Detalimogene voraplasmid Medicine 04 December 2025 Extended indication Detalimogene voraplasmid monotherapy for second line treatment of BCG-unresponsive non-muscle invasive bladder cancer in adults and elderly with carcinoma in situ (CIS) with or without coexisting papillary Ta/T1 tumors. Therapeutic value No estimate possible yet Total cost € 109,375,000.00 Registration phase Clinical trials Product Active substance Detalimogene voraplasmid Domain Oncology Reason of inclusion New medicine (specialité) Main indication Bladder cancer Extended indication Detalimogene voraplasmid monotherapy for second line treatment of BCG-unresponsive non-muscle invasive bladder cancer in adults and elderly with carcinoma in situ (CIS) with or without coexisting papillary Ta/T1 tumors. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer enGene Portfolio holder enGene Mechanism of action Route of administration Intravesical Therapeutical formulation Liquid Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP Submission date September 2026 Expected Registration October 2027 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Radicale cystectomie, chemohyperthermie (HIVEC®) als blaas besparend alternatief, off-label intravesicale chemotherapie (gemcitabine/docetaxel), en in studieverband of beperkt off-label immunotherapie zoals pembrolizumab Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation De LEGEND fase II studie onderzoekt detalimogene voraplasmid bij BCG-ongevoelige blaaskankerpatiënten met CIS. 71% van de behandelde patiënten bereikte een complete respons (67% na drie maanden en 47% na zes maanden). De studie is nog gaande. Duration of treatment Frequency of administration 1 times a week Dosage per administration 0,5mg detalimogene voraplasmid per instillatie References NCT04752722 (LEGEND); Expertopinie Additional remarks Behandelduur: – Inductie: zes weken – Mogelijk onderhoudsbehandeling tot maximaal een jaar Expected patient volume per year Patient volume 450 - 800 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Babjuk et al. 2024 (1); NKR 2023 (2); Kamat et al. 2014 (3) Additional remarks Jaarlijks krijgen ongeveer 6.500 mensen in Nederland blaaskanker, waarvan circa 5.000 patiënten een niet-spierinvasieve vorm (NMIBC) hebben (IKNL, 2023). Van deze groep worden ongeveer 1.500 tot 2.000 patiënten behandeld met BCG vanwege hooggradige Ta/T1-laesies of carcinoma in situ (CIS) (1,2). Naar schatting wordt 30 tot 40% BCG-ongevoelig, wat neerkomt op 450 tot 800 patiënten per jaar (3) Expected cost per patient per year Cost € 150,000.00 - 200,000.00 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 ICER; Drugs.com Additional remarks Er is nog geen marktprijs beschikbaar. Een vergelijkbare behandeling nadofaragene firadenovec (Adstiladrin) kost in de Verenigde Staten ongeveer $240.000 per jaar. Schatting cretostimogene grenadenorepvec: €150.000 tot €200.000 per jaar. Potential total cost per year Total cost € 109,375,000.00 Total cost for sluice Additional remarks Off label use Off label use Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks