Eliglustat

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
Registration phase	Registration application pending

Product

Active substance	Eliglustat
Domain	Metabolism and Endocrinology
Reason of inclusion	Indication extension
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)
Proprietary name	Cerdelga
Manufacturer	Sanofi
Therapeutical formulation	Tablet
Budgetting framework	Extramural (GVS)
Additional remarks	Glucosylceramide synthase inhibitors

Submission date	December 2023
Expected Registration	September 2024
Orphan drug	No
Registration phase	Registration application pending
Additional remarks	NCT03485677

Therapeutic value	No estimate possible yet

There is currently nothing known about the expected patient volume.

There is currently nothing known about the expected cost.

There is currently nothing known about the possible total cost.

There is currently nothing known about off label use.

There is currently nothing known about indication extensions.

There is currently no futher information available.