ZOKINVY (lonafarnib)
Zokinvy (lonafarnib) is a farnesyltransferase inhibitor indicated for patients 12 months of age and older with a body surface area (BSA) of $0.39 \text{ m}^2$ and above. It is indicated to reduce the risk of mortality in patients with Hutchinson-Gilford Progeria Syndrome (HGPS) and for the treatment of processing-deficient Progeroid Laminopathies characterized by either heterozygous *LMNA* mutations with progerin-like protein accumulation or homozygous/compound heterozygous *ZMPSTE24* mutations. **Limitations of Use:** Zokinvy is not indicated for other Progeroid Syndromes or processing-proficient Progeroid Laminopathies, as the mechanism of action is not expected to be effective in those populations.
How ZOKINVY Works
Lonafarnib inhibits the enzyme farnesyltransferase to prevent the farnesylation and subsequent accumulation of progerin and progerin-like proteins in the inner nuclear membrane. By blocking this post-translational modification, the drug mitigates the permanent anchoring of these toxic proteins to the nuclear envelope.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2020-11-20
- Patent Cliff
- 2029
- Routes
- ORAL
- Dosage Forms
- CAPSULE
ZOKINVY Approval History
What ZOKINVY Treats
2 indicationsZOKINVY is approved for 2 conditions since its original approval in 2020. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Hutchinson-Gilford Progeria Syndrome
- Progeroid Laminopathies
Clinical Trial Registry
11 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT05229991 LOWR6 | SOR-0527-20-CTIL SCRC20042 | Ph 3 | completed | Once Daily Dosing of Lonafarnib Co-administered With Ritonavir for Treatment of Chronic Hepatitis D Virus Infection |
| NCT06775041 | PRG-PRO-005 | Ph 2 | active not recruiting | Study to Determine Optimal Dose and Evaluate Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS) |
| NCT02527707 LOWR-4 results posted | EIG-LNF-002 | Ph 2 | completed | Titrating-Dose of Lonafarnib in Combination With Ritonavir |
| NCT03719313 D-LIVR | EIG-LNF-011 | Ph 3 | completed | Study of the Efficacy and Safety of Lonafarnib / Ritonavir With and Without Pegylated Interferon -Alfa-2a |
| NCT02430194 LOWR-2 results posted | EIG-300-Amendment 3 | Ph 2 | completed | Lonafarnib Boosted With Ritonavir With and Without Peginterferon Alfa-2a (PEG IFN-a) in HDV (LOWR-2) |
| NCT02430181 LOWR-1 results posted | EIG-300 Application #1128309 | Ph 2 | completed | Lonafarnib With and Without Ritonavir in HDV (LOWR-1) |
| NCT03600714 results posted | 180123 18-DK-0123 | Ph 2 | completed | Treatment of Chronic Delta Hepatitis With Lonafarnib, Ritonavir and Lambda Interferon |
| NCT00879034 results posted | 09-02-0074 P06087 | Ph 2 | completed | A Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria |
| NCT02511431 results posted | 150170 15-DK-0170 | Ph 2 | completed | Treatment of Chronic Delta Hepatitis With Lonafarnib and Ritonavir |
| NCT01495585 results posted | 120046 12-DK-0046 | Ph 2 | completed | Lonafarnib for Chronic Hepatitis D |
| NCT00773474 results posted | HOG BRE07-126 | Ph 2 | terminated | Lonafarnib in Metastatic Breast Cancer |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
ZOKINVY FDA Label Details
Indications & Usage
FDA Label (PDF)ZOKINVY is indicated for the treatment of Hutchinson-Gilford Progeria Syndrome; Progeroid Laminopathies.
ZOKINVY Patents & Exclusivity
Patents (1 active)
Exclusivity
Pro Intelligence Preview
Deep insights for ZOKINVY
Revenue Insights
- • Quarterly revenue tracking
- • Historical trend analysis
Patent Timeline
- • Cliff: 2029
- • 4 active patents
Trial Analysis
- • 11 total trials
- • Stage: Declining
Competitive Landscape
- • Competitor tracking
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment