Explore

Companies

Drugs

About·Data Management·Terms·Privacy

Stay informed

© 2026 VivoSeq. All rights reserved.

Zokinvy

(lonafarnib)

ApprovedSmall molecule

SSentynl Therapeutics, Inc.

Lonafarnib is used to treat progeria, a rare genetic disease that causes rapid aging in children. It works by slowing down the production of an abnormal protein that damages cells.[2] Zokinvy is currently approved and in use across 6 indications[4].[1]

Zokinvy is one of 176 small molecule programs targeting Progeroid Syndrome. It is currently in Approved.

6

Indications

27

Total Trials

2

Active Trials

175

Competitors

4

FDA Applications

NDA/BLA/ANDA regulatory filings

1

Patents

Pipeline

Status by Indication[4][1]

Indication

Progeroid SyndromeActive, not recruiting

MetastasisApproved

Myelodysplastic SyndromeApproved

Central Nervous System NeoplasmApproved

Hutchinson-Gilford Progeria SyndromeActive, not recruiting

Glioblastoma MultiformeActive, not recruiting

Progeroid SyndromeApproved

Active, not recruiting

MetastasisApproved

Approved

Myelodysplastic SyndromeApproved

Approved

Central Nervous System NeoplasmApproved

Approved

Hutchinson-Gilford Progeria SyndromePhase 2

Active, not recruiting

Glioblastoma MultiformePhase 1

Active, not recruiting

Competition

Competitive Landscape

64 competitors in Metastasis

#DrugCompanyPhaseTrials

Zokinvy

SSentynl Therapeutics, Inc.

Verzenio

Zometa

Taxol

Tukysa

Lynparza

Neoral

Norvir

Gemzar

Johnson & Johnson

Camptosar

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Clinical

Trials by Indication[1]

Advanced Cancer3 trials

Interaction of Docetaxel and Lonafarnib in Patients With Advanced Cancer

Terminated·EEmory University·Phase 1·NCT00288444·38 enrolled

SCH 66336 Plus Gemcitabine in Treating Patients With Advanced Cancer of the Urinary Tract

Completed·EEuropean Organisation for Research and Treatment of Cancer - EORTC·Phase 2·NCT00006351·34 enrolled

Combination Chemotherapy in Treating Patients With Advanced Cancer

Completed·MMemorial Sloan Kettering Cancer Center·Phase 1·NCT00003956·25 enrolled

Breast Cancer3 trials

Lonafarnib in Metastatic Breast Cancer

Terminated·GGeorge Sledge·Phase 2·NCT00773474·29 enrolled

Lonafarnib, Trastuzumab, and Paclitaxel in Treating Patients With HER2/Neu-Overexpressing Stage IIIB, Stage IIIC, or Stage IV Breast Cancer

Completed·EEuropean Organisation for Research and Treatment of Cancer - EORTC·Phase 1·NCT00068757·23 enrolled

Anastrozole Plus Lonafarnib (SCH 66336) or Plus Placebo for the Treatment of Advanced Breast Cancer (P03480)

Completed· Merck & Co. logo

Merck & Co.·Phase 2·NCT00081510·110 enrolled

Hepatitis2 trials

Treatment of Chronic Delta Hepatitis With Lonafarnib and Ritonavir

Completed·NNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)·Phase 2·NCT02511431·22 enrolled

Treatment of Chronic Delta Hepatitis With Lonafarnib, Ritonavir and Lambda Interferon

Completed·NNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)·Phase 2·NCT03600714·26 enrolled

Hutchinson-Gilford progeria syndrome2 trials

Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome or Progeroid Laminopathy

Approved for marketing·EEiger BioPharmaceuticals··NCT03895528

Study to Determine Optimal Dose and Evaluate Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS)

Active, not recruiting·PPRG Science & Technology Co., Ltd.·Phase 2·NCT06775041·10 enrolled

Chronic Hepatitis1 trial

Lonafarnib for Chronic Hepatitis D

Completed·NNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)·Phase 2·NCT01495585·14 enrolled

14 more indications

Efficacy and Safety1 trial

Study of the Efficacy and Safety of Lonafarnib / Ritonavir With and Without Pegylated Interferon -Alfa-2a

Completed·EEiger BioPharmaceuticals·Phase 3·NCT03719313·407 enrolled

Non-Small Cell Lung Cancer1 trial

Study of Lonafarnib in Combination With Paclitaxel and Carboplatin in Patients With Non-Small Cell Lung Cancer (Study P01901)(TERMINATED)

