Indication1
Zokinvy is indicated for the treatment of patients ≥12 months of age with a genetically confirmed diagnosis of Hutchinson-Gilford progeria syndrome or a processing-deficient progeroid laminopathy associated with either a heterozygous LMNA mutation with progerin-like protein accumulation or a homozygous or compound heterozygous ZMPSTE24 mutation.
Mechanism of action1
Zokinvy is a disease-modifying agent that prevents farnesylation, thereby reducing the accumulation of aberrant progerin and progerin-like proteins in the cell’s inner nuclear membrane.
The reduced accumulation of progerin and progerin-like proteins maintains cell integrity and normal cell function.
By preventing farnesylation, Zokinvy targets a key step in the pathophysiology of HGPS and PL.
A cell nucleus* with normal Lamin A, a LMNA protein, critical to many cellular functions.
LMNA mutations lead to farnesylated aberrant progerin and progerin-like proteins which will incorporate into the inner nuclear membranes of cells.
Zokinvy inhibits farnesylation, preventing accumulation of progerin or progerin-like proteins in the inner nuclear membrane.
Did you know?
Zokinvy prevents the accumulation of progerin and progerin-like proteins, maintaining cell integrity and normal function.