Drug · Small Molecule

Lonafarnib

CI-DRUG-00000348Explore in graph →NCIt C1829 CHEMBL298734 CIViC Approved

Regulatory

Approvals (1)

Each record names the authority, jurisdiction, indication text and status. A drug approved in one jurisdiction for one indication is not 'approved' in general.

openfdaRegulatory1 approval record · authority, jurisdiction and indication text as published.

Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
US FDA	—	ZOKINVY is indicated in patients 12 months of age and older with a body surface area (BSA) of 0.39 m 2 and above: To reduce the risk of mortality in Hutchinson-Gilford Progeria Syndrome (HGPS) For the treatment of processing-deficient Progeroid Laminopathies with either: Heterozygous LMNA mutation with progerin-like protein accumulation Homozygous or compound heterozygous ZMPSTE24 mutations ZOKINVY is a farnesyltransferase inhibitor indicated in patients 12 months of age and older with a body surface area of 0.39 m 2 and above: ( 1 ) To reduce risk of mortality in Hutchinson-Gilford Progeria Syndrome. For treatment of processing-deficient Progeroid Laminopathies with either: Heterozygous LMNA mutation with progerin-like protein accumulation. Homozygous or compound heterozygous ZMPSTE24 mutations. Limitations of Use Not indicated for other Progeroid Syndromes or processing-proficient Progeroid Laminopathies. Based upon its mechanism of action, ZOKINVY would not be expected to be effective in these populations. ( 1 ) Limitations of Use ZOKINVY is not indicated for other Progeroid Syndromes or processing-proficient Progeroid Laminopathies. Based upon its mechanism of action, ZOKINVY would not be expected to be effective in these populations.	approved	Nov 20, 2020	openfda

Data updated 2 days agoSource updated unknown

Derived

Development pipeline

Most advanced stage across all cancers, then per top-level cancer reached through trial conditions or approval indications. Approval in any ingested jurisdiction outranks trial phase; counts are interventional studies.

Scope	Stage	Max phase	Active	Phase 3	Trials	Approvals	Jurisdictions	First approval	First trial
All cancers	Approved	Phase 3	1	2	18	1	US	Nov 20, 2020	1998-10
Leukemia	Phase3	Phase 3	0	1	3	0	—	—	Apr 30, 2001
Malignant Lung Neoplasm	Phase3	Phase 3	0	1	2	0	—	—	2002-12
Malignant Bladder Neoplasm	Phase2	Phase 2	0	0	1	0	—	—

Curated evidence

Clinical evidence (1)

CIViC items in which this therapy appears, grouped by cancer context, then molecular profile. 50 items per page.

civicCuratedShowing 1–1 of 1 evidence items · levels, directions and significance as curated at the source; each row links to its CIViC record.

Therapy	Cancer	Type	Level	Direction · significance	Rating (1–5)	Status	Evidence	Source
FNTB RS116238661
Lonafarnib	Malignant Ovarian Neoplasm

Clinical trials

Trials with this intervention (18)

Most recently updated first, 50 per page.

clinicaltrialsPublishedShowing 1–18 of 18 studies · status and phase as posted by the registrant; each row links to the study record.

NCT	Title	Status	Phase	Enrollment (n)	Sponsor	Countries	Last update	Source
NCT00102648	Lonafarnib and Temozolomide in Treating Patients with Glioblastoma Multiforme That is Recurrent or Did Not Respond to Previous Treatment with Temozolomide	active not recruiting	Phase 1	34	M.D. Anderson Cancer CenterOTHER	1	Oct 17, 2024	clinicaltrials
NCT00020774	SCH 66336 With or Without Gemcitabine Followed by Surgery Compared With Surgery Alone in Treating Patients With Primary Liver Cancer

Molecule

Record

Kind: Small Molecule
Mechanism: Protein farnesyltransferase inhibitor
Targets: 2 genes
UNII: IOW153004F
Aliases: 4-[2-[4-[(11R)-3,10-Dibromo-8-chloro-6,11-dihydro-5H-benzo[5,6]cyclohepta[1,2-b]pyridin-11-yl]-1-piperidinyl]-2-oxoethyl]-1-piperidinecarboxamide, 4-[2-[4-(3,10-Dibromo-8-chloro-6,11-dihydro-5H-benzo[5,6]cyclohepta[1,2-b]pyridin-11-yl)-1-piperidinyl]-2-oxoethyl]-1-piperidinecarboxamide, Lonafarnib, Sarasar, SCH 66336, SCH66336, ZOKINVY

Data updated 11 hours agoSource updated unknown

Identifiers

Codes (0)

Upstream identifiers kept as first-class codes; each links to its registry where a stable public URL exists.

Data not yet available

No cross-reference code recorded yet beyond the record identifiers above.

Regulatory status is jurisdiction-specific and time-bound. Nothing on this page is a treatment recommendation.