Last reviewed 2026-04-20 · How we verify

Zokinvy (LONAFARNIB)

Lonafarnib prevents farnesylation of progerin and similar proteins by inhibiting farnesyltransferase.

Zokinvy (Lonafarnib) is a small molecule farnesyltransferase inhibitor developed by Eiger Biopharmaceuticals Inc and currently owned by Sentynl Therapeutics Inc. It targets the GTPase HRas, a protein involved in cell growth and division. Zokinvy is FDA-approved to treat Hutchinson-Gilford syndrome and processing-deficient progeroid laminopathies. The drug is patented and has no generic manufacturers. Key safety considerations include the need for careful monitoring of liver function and potential side effects such as diarrhea and fatigue.

At a glance

Mechanism of action

Lonafarnib works by blocking the enzyme farnesyltransferase, which is responsible for adding a farnesyl group to proteins like progerin. This prevents these proteins from accumulating in the inner nuclear membrane, where they can cause cellular damage.

Approved indications

• Hutchinson-Gilford syndrome
• Processingdeficient progeroid laminopathies

Common side effects

• Vomiting
• Diarrhea
• Nausea
• Abdominal pain
• Constipation
• Fatigue
• Pyrexia
• Infection
• Upper respiratory tract infection
• Rhinitis
• Decreased appetite (anorexia)
• Electrolyte abnormalities

Key clinical trials

• Study to Determine Optimal Dose and Evaluate Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS) (PHASE2)
• Lonafarnib and Temozolomide in Treating Patients with Glioblastoma Multiforme That is Recurrent or Did Not Respond to Previous Treatment with Temozolomide (PHASE1)
• Peginterferon Lambda and Lonafarnib Boosted With Ritonavir 48-Week Combination Therapy for Delta Hepatitis (PHASE2)
• Titrating-Dose of Lonafarnib in Combination With Ritonavir (PHASE2)
• Once Daily Dosing of Lonafarnib Co-administered With Ritonavir for Treatment of Chronic Hepatitis D Virus Infection (PHASE3)
• Study of the Efficacy and Safety of Lonafarnib / Ritonavir With and Without Pegylated Interferon -Alfa-2a (PHASE3)
• Lonafarnib Boosted With Ritonavir With and Without Peginterferon Alfa-2a (PEG IFN-a) in HDV (LOWR-2) (PHASE2)
• Phase I/II Trial of Everolimus in Combination With Lonafarnib in Progeria (PHASE1,PHASE2)

Patents

Primary sources

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