Cabozantinib Medicine 12 June 2018 Extended indication Cabometyx is indicated as monotherapy for the treatment of hepatocellular carcinoma (HCC) in adults who have previously been treated with sorafenib. Therapeutic value Possible added value Total cost € 3,950,000.00 Registration phase Registered and reimbursed Product Active substance Cabozantinib Domain Oncology and Hematology Reason of inclusion Indication extension IND Main indication Liver cancer Extended indication Cabometyx is indicated as monotherapy for the treatment of hepatocellular carcinoma (HCC) in adults who have previously been treated with sorafenib. Current proprietary name Already available biosimilars / generics Proprietary name Cabometyx Manufacturer Ipsen Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Tablet Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Inhibitor of specific receptor tyrosine kinases. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date March 2018 Expected Registration November 2018 Orphan drug No Registration phase Registered and reimbursed Reimbursement Medicine sluice Additional remarks Scrip pipeline watch 19 januari 2018: Updated Phase III Results. CELESTIAL; improved overall survival, PFS. Positieve CHMP-opinie september 2018. Geregistreerd in november 2018. Therapeutic value Current treatment options Sorafenib Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Langere OS en PFS versus placebo na resistentie tegen sorafenib. Duration of treatment Average 3.8 month / months Frequency of administration 1 times a day Dosage per administration 60 mg References NCT01908426; Abou-Alfa et al. N Engl J Med. 2018 Jul 5;379(1):54-63 Additional remarks Expected patient volume per year Patient volume < 158 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR Additional remarks Totaal aantal patiënten in 2016 met hepatocellulair carcinoom stadium 4 betreft 158. Expected cost per patient per year Cost € 25,000.00 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 Medicijnkosten.nl; Farmacotherapeutisch Kompas Additional remarks Op basis van de gemiddelde behandelduur van 3,8 maanden zal de patiënt gemiddeld 114 tabletten verbruiken. Dit komt neer op een totaal van €24.320 per patiënt per jaar. Potential total cost per year Total cost € 3,950,000.00 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 Prostate Cancer, Carcinoid Tumor References Clinicaltrials.gov Additional remarks Other information Additional remarks