Terminated· Merck & Co. logo

Merck & Co.·Phase 3·NCT00050336·702 enrolled

Chronic Myelogenous Leukemia1 trial

Study of Lonafarnib and Gleevec in Chronic Myelogenous Leukemia

Completed·MM.D. Anderson Cancer Center·Phase 1·NCT00047502·23 enrolled

TED1 trial

Lonafarnib Boosted With Ritonavir With and Without Peginterferon Alfa-2a (PEG IFN-a) in HDV (LOWR-2)

Completed·EEiger BioPharmaceuticals·Phase 2·NCT02430194·55 enrolled

Glioblastoma Multiforme1 trial

Lonafarnib and Temozolomide in Treating Patients with Glioblastoma Multiforme That is Recurrent or Did Not Respond to Previous Treatment with Temozolomide

Active, not recruiting·MM.D. Anderson Cancer Center·Phase 1·NCT00102648·34 enrolled

Progeria1 trial

A Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria

Completed·—·Phase 2·NCT00879034·5 enrolled

Liver Cancer1 trial

SCH 66336 With or Without Gemcitabine Followed by Surgery Compared With Surgery Alone in Treating Patients With Primary Liver Cancer

Withdrawn·JJonsson Comprehensive Cancer Center·Phase 2·NCT00020774

Hepatitis D Virus Infection1 trial

Once Daily Dosing of Lonafarnib Co-administered With Ritonavir for Treatment of Chronic Hepatitis D Virus Infection

Unknown status·SSoroka University Medical Center·Phase 3·NCT05229991·30 enrolled

Ovarian Cancer1 trial

Comparison of Paclitaxel/Carboplatin and Lonafarnib to Paclitaxel/Carboplatin for First-line Treatment of Ovarian Cancer

Completed·AAGO Study Group·Phase 2·NCT00281515·105 enrolled

Brain Tumors1 trial

SCH 66336 in Treating Children With Recurrent or Progressive Brain Tumors

Completed·PPediatric Brain Tumor Consortium·Phase 1·NCT00015899·53 enrolled

Chronic Myelomonocytic Leukemia1 trial

Study of Lonafarnib Versus Placebo in Subjects With Either Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMML) (Study P02978AM3)(TERMINATED)

Terminated· Merck & Co. logo

Merck & Co.·Phase 3·NCT00109538·47 enrolled

Phase II1 trial

Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria

Completed·—·Phase 2·NCT00425607·29 enrolled

Glioma1 trial

Lonafarnib and Temozolomide in Treating Patients With Recurrent Primary Supratentorial Gliomas

Unknown status·EEuropean Organisation for Research and Treatment of Cancer - EORTC·Phase 1·NCT00083096·30 enrolled

Unspecified3 trials

Lonafarnib With and Without Ritonavir in HDV (LOWR-1)

Completed·EEiger BioPharmaceuticals·Phase 2·NCT02430181·21 enrolled

Titrating-Dose of Lonafarnib in Combination With Ritonavir

Completed·EEiger BioPharmaceuticals·Phase 2·NCT02527707·15 enrolled

A Study of Lonafarnib With or Without Ritonavir in Patients With HDV

Withdrawn·EEiger BioPharmaceuticals·Phase 2·NCT02968641

Regulatory

FDA Approval Timeline[5]

Nov 20, 2020

ZOKINVY

NDA NDA213969

Nov 20, 2020

ZOKINVY

NDA NDA213969

Mar 21, 2024

ZOKINVY

NDA NDA213969

Mar 21, 2024

ZOKINVY

NDA NDA213969

Sources

References

[1]
ClinicalTrials.gov — 27 trial records for lonafarnib. U.S. National Library of Medicine, National Institutes of Health.
[2]
MedlinePlus Drug Information — Zokinvy drug information. U.S. National Library of Medicine, National Institutes of Health.
[3]
ChEMBL — lonafarnib compound record. European Bioinformatics Institute (EMBL-EBI).
[4]
Open Targets Platform — Drug–disease associations for lonafarnib. EMBL-EBI & Wellcome Sanger Institute.
[5]
OpenFDA — FDA approval records (NDA NDA213969). U.S. Food & Drug Administration.
[6]
FDA Orange Book — Patent and exclusivity data for Zokinvy. U.S. Food & Drug Administration.
[7]
SEC EDGAR — Sentynl Therapeutics, Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Protein Farnesyltransferase Inhibitor[3]
Modality: Small molecule[3]
Highest Phase: Approved[1]
Company: SSentynl Therapeutics, Inc.

Patent Landscape[6]

Patent Cliff

Jul 26, 2029

Earliest patent expiration — generic entry possible after this date

Patent #Expires

7838531Jul 26, 2029

Related News