logo
Menu

This search function provides links to outputs produced by NIHR Innovation Observatory. These are briefing notes or reports on new or repurposed technologies. This search will not return all technologies currently in development as these outputs are produced as required for our stakeholders.

Innovation Observatory > Reports > Drugs > Lonafarnib for Hutchinson-Gilford Progeria syndrome and progeroid laminopathies

< Back

Lonafarnib for Hutchinson-Gilford Progeria syndrome and progeroid laminopathies

Drugs

September 2020


Lonafarnib is in clinical development for the treatment of Hutchinson-Gilford Progeria syndrome (HGPS) and other progeroid laminopathies (PL). Progeroid laminopathies are characterised by the premature appearance of certain signs of physiological ageing in a subset of tissues, and the most prevalent of these rare diseases is HGPS. HGPS is caused by genetic abnormalities in a gene, which produces a protein called lamin A that helps to keep cells of the body strong and stable. HGPS is a severe and life-threatening condition which leads to premature death, primarily due to cardiovascular morbidities, such as heart attacks and strokes. The mean life expectancy in progeria is 14.5 years of age.

Lonafarnib, administered orally, works by preventing the formation of abnormal lamin A. An abnormal quantity of lamin A causes overproduction of a defective protein called progerin, which is believed to cause premature ageing in children. If licensed, lonafarnib will provide the first and only approved treatment option for patients with HGPS and PL.

Innovation Observatory Voice 0

Leave a Reply

Your email address will not be published. Required fields are marked *

Post Comment

Download Full Article



 

Connect to the Innovation Observatory

Twitter

Get Alerts