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Drug

Lonafarnib

Approved · EMA
Emerging researchLate-stage development

Also known as Zokinvy, Lonafarnibum.

RxNorm2467553ChEMBLCHEMBL298734UNIIIOW153004F
1
Research papers
2
Active clinical trials
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Phase 2a, Randomized, Open-Label Study to Determine the Optimal Dose and Evaluate the Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS)

Clinical trial2026-03-01Primary completion · ClinicalTrials.gov

Phase I/Ib Study of Sarasar and Temodar in Patients with Recurrent or Temodar-Refractory Glioblastoma Multiforme

Clinical trial2025-03-01Primary completion · ClinicalTrials.gov

Once Daily (QD) Dosing of Lonafarnib (LNF) Co-administered With Ritonavir (RTV) for Treatment of Chronic Hepatitis D Virus Infection

Clinical trial2025-02-05Completed · ClinicalTrials.gov

A Phase 2, Open-Label Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Activity of a Titrating-Dose Lonafarnib/Ritonavir in Patients Chronically Infected With Hepatitis Delta Virus

Clinical trial2023-06-22Results posted · ClinicalTrials.gov

A Phase 3, Matrix Design, Partially Double-Blind, Randomized Study of the Efficacy and Safety of 50 mg Lonafarnib/100 mg Ritonavir BID With and Without 180 mcg PEG IFN-alfa-2a for 48 Weeks Compared With PEG IFN-alfa-2a Monotherapy and Placebo Treatment in Patients Chronically Infected With Hepatitis Delta Virus Being Maintained on Anti-HBV Nucleos(t)Ide Therapy (D-LIVR)

Clinical trial2023-03-24Completed · ClinicalTrials.gov

An Open-label, Dose-ranging, Proof-of-Concept Study to Evaluate the Safety and Efficacy of Lonafarnib With Ritonavir-Boosting +/- Peginterferon Alfa-2a in Patients Chronically Infected With Delta Hepatitis (HDV) (LOWR-2)

Clinical trial2023-03-03Results posted · ClinicalTrials.gov

An Open-label, Dose-ranging, Proof-of-Concept Study to Evaluate the Safety and Efficacy of Lonafarnib With and Without Ritonavir Boosting in Patients Chronically Infected With Delta Hepatitis (HDV) (LOWR-1)

Clinical trial2022-11-29Results posted · ClinicalTrials.gov

Approval: Zokinvy (EMA)

Regulatory2022-07-18EMA

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

Clinical trial2021-12-14Results posted · ClinicalTrials.gov

A Phase 2b, Open-Label, Randomized Study of the Safety, Tolerability, and Pharmacodynamic Activity of Lonafarnib With or Without Ritonavir in Patients Chronically Infected With Hepatitis Delta Virus (LOWR-5)

Clinical trial2021-02-16Withdrawn · ClinicalTrials.gov

A Phase IB Clinical Study Of The Farnesyltransferase Inhibitor SCH 66336 And Gemcitabine In Patients With Resectable Primary Liver Neoplasms

Clinical trial2020-07-31Withdrawn · ClinicalTrials.gov

Neurologic features of Hutchinson-Gilford progeria syndrome after lonafarnib treatment.

Research2013-06-28Neurology

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Lonafarnib
Aliases & brands
ZokinvyLonafarnibum
RxNorm CUI
2467553
ChEMBL ID
CHEMBL298734
UNII
IOW153004F
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2022-07-18
Latest approval
2022-07-18
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2022-07-18Approval
Approval: Zokinvy (EMA)
Indication: Zokinvy is indicated for the treatment of patients 12 months of age and older with a genetically confirmed diagnosis of Hutchinson-Gilford progeria syndrome or a… Show full indication

Zokinvy is indicated for the treatment of patients 12 months of age and older with a genetically confirmed diagnosis of Hutchinson-Gilford progeria syndrome or a processing-deficient progeroid laminopathy associated with either a heterozygous LMNA mutation with progerin-like protein accumulation or a homozygous or compound heterozygous ZMPSTE24 mutation.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

26 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
26
registered trials across all phases
LATEST COMPLETION 2025
2
Active studies
4
Late-stage (III+)
17
Completed
7
Discontinued
PHASE DISTRIBUTIONn = 26
Phase 17Phase 215Phase 34

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

Once Daily (QD) Dosing of Lonafarnib (LNF) Co-administered With Ritonavir (RTV) for Treatment of Chronic Hepatitis D Virus Infection

CompletedPH IIISoroka University Medical Centern=102025

A Phase 3, Matrix Design, Partially Double-Blind, Randomized Study of the Efficacy and Safety of 50 mg Lonafarnib/100 mg Ritonavir BID With and Without 180 mcg PEG IFN-alfa-2a for 48 Weeks Compared With PEG IFN-alfa-2a Monotherapy and Placebo Treatment in Patients Chronically Infected With Hepatitis Delta Virus Being Maintained on Anti-HBV Nucleos(t)Ide Therapy (D-LIVR)

CompletedPH IIIEiger BioPharmaceuticalsn=4072023

A Pivotal Randomized Study of Lonafarnib Versus Placebo in the Treatment of Subjects With Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMML) Who Are Platelet Transfusion Dependent With or Without Anemia

TerminatedPH IIIMerck Sharp & Dohme LLCn=472008

A Phase 3 Randomized Study of Lonafarnib in Combination With Paclitaxel and Carboplatin vs. Placebo in Combination With Paclitaxel and Carboplatin in Patients With Non-Small Cell Lung Cancer

TerminatedPH IIIMerck Sharp & Dohme LLCn=7022004

Recent completions

Trials that read out recently, adding to the completed evidence base.

Once Daily (QD) Dosing of Lonafarnib (LNF) Co-administered With Ritonavir (RTV) for Treatment of Chronic Hepatitis D Virus Infection

CompletedPH IIISoroka University Medical Centern=102025

A Phase 3, Matrix Design, Partially Double-Blind, Randomized Study of the Efficacy and Safety of 50 mg Lonafarnib/100 mg Ritonavir BID With and Without 180 mcg PEG IFN-alfa-2a for 48 Weeks Compared With PEG IFN-alfa-2a Monotherapy and Placebo Treatment in Patients Chronically Infected With Hepatitis Delta Virus Being Maintained on Anti-HBV Nucleos(t)Ide Therapy (D-LIVR)

CompletedPH IIIEiger BioPharmaceuticalsn=4072023

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

CompletedPH IINational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)n=262020

An Open-label, Dose-ranging, Proof-of-Concept Study to Evaluate the Safety and Efficacy of Lonafarnib With Ritonavir-Boosting +/- Peginterferon Alfa-2a in Patients Chronically Infected With Delta Hepatitis (HDV) (LOWR-2)

CompletedPH IIEiger BioPharmaceuticalsn=552017

Treatment of Chronic Delta Hepatitis With Lonafarnib and Ritonavir

CompletedPH IINational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)n=222017

A Phase 2, Open-Label Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Activity of a Titrating-Dose Lonafarnib/Ritonavir in Patients Chronically Infected With Hepatitis Delta Virus

CompletedPH IIEiger BioPharmaceuticalsn=152017

Research activity

1 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Journals, researchers & institutions
Top journals
  • Neurology1
Leading researchers
  • Cho YJ1
  • Giobbie-Hurder A1
  • Gordon LB1
  • Kieran MW1
  • Kleinman ME1
  • Miller DT1
  • Neuberg D1
  • Silvera VM1
Leading institutions
free-text, unnormalised
  • Boston Children's Hospital and Harvard Medical School1
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.