Extended indication Epidermolysis bullosa
Therapeutic value Possible added value
Registration phase Registration application pending

Product

Active substance Berkenschorsextract
Domain Chronic immune diseases
Reason of inclusion New medicine (specialité)
Main indication Skin diseases
Extended indication Epidermolysis bullosa
Proprietary name Filsuvez
Manufacturer Amryt
Route of administration Local
Therapeutical formulation Gel
Budgetting framework Extramural (GVS)

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date March 2021
Expected Registration April 2022
Orphan drug Yes
Registration phase Registration application pending

Therapeutic value

Current treatment options Palliatieve zorg
Therapeutic value Possible added value
Substantiation Er is momenteel geen behandeling voorhanden die de aandoening kan genezen.
Duration of treatment continuous
Frequency of administration 1 times every 3 days
Dosage per administration 10%
References NCT03068780
Additional comments De behandelingsduur zal mogelijk duren tot wondgenezing. De toedieningsfrequentie is 1 maal per 3-4 dagen.

Expected patient volume per year

Patient volume

< 464

Market share is generally not included unless otherwise stated.

References Baarman et al. J Eur Acad Dermatol Venereol. 2020 Oct 23.
Additional comments In Nederland zijn momenteel 464 patiënten bekend. Aangezien er momenteel geen goede behandelopties zijn zullen er veel van deze patiëntengroep in aanmerking kunnen komen.

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

Additional comments Epidermolysis Bullosa is geen immunologische aandoening maar is toegevoegd aan het domein Chronische Immuunziekten vanwege technische redenen.