Lonafarnib Medicine 10 June 2020 Extended indication Zokinvy is indicated for the treatment of patients 12 months of age and older with a genetically confirmed diagnosis of Hutchinson-Gilford progeria syndrome or a processing-deficient progeroid laminopathy associated with either a heterozygous LMNA mutation with progerin-like protein accumulation or a homozygous or compound heterozygous ZMPSTE24 mutation. Therapeutic value No estimate possible yet Total cost € 2,800,000.00 Registration phase Registered Product Active substance Lonafarnib Domain Metabolism and Endocrinology Reason of inclusion New medicine (specialité) Main indication Metabolic diseases Extended indication Zokinvy is indicated for the treatment of patients 12 months of age and older with a genetically confirmed diagnosis of Hutchinson-Gilford progeria syndrome or a processing-deficient progeroid laminopathy associated with either a heterozygous LMNA mutation with progerin-like protein accumulation or a homozygous or compound heterozygous ZMPSTE24 mutation. Current proprietary name Already available biosimilars / generics Proprietary name Zokinvy 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 July 2022 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks Positieve CHMP opinie ontvangen in mei 2022. 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; SmPC Additional remarks Na 4 maanden behandeling met de startdosis van 115mg/m2 tweemaal daags, moet de dosis verhoogd tot de onderhoudsdosering van 150mg/m2 tweemaal daags (ochtend en avond). 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 € < 2,800,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both 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/. drugs.com Additional remarks De prijs zal in dezelfde range liggen als andere ultraweesgeneesmiddelen en dus aanzienlijk zijn. De kosten voor Zokinvy orale capsule 50mg zijn ongeveer $24.508 voor een levering van 30 capsules. Uitgaande van een lichaamsoppervlakte van 1,7m2 (gemiddelde vanuit de KNMP) en een behandelduur van een jaar komt dat neer op €2.800.000. Potential total cost per year Total cost € 2,800,000.00 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