PROGERIA: First-Ever Treatment (Zokinvy) for Ultra-Rare, Rapid-Aging Disease Receives U.S. FDA Approval; “Monumental Event,” Foundation Leader States; Connections with Heart Disease & Aging May Result in Much Broader Insights Applicable to Millions

On November 20, 2020, the Progeria Research Foundation (PRF) announced a historic medical milestone with the U.S. Food and Drug Administration (FDA)’s approval (see link below) of Zokinvy™ (lonafarnib), for the treatment of Progeria and for processing-deficient Progeroid Laminopathies (PL). Progeria (https://en.wikipedia.org/wiki/Progeria) is an ultra-rare, fatal, pediatric, rapid-aging, autosomal dominant disease. The PRF, a pioneer in the rare disease research foundation space, has led Zokinvy clinical trial research since 2007. Zokinvy is a farnesyltransferase inhibitor (FTI) that has shown survival benefit in children with Progeria. Data based on information from the PRF International Patient Registry and clinical trials co-coordinated by the PRF and Boston Children’s Hospital demonstrated that in patients with Progeria, Zokinvy reduced the incidence of mortality by 60% (p=0.0064) and increased average survival time by 2.5 years. Without Zokinvy treatment, children with Progeria die of heart disease at an average age of 14.5 years. Eiger BioPharmaceuticals (Eiger) began supplying Zokinvy for the Progeria clinical trials in 2015, and entered into a pioneering partnership with the PRF in 2018 with the objective of leading Zokinvy through the FDA approval process. “Today, we have achieved one important piece of PRF’s mission—the first-ever approved treatment for these beautiful children,” said Audrey Gordon, President and Executive Director of The Progeria Research Foundation. “Progeria is now one of the few rare diseases with an FDA-approved treatment.
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