Extended indication Generalized Myasthenia Gravis
Therapeutic value No estimate possible yet
Registration phase Registration application pending

Product

Active substance Zilucoplan
Domain Neurological disorders
Reason of inclusion New medicine (specialité)
Main indication Neurological disorders other
Extended indication Generalized Myasthenia Gravis
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 comments 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

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date 2022
Expected Registration 2023
Registration phase Registration application pending
Additional comments submission date expected late second half of 2022

Therapeutic value

Current treatment options Cholinesterase inhibitors, corticosteroids, Immunosuppressants, Intravenous, Immunoglobulins, bloodplasma transfer, Rituximab, Eculizumab
Therapeutic value No estimate possible yet
Substantiation Fase 2 studie 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

< 1,530

Market share is generally not included unless otherwise stated.

References Bol P. Myasthenia gravis. Ned Tijdschr Tandheelkd. 2001; 108:416-7
Additional comments De prevalentie van myasthenia gravis ligt wereldwijd tussen de 5 en 20 op de 100.000 inwoners (1). In Nederland is de prevalentie geschat op 8 tot 9 op de 100.000 (1). In NL zou dit neerkomen op 1360-1530 (uitgaande van 17 miljoen inwoners). Het product zal moeten concurreren met andere middelen in de pijplijn voor deze indicatie. Het is op dit moment nog niet mogelijk een inschatting te doen welk middel welke plaats in de behandeling krijgt.

Expected cost per patient per year

There is currently nothing known about the expected cost.

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.