Eliglustat Medicine 04 June 2024 Extended indication Extension of indication to include treatment of paediatric patients with GD1 who are 6 years and older with a minimum body weight of 15 kg, who have been previously treated with enzyme replacement therapy (ERT), and who are CYP2D6 poor metabolisers (PMs), intermediate metabolisers (IMs) or extensive metabolisers (EMs) Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Eliglustat Domain Metabolism and Endocrinology Reason of inclusion Indication extension IND Main indication Metabolic diseases Extended indication Extension of indication to include treatment of paediatric patients with GD1 who are 6 years and older with a minimum body weight of 15 kg, who have been previously treated with enzyme replacement therapy (ERT), and who are CYP2D6 poor metabolisers (PMs), intermediate metabolisers (IMs) or extensive metabolisers (EMs) Current proprietary name Already available biosimilars / generics Proprietary name Cerdelga Manufacturer Sanofi Portfolio holder Mechanism of action Unknown Route of administration Unknown Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Glucosylceramide synthase inhibitors Registration Registration route Unknown Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date December 2023 Expected Registration September 2024 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks NCT03485677 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 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