Elexacaftor / tezacaftor / ivacaftor Medicine 08 December 2020 Extended indication An Extension of indication for Sarclisa to add combination with carfilzomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy. Therapeutic value No judgement Total cost Registration phase Registration application pending Product Active substance Elexacaftor / tezacaftor / ivacaftor Domain Lung diseases Reason of inclusion Indication extension IND Main indication Cystic fibrosis Extended indication An Extension of indication for Sarclisa to add combination with carfilzomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy. Current proprietary name Already available biosimilars / generics Proprietary name Kaftrio Manufacturer Vertex Portfolio holder Mechanism of action Protein chaperone Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date September 2020 Expected Registration July 2021 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References 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 Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks