Pelabresib Medicine 06 June 2023 Extended indication Myelofibrosis Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Pelabresib Domain Hematology Reason of inclusion New medicine (specialité) Main indication Myeloproliferative disorders Extended indication Myelofibrosis Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Novartis Portfolio holder Novartis Mechanism of action Route of administration Oral Therapeutical formulation Tablet Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks MoA Bromodomain and extraterminal domain protein inhibitors. Registration Registration route Centralised (EMA) Type of trajectory Particularity ATMP No Submission date September 2026 Expected Registration October 2027 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Fabrikant verwacht registratie in oktober 2027. 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 1 times a day Dosage per administration References NCT04603495 Additional remarks Een cyclus is 21 dagen waarbij op dag een tot en met veertien dagelijks wordt behandeld en dag vijftien tot en met eenentwintig geen behandeling wordt gegeven. Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR (1) Additional remarks In 2020 waren er 111 patiënten met Myelofibrosis.(1) Expected cost per patient per year Cost References Additional remarks Er zijn nog geen gegevens bekend over de kosten. Potential total cost per year Total cost 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 Adis Insight Additional remarks Geen andere fase 3 studies op dit moment. Other information Additional remarks