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Lonafarnib

Generic Name
Lonafarnib
Brand Names
Zokinvy
Drug Type
Small Molecule
Chemical Formula
C27H31Br2ClN4O2
CAS Number
193275-84-2
Unique Ingredient Identifier
IOW153004F

Overview

Hutchinson-Gilford progeria syndrome (HGPS) is a rare autosomal dominant disorder estimated to affect approximately one in 20 million individuals resulting in adverse symptoms associated with premature ageing: skeletal dysplasia, joint contractures, atherosclerosis, myocardial fibrosis/dysfunction, scleroderma-like cutaneous effects, lipoatrophy, alopecia, and a severe failure to thrive; HGPS is uniformly fatal. Mechanistically, HGPS is underpinned by a single heterozygous C-to-T mutation at position 1824 of the LMNA gene, which results in the accumulation of an aberrant farnesylated form of lamin A called progerin in the inner nuclear membrane. Lonafarnib is a farnesyl transferase (FTase) inhibitor (FTI), which reduces the farnesylation of numerous cellular proteins, including progerin; as progerin farnesylation is important for localization to the nuclear membrane, lonafarnib inhibits progerin accumulation and improves symptoms in HGPS patients. Merck originally developed Lonafarnib and subsequently licensed it to Eiger Biopharmaceuticals Inc., which currently markets it under the trademark ZOKINVY™. Lonafarnib was granted FDA approval on November 20, 2020, and is the first FDA-approved treatment for HGPS and other related progeroid laminopathies.

Indication

Lonafarnib is a farnesyltransferase inhibitor indicated in patients aged 12 months and older with a body surface area of at least 0.39 m to reduce the risk of mortality associated with Hutchinson-Gilford progeria syndrome (HGPS). It is also indicated in this same population for the treatment of processing-deficient progeroid laminopathies that either involve a heterozygous LMNA mutation resulting in the accumulation of a progerin-like protein or homozygous/compound heterozygous mutations in ZMPSTE24.

Associated Conditions

  • Death
  • Hutchinson-Gilford Progeria Syndrome
  • Processing-deficient Progeroid Laminopathies

Clinical Trials

Title
Posted
Study ID
Phase
Status
Sponsor
2025/01/14
Phase 2
Recruiting
2022/02/08
Phase 3
Active, not recruiting
2019/03/29
N/A
APPROVED_FOR_MARKETING
Eiger BioPharmaceuticals
2018/10/25
Phase 3
Completed
Eiger BioPharmaceuticals
2018/07/26
Phase 2
Completed
2016/11/18
Phase 2
Withdrawn
Eiger BioPharmaceuticals
2015/08/19
Phase 2
Completed
Eiger BioPharmaceuticals
2015/07/30
Phase 2
Completed
2015/04/30
Phase 2
Completed
Eiger BioPharmaceuticals
2015/04/30
Phase 2
Completed
Eiger BioPharmaceuticals

FDA Drug Approvals

Approved Product
Manufacturer
NDC Code
Route
Strength
Effective Date
Sentynl Therapeutics, Inc.
42358-475
ORAL
75 mg in 1 1
7/2/2025
Sentynl Therapeutics, Inc.
42358-450
ORAL
50 mg in 1 1
7/2/2025

EMA Drug Approvals

HSA Drug Approvals

Approved Product
Manufacturer
Approval Number
Dosage Form
Strength
Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product
Company
Approval Number
Drug Type
Dosage Form
Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product
Registration No.
Company
Licence No.
Strength
Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product
ARTG ID
Sponsor
Registration Type
Status
Registration Date
No TGA approvals found for this drug.

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