Extended indication Hepatitis D infection with ritonavir
Therapeutic value Possible added value
Registration phase Clinical trials

Product

Active substance Lonafarnib
Domain Infectious diseases
Reason of inclusion New medicine (specialité)
Main indication Other viral infections
Extended indication Hepatitis D infection with ritonavir
Proprietary name Sarasar
Manufacturer Eiger Bio
Mechanism of action Virus inhibitor
Route of administration Oral
Therapeutical formulation Capsule
Budgetting framework Extramural (GVS)
Additional comments 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)
Submission date 2021
Expected Registration 2022
Orphan drug Yes
Registration phase Clinical trials
Additional comments PRIME-status verkregen in december 2018.

Therapeutic value

Current treatment options Geen
Therapeutic value Possible added value
Substantiation Uitbreiding van behandelingsmogelijkheden voor hepatitis delta heeft zeker therapeutische waarde, gezien de afwezigheid van mogelijkheden tot nu toe.
Frequency of administration 2 times a day
Dosage per administration 25 mg
References NCT02968641
Additional comments 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.

References Derickx et al. Ned Tijdschr Geneeskd. 2011;155:A3513
Additional comments 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

Additional comments 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

There is currently nothing known about the possible total cost.

Off label use

Off label use No

Indication extension

Indication extension Yes
Indication extensions Progeria
References Clinicaltrials.gov

Other information

There is currently no futher information available.