Lonafarnib Medicine 10 June 2020 Extended indication Hutchinson-Gilford Progeria Syndrome and Progeroid Laminopathies. Therapeutic value No estimate possible yet 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 Other, see general comments Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Protein farnesyltransferase inhibitor Registration Registration route Centralised (EMA) Type of trajectory Accelerated assessment Particularity Unknown ATMP Unknown Submission date April 2020 Expected Registration August 2021 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Uitsluitend symptoombehandeling Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment 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; Additional remarks Expected patient volume per year Patient volume > 1 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References orpha.net Additional remarks 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 Cost 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 remarks De prijs zal in dezelfde range liggen als andere ultraweesgeneesmiddelen en dus aanzienlijk zijn. 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 No Indication extensions References adisinsight Additional remarks Geen indicatie-uitbreidingen buiten de indicaties in de Horizonscan Geneesmiddelen. Other information Additional remarks