Extended indication Hutchinson-Gilford Progeria Syndrome and Progeroid Laminopathies.
Therapeutic value No judgement
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.
Proprietary name Sarasar
Manufacturer Eiger Bio
Mechanism of action Virus inhibitor
Route of administration Oral
Therapeutical formulation Capsule
Budgetting framework Extramural (GVS)

Registration

Registration route Centralised (EMA)
Type of trajectory Accelerated assessment
Submission date April 2020
Expected Registration December 2020
Orphan drug Yes
Registration phase Registration application pending

Therapeutic value

Therapeutic value No judgement
References NCT02579044

Expected patient volume per year

There is currently nothing known about the expected patient volume.

Expected cost per patient per year

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.