For immediate release:

Today, the United States Food and Drug Administration approved Zokinvy (lonafarnib) capsules to reduce the risk of death from Hutchinson-Gilford progeria syndrome and for the treatment of certain treatment-deficient progeroid laminopathies in patients aged one year and over. Zokinvy is not approved for use in patients with other progeroid syndromes or laminopathies.

“Hutchinson-Gilford progeria syndrome and progeroid laminopathies are rare genetic diseases that cause premature aging and death and have a debilitating effect on people’s lives,” said Hylton V. Joffe, MD, MMSc, director of the Office of Rare Diseases, Pediatrics, Urology and Reproductive Medicine at the FDA’s Center for Drug Evaluation and Research. “With today’s approval, Zokinvy is the first drug approved by the FDA for these devastating diseases. The FDA will continue to work with stakeholders to advance the development of new therapies that are additional, effective and safe for these patients.

Patients with Hutchinson-Gilford progeria syndrome and progeroid laminopathies have accelerated cardiovascular disease due to the buildup of defective progerin or progerin-like protein in cells. Most patients die before the age of 15 from heart failure, heart attack, or stroke. Prior to today’s approval, the only treatment options included supportive care and therapy focused on complications arising from the disease.

Zokinvy, a farnesyltransferase inhibitor, is an oral medicine that helps prevent the buildup of defective progerin or progerin-like protein. The efficacy of Zokinvy for the treatment of Hutchinson-Gilford progeria syndrome was demonstrated in 62 patients from two single-arm trials that were compared to untreated matched patients from a separate natural history study. Compared with untreated patients, the lifespan of patients with Hutchinson-Gilford progeria syndrome treated with Zokinvy increased by an average of three months during the first three years of treatment and by an average of 2.5 years during the period. maximum follow-up period of 11 years. The approval of Zokinvy for the treatment of certain treatment-deficient progeroid laminopathies which are very rare took into account the similarities in the underlying genetic mechanism of the disease and other available data.

The most common side effects included nausea, vomiting, diarrhea, infections, decreased appetite, and fatigue.

Zokinvy is contraindicated when co-administered with strong or moderate inhibitors and inducers of CYP3A, as well as with midazolam and certain cholesterol lowering drugs. Some patients treated with Zokinvy have developed abnormal laboratory tests, such as changes in blood sodium and potassium levels, a decrease in the number of white blood cells, and an increase in liver blood tests. Routine blood tests should be done periodically. Ocular toxicity has been observed in animals, so eye exams are recommended periodically and for new visual changes.

The FDA has granted this application priority review designation. Zokinvy received the Orphan Drug Designation, which provides incentives to aid and encourage drug development for rare diseases, and the Breakthrough Therapy designation. In addition, the manufacturer has received a Rare Pediatric Disease Priority Review Voucher. The FDA’s Rare Pediatric Disease Priority Review Voucher program aims to encourage the development of new drugs and biologics to prevent and treat rare diseases in children. FDA has granted Zokinvy approval to Eiger BioPharmaceuticals, Inc.

The FDA, an agency of the US Department of Health and Human Services, protects public health by ensuring the safety, efficacy, and safety of human and veterinary drugs, vaccines and other biologicals for human use, and medical devices . The agency is also responsible for the safety and security of the food supply, cosmetics, dietary supplements, products that emit electronic radiation, and the regulation of tobacco products.

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