NDAORALCAPSULEPriority Review
Approved
Nov 2020
Lifecycle
Peak
Competitive Pressure
8/100
Clinical Trials
7
Data completeness
4/8 — Partial
Priority Review
ZOKINVY (lonafarnib) by Sentynl Therapeutics is farnesyltransferase inhibitors [moa]. First approved in 2020.
Drug data last refreshed 2h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
ZOKINVY (lonafarnib) is an oral farnesyltransferase inhibitor approved in November 2020 for treating progeria and related laminopathies. It works by blocking farnesylation of mutant lamin A protein, reducing the accumulation of toxic protein that drives premature aging in these rare genetic diseases. The drug represents a targeted approach to a previously untreatable ultra-rare patient population.
Peak3.1 years to LOE
Ultra-rare indication with limited commercial scale; brand team likely focused on patient identification and long-term outcomes tracking rather than high-volume sales growth.
Mechanism of Action
Farnesyltransferase Inhibitors
Pharmacologic Class:
Farnesyltransferase Inhibitor
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (7)
Predicting Response and Toxicity in Patients Receiving Lonafarnib for Breast Cancer
Started Aug 2009
27 enrolled
Breast Cancer
Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria
Started Aug 2009
85 enrolled
Progeria
A Study to Evaluate the Safety, Tolerability, and Activity of Lonafarnib and Docetaxel (Study P04467AM1)(TERMINATED)
Started Jun 2007
5 enrolled
Prostate CancerBreast CancerOvarian Cancer+2 more
Study of Lonafarnib Versus Placebo in Subjects With Either Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMML) (Study P02978AM3)(TERMINATED)
Started May 2005
47 enrolled
Myelodysplastic SyndromesLeukemia, Myelomonocytic, ChronicMyelodysplasia+1 more
Anastrozole Plus Lonafarnib (SCH 66336) or Plus Placebo for the Treatment of Advanced Breast Cancer (P03480)
Started Dec 2003
110 enrolled
Breast Cancer
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.