First Treatment for Hutchinson-Gilford Progeria Syndrome Secures FDA Approval
The FDA has approved Eiger BioPharmaceuticals’ Zokinvy (lonafarnib) as the first treatment for children suffering from two rare genetic disorders, Hutchinson-Gilford progeria syndrome and processing-deficient progeroid laminopathies.
Both genetic disorders cause premature aging and death. Most children afflicted die before age 15 from cardiovascular disease caused by excessive build-up of defective progerin or progerin-like proteins.
Zokiny stops the build-up of these defective proteins in cells. The approval was supported by trial data from 62 enrollees showing the drug increased lifespans by 2.5 years.