Features and properties of lonafarnib
| Alternative names | EBP 994; MK-6336; Sarasar; SCH 066336; SCH 66336; Zokinvy™ |
|---|---|
| Class | Amides; antineoplastics; antivirals; benzene derivatives; halogenated hydrocarbons; piperidines; pyridines; small molecules |
| Mechanism of Action | Inhibits farnesyltransferase to prevent farnesylation and subsequent accumulation of progerin and progerin-like proteins in the inner nuclear membrane |
| Route of Administration | Oral |
| Pharmacodynamics | Blocked farnesylation of progerin-transfected cells and restored normal nuclear architecture |
| Reduced nuclear blebbing in human HGPS fibroblasts | |
| Pharmacokinetics | Median tmax at steady state 2 and 4 h after 115 and 150 mg/m2 twice daily with food in patients with HGPS |
| Mean half-life ≈ 4–6 h after 100 mg twice daily in healthy subjects | |
| Most frequent adverse events | Vomiting, diarrhoea, infection, nausea, decreased appetite, fatigue, upper respiratory tract infection |
| ATC codes | |
| WHO ATC code | J05A-X (other antivirals); L01 (antineoplastic agents); N07 (other nervous system drugs) |
| EphMRA ATC code | J5B1 (viral hepatitis products); L1 (antineoplastics); N7 (other CNS drugs) |
| Chemical Name | 4-[2-[4-[(11R)-3,10-dibromo-8-chloro-6,11-dihydro-5Hbenzo[1,2] cyclohepta [2,4-b]pyridin-11-yl]piperidin-1-yl]-2- oxoethyl] piperidine-1-carboxamide |
HGPS Hutchinson-Gilford Progeria Syndrome, tmax time to peak plasma concentration