient volume per year

Patient volume

< 240

Market share is generally not included unless otherwise stated.

References 1) Spierziekten Nederland; Meriggioli M; Front Neurol Neurosci. 2009;26:94-108. doi: 10.1159/000212371; 3) Expert opinie
Additional remarks In Nederland wordt de prevalentie van gegeneraliseerde Myasthenia Gravis (gMG) patiënten geschat op 1 op 10.000 inwoners. Dit komt overeen met 1.750 patiënten uitgaande van 17,5 miljoen inwoners (1). Van de gMG patiënten heeft 85% anti- acetylcholine receptor antilichamen wat neerkomt op 1.488 patiënten (2). Op basis van gebruikersaantallen is de inschatting dat er waarschijnlijk 240 patiënten in aanmerking komen (3). Er rekening mee houdend dat een deel van de patiënten kortdurend met IVIG of plasmaferse behandeld wordt in een acute crisis en niet als onderhoudsbehandeling. Dit is echter nog niet zeker .

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

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.