Pacritinib Medicine 12 June 2018 Extended indication Patients with myelofibrosis who have thrombocytopenia. Therapeutic value No estimate possible yet Total cost € 945,000.00 Registration phase Clinical trials Product Active substance Pacritinib Domain Hematology Reason of inclusion New medicine (specialité) Main indication Myeloproliferative disorders Extended indication Patients with myelofibrosis who have thrombocytopenia. Current proprietary name Already available biosimilars / generics Proprietary name Epjevy Manufacturer Cti Portfolio holder Mechanism of action JAK tyrosine kinase inhibitor Route of administration Oral Therapeutical formulation Capsule Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Tyrosine kinase inhibitor gericht tegen JAK2 en FLT3. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date March 2027 Expected Registration April 2028 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Pacritinib was al eerder ingediend in juni 2016 maar werd teruggetrokken in maart 2017 vanwege tijd tekort om nieuwe data in de aanvraag te includeren. De aanvraag die in juli 2017 is ingediend is in februari 2019 teruggetrokken met als reden dat CTI niet is staat is data te leveren binnen de procedurele tijdslijn om de vragen van de EMA te adresseren. Therapeutic value Current treatment options Ruxolitinib Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Median 15.6 month / months Frequency of administration 1 times a day Dosage per administration 400mg References NCT03165734 (PACIFICA) Additional remarks Expected patient volume per year Patient volume 21 - 33 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR; Mesa et al. Lancet Haematol. 2017 May;4(5):e225-e236. Additional remarks 118 diagnoses in 2016. Trombocytopenie komt voor bij 18 tot 28% van de myelofibrosis patiënten (21 tot 33 patiënten). Expected cost per patient per year Cost € 30,000.00 - 40,000.00 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 GIPdatabank Additional remarks De vergoeding per patiënt in 2015 voor de huidige behandelmethode met ruxolitinib was €32.174,00. Potential total cost per year Total cost € 945,000.00 Total cost for sluice Additional remarks Off label use Off label use Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References Additional remarks Geen fase 3 studies voor andere indicaties dan in de Horizonscan geneesmiddelen Other information Additional remarks