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2016/11/18 | Phase 2 | Withdrawn | Eiger BioPharmaceuticals | ||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 |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
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Authorised | 7/18/2022 |
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
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No HSA approvals found for this drug. |
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
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No NMPA approvals found for this drug. |
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
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No PPB approvals found for this drug. |
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
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No TGA approvals found for this drug. |
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