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FDA approves Zokinvy to reduce death from Hutchinson-Gilford progeria syndrome


The US Food and Drug Administation (FDA) has approved Zokinvy to reduce risk of death due to Hutchinson-Gilford progeria syndrome and to treat some progeroid laminopathies.

The US Food and Drug Administration (FDA) has approved Zokinvy (lonafarnib) capsules from Eiger BioPharmaceuticals, Inc.1 The drug, which is a farnesyltransferase inhibitor, was approved to reduce the risk of death because of Hutchinson-Gilford progeria syndrome as well as treating certain processing-deficient progeroid laminopathies. It has been approved for use in children aged 1 year and older, but not approved for other progeroid syndromes or laminopathies.

The efficacy of Zokinvy was assessed in 62 patients who had been enrolled in 2 single-arm trials. In the studies, when compared to patients who were not treated, patients who had been administered Zokinvy had a lifespan that increased an average of 3 months during the first 3 years of treatment and, through the maximum follow-up period of 11 years, the average increase in lifespan was 2.5 years. Common side effects seen in the participants included nausea, vomiting, infection, diarrhea, fatigue, and decreased appetite.

The drug is contraindicated for co-administration with moderate of strong CYP3A inhibitors, certain cholesterol-lowering drugs, and midazolam. Additionally, some patients who were administered Zokinvy developed laboratory test abnormalities, like lowered white blood cell counts, increased liver blood tests, changes in blood sodium levels, and changes in blood potassium levels. Due to this possibility, it is recommended that routine blood laboratory testing occur on a periodic basis. Additionally, because of eye toxicity seen in animals, eye exams should be performed periodically or when visual changes are noted by patients.

In the press release for the drug’s approval, 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 said “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. 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.”


1. US Food and Drug Administration. FDA approves first treatment for Hutchinson-Gilford progeria syndrome and some progeroid laminopathies. Published November 20, 2020. Accessed November 23, 2020. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-hutchinson-gilford-progeria-syndrome-and-some-progeroid-laminopathies

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