Raw research findings for this medication. Mostly single studies, not curated or confirmed robust.
Lonafarnib is a farnesyltransferase inhibitor, the first medicine approved (2020) for Hutchinson-Gilford progeria syndrome and certain related progeroid laminopathies, rare conditions of rapid ageing in children. It blocks the attachment of a fatty anchor to progerin, the faulty protein that damages the cell nucleus, and extends survival. It is also used for chronic hepatitis D. It is sold as Zokinvy.
Lonafarnib is meant for progeria caused by a specific LMNA variant that produces progerin, and for related processing-deficient laminopathies. It is broken down mainly by CYP3A4. The gene study listed looked at FNTB, the gene for part of its target enzyme. There is no CPIC or DPWG guideline.
Every published finding on gene variants and Lonafarnib. Upload your DNA file to see which of these rows apply to you.
PharmGKB weighs all studies of a variant and rates how strong the evidence is, from 1A (strongest) to 4.
FNTB rs11623866: Level 3. Efficacy: Ovarian Neoplasms. 1.
Studies of how well it worked in people carrying a variant.
Study findings and evidence levels: PharmGKB (CC BY-SA 4.0). Guidelines: CPIC and DPWG, as bundled in PharmCAT. Most studies were done mainly in people of European ancestry.
More drugs from the same category
Tipifarnib (Farnesyltransferase Inhibitors)
Not a prescribing recommendation. This page is educational information about pharmacogenomic research and clinical guidelines - it is not medical or dosing advice, and not a prediction of how any medication will work for you. Never start, stop, or change a medication or dose based on it; talk to your doctor or pharmacist, who can order clinical-grade testing if appropriate.
Content updated September 2026.
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