Drug intelligence / Profile preview

placebo + lonafarnib

Development stage
Unknown
Lead developer
Eiger BioPharmaceuticals
Modality
Small Molecules
Administration
Oral
01

Overview

This is a combination used in clinical trials where one group receives the active drug lonafarnib and another group receives a placebo, allowing for comparison of efficacy and safety. Lonafarnib is a farnesyltransferase inhibitor used to reduce mortality in patients with Hutchinson-Gilford progeria syndrome (HGPS) and certain processing-deficient progeroid laminopathies. It works by inhibiting the farnesylation of proteins such as progerin, preventing their accumulation and subsequent cellular damage[1][2][5]. The term "placebo + lonafarnib" does not refer to a single product but rather to two separate arms within a clinical trial or study design.

Other names
placebo and lonafarniblonafarnib plus placebo
02

Targets

FTase (Protein Farnesyltransferase)

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