Version 5

Daxibotulinumtoxine A

Medicine

Extended indication Adults With Isolated Cervical Dystonia
Therapeutic value No estimate possible yet
Registration phase Clinical trials

Product

Active substance Daxibotulinumtoxine A
Domain Neurological disorders
Reason of inclusion New medicine (specialité)
Main indication Muscular diseases other
Extended indication Adults With Isolated Cervical Dystonia
Manufacturer Revance
Mechanism of action Unknown
Route of administration Intramuscular
Therapeutical formulation Injection
Budgetting framework Intermural (MSZ)

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Particularity Unknown
ATMP No
Submission date 2024
Expected Registration 2025
Orphan drug No
Registration phase Clinical trials
Additional remarks Primary completion date: december 2019

Therapeutic value

Current treatment options botulinetoxine A, Trihexyfenidyl
Therapeutic value No estimate possible yet

This assessment does not indicate any potential inclusion in the package.
References https://movementdisorders.onlinelibrary.wiley.com/doi/epdf/10.1002/mdc3.12613 Jankovic J, Truong D, Patel AT, Brashear A, Evatt M, Rubio RG, Oh CK, Snyder D, Shears G, Comella C. Injectable DaxibotulinumtoxinA in Cervical Dystonia: A Phase 2 Dose-Escalation Multicenter Study. Mov Disord Clin Pract. 2018 Apr 26;5(3):273-282. doi: 10.1002/mdc3.12613. PMID: 30009213; PMCID: PMC6032882. NCT03608397 Behandel richtlijn cervicale dystonie
Additional remarks Proefpersonen met matige tot ernstige geïsoleerde cervicale dystonie werden geïncludeerd in opeenvolgende cohorten om een enkele open-label, intramusculaire dosis injecteerbare daxibotulinumtoxineA van maximaal 200U, 200 tot 300U of 300 tot 450U te ontvangen.

Expected patient volume per year

Patient volume 2,500

Market share is generally not included unless otherwise stated.
References Behandel richtlijn cervicale dystonie (1); Snaith, A., Wade, D., 2008. Dystonia. Clin.Evid.(Online.) 2008 (2); Expert opinie (3)
Additional remarks In Nederland zijn er geen epidemiologische studies gedaan naar CD. De schatting is dat er 8.000 patiënten in Nederland bekend zijn met een cervicale dystonie (1). De prevalentie van cervicale dystonie in Europa is 5.7/100.000 (2). Naar schatting over eerder gebruik van vergelijkbare middelen zal het gaan om ongeveer 2.500 patiënten (3).

Expected cost per patient per year

Potential total cost per year

Off label use

Off label use Unknown

Indication extension

Indication extensions Unknown

Other information