Lonafarnib: First Approval

Sohita Dhillon

Drugs. 2021 Feb;81(2):283-289. doi: 10.1007/s40265-020-01464-z.

 

This article summarizes the milestones in the development of Lonafarnib leading to this first approval. In Hutchinson-Gilford Progeria Syndrome (HGPS), lonafarnib inhibits farnesyltransferase to prevent farnesylation and subsequent accumulation of progerin and progerin-like proteins in the nucleus and cellular cytoskeleton. In November 2020, lonafarnib received its first approval in the USA to reduce the risk of mortality in HGPS and for the treatment of processing-deficient progeroid laminopathies in patients ≥ 12 months of age with a body surface area (BSA) of ≥ 0.39 m2. Lonafarnib is under regulatory review in the European Union.

 

medline link: https://pubmed.ncbi.nlm.nih.gov/33590450/