Lonafarnib

Lonafarnib
Clinical data
Trade names	Zokinvy
Other names	SCH 66336
License data	US DailyMed: Lonafarnib;
Routes of; administration	By mouth
ATC code	A16AX20 (WHO) ;
Legal status
Legal status	US: ℞-only; EU: Rx-only;
Identifiers
	IUPAC name 4-(2-{4-[(11R)-3,10-dibromo-8-chloro-6,11-dihydro-5H-benzo[5,6]cyclohepta[1,2-b]pyridin-11-yl]piperidin-1-yl}-2-oxoethyl)piperidine-1-carboxamide;
CAS Number	193275-84-2 ;
PubChem CID	148195;
IUPHAR/BPS	8024;
DrugBank	DB06448;
ChemSpider	130645 ;
UNII	IOW153004F;
KEGG	D04768 ;
ChEBI	CHEBI:47097 ;
ChEMBL	ChEMBL298734 ;
PDB ligand	336 (PDBe, RCSB PDB);
CompTox Dashboard (EPA)	DTXSID90172927 ;
ECHA InfoCard	100.204.509
Chemical and physical data
Formula	C27H31Br2ClN4O2
Molar mass	638.83 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES C=12CCC=3C=C(C=C(C3[C@H](C1N=CC(=C2)Br)C4CCN(CC4)C(=O)CC5CCN(CC5)C(N)=O)Br)Cl;
	InChI InChI=1S/C27H31Br2ClN4O2/c28-20-12-19-2-1-18-13-21(30)14-22(29)24(18)25(26(19)32-15-20)17-5-9-33(10-6-17)23(35)11-16-3-7-34(8-4-16)27(31)36/h12-17,25H,1-11H2,(H2,31,36)/t25-/m1/s1 ; Key:DHMTURDWPRKSOA-RUZDIDTESA-N ;

Lonafarnib, sold under the brand name Zokinvy, is a medication used to reduce the risk of death due to Hutchinson-Gilford progeria syndrome and for the treatment of certain processing-deficient progeroid laminopathies in people one year of age and older.^[3]^[4] It is under trial for its use as combination treatment for Hepatitis D Virus.^[5]

The most common side effects included nausea, vomiting, headache, diarrhea, infection, decreased appetite and fatigue.^[3]

Lonafarnib was approved for medical use in the United States in November 2020,^[3]^[6] and in the European Union in July 2022.^[2] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.^[7]

^ "Lonafarnib capsule". DailyMed. Archived from the original on 22 January 2021. Retrieved 17 January 2021.
^ ^a ^b "Zokinvy EPAR". European Medicines Agency. 17 May 2022. Archived from the original on 2 August 2022. Retrieved 1 August 2022. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
^ ^a ^b ^c "FDA Approves First Treatment for Hutchinson-Gilford Progeria Syndrome and Some Progeroid Laminopathies". U.S. Food and Drug Administration (FDA) (Press release). 20 November 2020. Archived from the original on 28 November 2020. Retrieved 20 November 2020. This article incorporates text from this source, which is in the public domain.
^ "Drug Trials Snapshots: Zokinvy". U.S. Food and Drug Administration. 20 November 2020. Archived from the original on 12 December 2020. Retrieved 11 December 2020. This article incorporates text from this source, which is in the public domain.
^ Yurdaydin C, Keskin O, Yurdcu E, Çalişkan A, Önem S, Karakaya F, et al. (June 2022). "A phase 2 dose-finding study of lonafarnib and ritonavir with or without interferon alpha for chronic delta hepatitis". Hepatology. 75 (6): 1551–1565. doi:10.1002/hep.32259. PMID 34860418.
^ "Drug Approval Package: Zokinvy". U.S. Food and Drug Administration (FDA). 18 December 2020. Archived from the original on 22 January 2021. Retrieved 17 January 2021.
^ "New Drug Therapy Approvals 2020". U.S. Food and Drug Administration (FDA). 31 December 2020. Archived from the original on 18 January 2021. Retrieved 17 January 2021. This article incorporates text from this source, which is in the public domain.

[Zokinvy_FDA_label-1] "Lonafarnib capsule". DailyMed. Archived from the original on 22 January 2021. Retrieved 17 January 2021.

[Zokinvy_EPAR-2] "Zokinvy EPAR". European Medicines Agency. 17 May 2022. Archived from the original on 2 August 2022. Retrieved 1 August 2022. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.

[FDA_PR_20201120-3] "FDA Approves First Treatment for Hutchinson-Gilford Progeria Syndrome and Some Progeroid Laminopathies". U.S. Food and Drug Administration (FDA) (Press release). 20 November 2020. Archived from the original on 28 November 2020. Retrieved 20 November 2020. This article incorporates text from this source, which is in the public domain.

[FDA_snapshot-4] "Drug Trials Snapshots: Zokinvy". U.S. Food and Drug Administration. 20 November 2020. Archived from the original on 12 December 2020. Retrieved 11 December 2020. This article incorporates text from this source, which is in the public domain.

[Yurdaydin_2022-5] Yurdaydin C, Keskin O, Yurdcu E, Çalişkan A, Önem S, Karakaya F, et al. (June 2022). "A phase 2 dose-finding study of lonafarnib and ritonavir with or without interferon alpha for chronic delta hepatitis". Hepatology. 75 (6): 1551–1565. doi:10.1002/hep.32259. PMID 34860418.

[6] "Drug Approval Package: Zokinvy". U.S. Food and Drug Administration (FDA). 18 December 2020. Archived from the original on 22 January 2021. Retrieved 17 January 2021.

[7] "New Drug Therapy Approvals 2020". U.S. Food and Drug Administration (FDA). 31 December 2020. Archived from the original on 18 January 2021. Retrieved 17 January 2021. This article incorporates text from this source, which is in the public domain.

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