Padeliporfin Medicine 03 June 2025 Extended indication Padeliporfin monotherapy for first line treatment of low grade non-invasive Renal pelvis and ureter transitional cell cancer localized in adults and elderly. Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Padeliporfin Domain Oncology Reason of inclusion New medicine (specialité) Main indication Bladder cancer Extended indication Padeliporfin monotherapy for first line treatment of low grade non-invasive Renal pelvis and ureter transitional cell cancer localized in adults and elderly. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Steba Portfolio holder Mechanism of action Unknown Route of administration Intravenous Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP No Submission date September 2025 Expected Registration April 2026 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Tijdslijn op basis van IHSI-inschatting. Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Er is geen RCT fase 3 studie. Intensieve follow up noodzakelijk. Behoorlijke toxiciteit. Duration of treatment Frequency of administration Dosage per administration 3,66 mg/kg References NCT04620239; ENLIGHTED phase 3 study: Efficacy and safety of padeliporfin vascular targeted photodynamic therapy (VTP) for treatment of low-grade upper tract urothelial cancer (LG UTUC). (https://ascopubs.org/doi/10.1200/JCO.2024.42.16_suppl.TPS4622). Additional remarks Expected patient volume per year Patient volume < 120 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References IKNL2021(1); expertopinie (2) Additional remarks In 2021 waren er 1.453 gevallen van stadium 1 niercel carcinoma en 24 gevallen van stadium 1 carcinoom van de urethra. 350 per jaar hebben uppertract, waarvan een derde van deze groep low risk is (120 patiënten per jaar) (2). Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks