Spesolimab Medicine 08 June 2021 Extended indication Patients with Generalized pustular psoriasis (GPP) who experiencing acute moderate or severe disease flares. Therapeutic value Possible added value Total cost € 600,000.00 Registration phase Registration application pending Product Active substance Spesolimab Domain Chronic immune diseases Reason of inclusion New medicine (specialité) Main indication Skin diseases Extended indication Patients with Generalized pustular psoriasis (GPP) who experiencing acute moderate or severe disease flares. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Boehringer Ingelheim Portfolio holder Mechanism of action Antibody-drug conjugate Route of administration Intravenous Therapeutical formulation Solution Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Accelerated assessment Particularity Unknown ATMP No Submission date October 2021 Expected Registration January 2023 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Generalized pustular psoriasis is an extremely rare type of psoriasis that can present in a variety of forms. Unlike the most general and common forms of psoriasis, Generalized pustular psoriasis usually covers the entire body and with pus-filled blisters rather than plaques. Generalized pustular psoriasis can present at any age, but is rarer in young children. It can appear with or without previous psoriasis conditions or history, and can reoccur in periodic episodes. Therapeutic value Current treatment options Geen. Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Er is op dit moment geen goede behandeling voor deze indicatie. Dit betekent dat spesolimab mogelijk een grote therapeutische meerwaarde voor de patiënt zal hebben. De fase 3 studie loopt op dit moment nog. Duration of treatment Median 12 week / weeks Frequency of administration Dosage per administration References NCT03782792 Additional remarks De fabrikant geeft aan dat de behandelduur 12 weken betreft. Expected patient volume per year Patient volume 30 - 50 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Mirza HA, Badri T, Kwan E. Generalized Pustular Psoriasis. [Updated 2020 Sep 15]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2020 Jan-. Additional remarks In Frankrijk wordt de prevalentie van Generalized pustular psoriasis geschat op 1,76 per miljoen inwoners. Voor Nederland zou dit neerkomen op ongeveer 30 patiënten. De fabrikant schat in dat in Nederland mogelijk 30-50 patiënten hiervoor in aanmerking kan komen maar dat maar 80% van de GPP patiënten kan te maken krijgen met GPP flares waarvoor Spesolimab een uitkomst kan bieden. Expected cost per patient per year Cost € > 15,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References Fabrikant Additional remarks Potential total cost per year Total cost € 600,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions GPP maintenance (2024), Palmoplantar pustulosis (2028), Crohn's disease (2027) References Fabrikant Additional remarks Een indicatie uitbreiding naar Palmoplantar pustulosis zal in 2028 mogelijk voor een groei in het patiëntvolume zorgen. Prevalentie 0.05% (=max 8.500 patiënten in Nederland) waarvan een deel lokaal of met conventionele systemische middelen kan worden behandeld. Other information Additional remarks