Varegacestat Medicine 06 June 2023 Extended indication Desmoïd tumor (aggressive fibromatosis). Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Varegacestat Domain Oncology Reason of inclusion New medicine (specialité) Main indication Oncology other Extended indication Desmoïd tumor (aggressive fibromatosis). Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Ayala Portfolio holder Ayala Mechanism of action Other, see general comments Route of administration Unknown Therapeutical formulation Unknown Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Werkingsmechanisme: varegacestat (AL102) is an inhibitor of gamma secretase-mediated Notch signaling. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP No Submission date December 2025 Expected Registration January 2027 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Estimated Primary Completion Date: 12 januari 2025, maar mogelijk al eerder verwacht. Fase 2 en 3 studie besproken op ESMO 2022. Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Op dit moment zijn er nog enkel resultaten over de veiligheid van het geneesmiddel bekend. Duration of treatment Frequency of administration 1 times a day Dosage per administration 1,2 mg References NCT04871282; https://www.globenewswire.com/news-release/2022/11/17/2558123/0/en/Ayala-Pharmaceuticals-Presents-Poster-on-AL102-in-Desmoid-Tumors-at-the-Connective-Tissue-Oncology-Society-CTOS-2022-Annual-Meeting.html Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References https://www.desmoid.nl/desmoid-tumor/ Additional remarks Van alle wekedelen tumoren (sarcoom) is de desmoïd tumor (synoniem voor desmoïd-type fibromatose en agressieve fibromatose) een zeldzame variant, met ongeveer 3% van het totale aantal wekedelen tumoren, hetgeen betekent dat er in Nederland slechts enkele tientallen nieuwe patiënten per jaar worden gediagnosticeerd. 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 No Indication extensions References AdisInsight Additional remarks Geen andere lopende fase 2 / fase 3 studies Other information Additional remarks