Lonafarnib Medicine 12 June 2019 Extended indication Hepatitis D infection with ritonavir Therapeutic value Possible added value Total cost Registration phase Clinical trials Product Active substance Lonafarnib Domain Infectious diseases Reason of inclusion New medicine (specialité) Main indication Viral infections other Extended indication Hepatitis D infection with ritonavir Current proprietary name Already available biosimilars / generics Proprietary name Sarasar Manufacturer Eiger Bio Portfolio holder Mechanism of action Virus inhibitor Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks This medicine works by blocking an enzyme in the virus responsible for a chemical reaction called 'farnesylation', which involves the addition of a chemical group (farnesyl) to certain proteins. The hepatitis delta virus requires the farnesylation of its major protein in order to reproduce itself. By blocking this reaction, it is expected that the medicine will interfere with the ability of the hepatitis delta virus to reproduce, reducing the amount of the virus in the body and thereby helping to reduce damage to the liver. Registration Registration route Centralised (EMA) Type of trajectory Accelerated assessment Particularity New medicine with Priority Medicines (PRIME) ATMP Unknown Submission date 2021 Expected Registration 2022 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks PRIME-status verkregen in december 2018. Therapeutic value Current treatment options Geen Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Uitbreiding van behandelingsmogelijkheden voor hepatitis delta heeft zeker therapeutische waarde, gezien de afwezigheid van mogelijkheden tot nu toe. Duration of treatment Frequency of administration 2 times a day Dosage per administration 25 mg References NCT02968641 Additional remarks Wordt gebruikt in combinatie met ritonavir 100mg tweemaal per dag. Expected patient volume per year Patient volume < 2,500 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Derickx et al. Ned Tijdschr Geneeskd. 2011;155:A3513 Additional remarks Het aantal patiënten met hepatitis delta (hetgeen alleen maar voorkomt bij actieve hepatitis B) is in Nederland heel klein. De prevalentie van chronisch geïnfecteerde patiënten in Nederland wordt geschat op 2.500. Expected cost per patient per year Cost References Additional remarks Afhankelijk van de therapeutische waarde, gezien de PRIME-status die is afgegeven door de EMA, is het een veelbelovende therapie en dus mogelijk duur. Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use No Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Progeria References Clinicaltrials.gov Additional remarks Other information Additional remarks