Hutchinson-Gilford Progeria Medication

Updated: Nov 24, 2020
  • Author: Kara N Shah, MD, PhD; Chief Editor: Dirk M Elston, MD  more...
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Medication Summary

Lonafarnib (Zokinvy) is a farnesyltransferase inhibitor (FTI). It was approved by the FDA in November 2020 for Hutchinson-Gilford Progeria syndrome (HGPS) and processing-deficient progeroid laminopathies. A multinational, observational study (n = 258) showed treatment with lonafarnib monotherapy was associated with a lower mortality rate compared with no treatment after 2.2 years of follow-up. [34]


Farnesyltransferase Inhibitors

Class Summary

Farnesyltransferase is an enzyme involved in modification of proteins through a process called prenylation. Mutation in the LMNA gene causes over-production of progerin, a farnesylated-aberrant protein; persistent farnesylation causes progerin accumulation in the inner nuclear membrane and is, at least partly, responsible for HGPS. Accumulation of the defective lamin A protein makes the nucleus unstable, leading to the process of premature aging in children with progeria.

Lonafarnib (Zokinvy)

Lonafarnib is a farnesyltransferase inhibitor indicated to reduce the risk of mortality in Hutchinson-Gilford progeria syndrome (HGPS) in patients aged 12 months and older with body surface area greater than 0.39 m2. It is also indicated for processing-deficient progeroid laminopathies with heterozygous LMNA mutation with progerinlike protein accumulation or homozygous or compound heterozygous ZMPSTE24 mutations.