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 MorphoSys Portfolio holder MorphoSys Mechanism of action Unknown 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 Unknown Particularity Unknown ATMP No Submission date 2025 Expected Registration 2026 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 1 times a day Dosage per administration References NCT04603495 Additional remarks Een cyclus is 21 dagen waarbij op dag 1 tot en met 14 dagelijks wordt behandeld en dag 15 tot en met 21 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 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 Adis Insight Additional remarks Geen andere fase 3 studies op dit moment. Other information Additional remarks