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lonafarnib
- Development stage
- Approved
- Lead developer
- Sentynl Therapeutics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Lonafarnib is an orally active small molecule farnesyltransferase inhibitor used to reduce the risk of death in patients with Hutchinson-Gilford progeria syndrome (HGPS) and certain processing-deficient progeroid laminopathies. It works by inhibiting farnesyltransferase, thereby preventing the farnesylation and subsequent accumulation of defective proteins such as progerin, which are implicated in premature aging disorders. Lonafarnib was originally developed for oncology indications but is now primarily indicated for rare genetic conditions causing rapid aging. The drug was discovered by Merck & Co., and further developed and brought to market by Eiger BioPharmaceuticals under the brand name Zokinvy[1][2][3][5][8].
- Brand names
- Zokinvy
- Other names
- lonafarnib
02
Targets
RSV preF (Respiratory syncytial virus prefusion F glycoprotein)FTase (Protein Farnesyltransferase)
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