Patidegib Medicine 04 June 2026 Extended indication Patidegib monotherapy for preventive treatment of PTCH1 mutation-positive Basal cell carcinoma face in adults and elderly with Gorlin Syndrome. Therapeutic value No estimate possible yet Total cost € 11,880,000.00 Registration phase Clinical trials Product Active substance Patidegib Domain Oncology Reason of inclusion New medicine (specialité) Main indication Skin cancer Extended indication Patidegib monotherapy for preventive treatment of PTCH1 mutation-positive Basal cell carcinoma face in adults and elderly with Gorlin Syndrome. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Sol-Gel Portfolio holder Sol-Gel Mechanism of action Route of administration Cutaneous Therapeutical formulation Gel Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Patidegib, ook bekend als Saridegib of IPI-926, is een remmer van smoothened (SMO), een G-proteïne-gekoppelde receptor die deel uitmaakt van de hedgehog-signaalroute. Remming van de hedgehog (Hh)-route vindt plaats via de celmembraan-overspannende G-proteïne-gekoppelde receptor SMO, wat resulteert in onderdrukking van de Hh-signaaloverdracht en een afname van de proliferatie van tumorcellen. Registration Registration route Centralised (EMA) Type of trajectory Particularity ATMP No Submission date December 2026 Expected Registration January 2028 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 2 times a day Dosage per administration References NCT06050122 Additional remarks 2% gel tweemaal daags plaatselijk op het gezicht aanbrengen. Expected patient volume per year Patient volume < 360 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Orphanet Additional remarks Het syndroom van Gorlin, ook wel bekend als het Basaalcel Naevus Syndroom (BCNS), is een zeldzame erfelijke aandoening. De incidentie wordt geschat op ongeveer 2 op de 100.000 mensen. Dit zou betekenen dat er maximaal 360 patiënten in Nederland in aanmerking zouden kunnen komen. Expected cost per patient per year Cost € 33,000.00 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 IHSI Additional remarks Kosten op basis van IHSI inschatting, afgeleid van de mediane kosten van dezelfde ATC-klasse (L01XJ) geneesmiddelen in Nederland. Potential total cost per year Total cost € 11,880,000.00 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