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Related Experiment Video

Updated: Nov 17, 2025

Laparoscopic Anatomical Resection of the Right Anterior Lobe Based on the Laennec Capsule Technique

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Lonafarnib: First Approval.

Sohita Dhillon¹

¹Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. dru@adis.com.

|February 16, 2021

Summary

Lonafarnib is an oral farnesyltransferase inhibitor approved for Hutchinson-Gilford Progeria Syndrome and related conditions. It prevents harmful protein accumulation, offering a new treatment for these rare diseases.

Area of Science:

Pharmacology
Genetics
Virology

Background:

Lonafarnib is a farnesyltransferase inhibitor originally developed for oncology.
It targets the farnesylation process implicated in progeria and hepatitis D virus (HDV) infections.

Purpose of the Study:

To summarize the development milestones of lonafarnib.
To highlight its first regulatory approval for progeria and related conditions.

Main Methods:

Review of lonafarnib's drug discovery and development history.
Analysis of clinical data supporting its efficacy and safety in progeroid laminopathies.

Main Results:

Lonafarnib received US FDA approval in November 2020 for Hutchinson-Gilford Progeria Syndrome (HGPS) and processing-deficient progeroid laminopathies.

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The drug inhibits farnesyltransferase, preventing progerin accumulation in HGPS.

Conclusions:

Lonafarnib represents a significant therapeutic advancement for patients with progeria and related laminopathies.
Ongoing clinical development for HDV infections shows promise for broader applications.