FDA Approves First Treatment For Hutchinson-Gilford Progeria Syndrome & Some Progeroid Laminopathies

The U.S. Food and Drug Administration approved Zokinvy (lonafarnib) capsules to reduce the risk of death due to Hutchinson-Gilford progeria syndrome and for the treatment of certain processing-deficient progeroid laminopathies in people one year of age and older. Zokinvy is not approved for use in people with other progeroid syndromes or laminopathies.

People with Hutchinson-Gilford progeria syndrome and progeroid laminopathies experience accelerated cardiovascular disease from the buildup of defective progerin or progerin-like protein in cells. Most people die before the age of 15 years from heart failure, heart attack, or stroke. Before the approval, the . . .