The U.S. Food and Drug Administration (FDA) has approved Zokinvy (lonafarnib) to treat progeria, a rare disease often known as the advanced aging disease.

More specifically, the FDA has approved 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 patients one year of age and older. The drug is not approved for use in patients with other progeroid syndromes or laminopathies.

Hylton V. Joffe, MD, MMSc, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research stated, “With today’s approval, Zokinvy is the first FDA-approved medication for these devastating diseases. The FDA will continue to work with stakeholders to advance the development of additional new, effective and safe therapies for these patients.”

Children with progeria experience accelerated cardiovascular disease from the buildup of defective progerin or progerin-like protein in cells. Most persons with progeria die from heart failure, heart attack or stroke in their teen years.

Lonafarnib is a farnesyltransferase inhibitor that helps  to prevent the buildup of defective progerin or progerin-like protein.  The drug was initially developed by the Progeria Research Foundation, a non-profit organization founded by Drs Leslie Gordon and Scott Berns, the parents of Sam Berns.

Sam died in 2014 at the age of 17 due to complications from Progeria.  His 2013 TED talk about leading a happy life has been seen by over 40 million viewers.

In 2018, Eiger Pharmaceuticals partnered with the Progeria Research Foundation to develop the drug for FDA approval, and its subsequent supply and distribution. This is the first FDA approved drug for the biopharmaceutical company.

In a news release, Audrey Gordon, President and Executive Director of The Progeria Research Foundation stressed the team effort that was involved in getting the drug approved. Ms. Gordon states, “Progeria is now one of the few rare diseases with an FDA-approved treatment. This momentous event is here, thanks in large part to PRF’s key partnerships, including the expert Progeria research teams from Boston Children’s Hospital, Hasbro Children’s Hospital, Brigham and Women’s Hospital, Brown University, Boston University, and the National Institutes of Health. Pharmaceutical partners that supplied lonafarnib free of charge to PRF supported clinical trials were crucial as well, including Schering-Plough, Merck, and Eiger.”

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