Ponatinib Medicine 04 December 2025 Extended indication Extension of Indication to include treatment of paediatric patients aged 6 years and older with chronic phase chronic myeloid leukaemia (CP-CML) who are resistant or intolerant to at least one tyrosine kinase inhibitor Therapeutic value Total cost Registration phase Registration application pending Product Active substance Ponatinib Domain Hematology Reason of inclusion Indication extension IND Main indication CML Extended indication Extension of Indication to include treatment of paediatric patients aged 6 years and older with chronic phase chronic myeloid leukaemia (CP-CML) who are resistant or intolerant to at least one tyrosine kinase inhibitor Current proprietary name Already available biosimilars / generics Proprietary name Iclusig Manufacturer Incyte Portfolio holder Incyte Mechanism of action Tyrosine kinase inhibitor Route of administration Oral Therapeutical formulation Capsule Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date October 2025 Expected Registration November 2026 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value Substantiation Duration of treatment Frequency of administration Dosage per administration References NCT04501614 (Ponatinib-1501); NCT03934372 (INCB 84344-102) Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks 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 Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks