Lonafarnib Medicine 10 June 2020 Extended indication Hutchinson-Gilford Progeria Syndrome and Progeroid Laminopathies. Therapeutic value No judgement Total cost Registration phase Registration application pending Product Active substance Lonafarnib Domain Metabolism and Endocrinology Reason of inclusion New medicine (specialité) Main indication Metabolic diseases Extended indication Hutchinson-Gilford Progeria Syndrome and Progeroid Laminopathies. Current proprietary name Already available biosimilars / generics Proprietary name Sarasar Manufacturer Eiger Bio Portfolio holder Mechanism of action Virus inhibitor Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Accelerated assessment Particularity Unknown ATMP Unknown Submission date April 2020 Expected Registration December 2020 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 NCT02579044 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