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 only treatment options included supportive care and therapies directed towards the complications arising from the disease.
Zokinvy, a farnesyltransferase inhibitor, is an oral medication that helps prevent the buildup of defective progerin or progerin-like protein. The effectiveness of Zokinvy for the treatment of Hutchinson-Gilford progeria syndrome was demonstrated in 62 people from two single-arm trials that were compared to matched, untreated people from a separate natural history study. Compared to untreated people, the lifespan of Hutchinson-Gilford progeria syndrome people treated with Zokinvy increased by an average of three months through the first three years of treatment and by an average of 2.5 years through the maximum follow-up time of 11 years. Zokinvy’s approval for the treatment of certain processing-deficient progeroid laminopathies that are very rare took into account similarities in the underlying genetic mechanism of disease and other available data.
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
This was reported by the U.S. Food and Drug Administration on November 20, 2020.
Contact information: U.S. Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring, Maryland 20993; (888) 463-6332; Website: www.fda.gov