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 Other, see general comments
Route of administration Oral
Therapeutical formulation Capsule
Additional comments Protein farnesyltransferase inhibitor

Registration

Registration route Centralised (EMA)
Type of trajectory Accelerated assessment
Submission date April 2020
Expected Registration April 2021
Orphan drug Yes
Registration phase Registration application pending

Therapeutic value

Current treatment options Uitsluitend symptoombehandeling
Therapeutic value No judgement
Frequency of administration 2 times a day
Dosage per administration 150 mg/m2
References NCT02579044; Leslie B. et al.Association of Lonafarnib Treatment versus No Treatment With Mortality Rate in Patients With Hutchinson-Gilford Progeria Syndrome;

Expected patient volume per year

Patient volume

> 1

Market share is generally not included unless otherwise stated.

References orpha.net
Additional comments Hutchinson-Gilford Progeria is zeer zeldzaam, de prevalentie ligt lager dan 1 op de 1.000.000. Er is op dit moment één patiënt bekend in Nederland.

Expected cost per patient per year

References https://endpts.com/eiger-nabs-an-fda-approval-for-progeria-an-ultra-rare-premature-aging-disease-after-merck-gave-them-the-drug-for-free/
Additional comments De prijs zal in dezelfde range liggen als andere ultraweegeneesmiddelen en dus aanzienlijk zijn.

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

Indication extension No
References adisinsight
Additional comments Geen indicatie-uitbreidingen buiten de indicaties in de Horizonscan

Other information

There is currently no futher information available.