FDA Authorizes New Progeria Drug Trial, Building on Previous 1.6-Year Lifespan Extension

A new clinical trial for Progeria, an ultra-rare genetic condition causing rapid aging, has received authorization from the U.S. Food and Drug Administration (FDA). This development marks a significant step in the ongoing efforts to find more effective treatments for the devastating disease. The new trial will investigate Progerinin, an investigational drug, in combination with Zokinvy (lonafarnib), the current standard of care.

Progeria, formally known as Hutchinson-Gilford Progeria Syndrome (HGPS), accelerates the aging process dramatically. As described by The New Yorker, "Progeria is a disease of rapid, brutal aging: by the time children afflicted with it enter their teen-age years, their bodies have effectively aged eight or nine decades." Without treatment, children with Progeria typically succumb to heart disease at an average age of 14.5 years. The condition affects approximately 1 in 18 million individuals globally and is caused by a genetic mutation leading to the accumulation of a toxic protein called progerin.

The Progeria Research Foundation (PRF) has been at the forefront of treatment development since its founding in 1999, which led to the discovery of the Progeria gene in 2003. Their efforts culminated in the first FDA-approved treatment, lonafarnib (marketed as Zokinvy), in 2020. A 2018 study published in The Journal of the American Medical Association (JAMA) reported that lonafarnib alone extended survival in children with Progeria by at least 1.6 years.

The newly authorized Phase 2a clinical trial, a collaborative effort between PRG Science & Technology (PRG S&T), PRF, and Boston Children's Hospital, will assess Progerinin alongside Zokinvy. While Zokinvy works by blocking progerin's development, Progerinin appears to reduce the accumulated levels of the toxic protein. Preliminary data published in the journal Cells in April 2023 indicated that Progerinin showed a potential increase in Progeria mouse lifespan by 50%, compared to Zokinvy's 25% in similar models.

"This new trial represents another step forward in our mission to treat and cure these amazing children and young adults with Progeria," stated Audrey Gordon, Esq., Executive Director of PRF. Beyond the current trials, research continues into other promising avenues, including RNA therapy and advanced gene-editing techniques, which hold potential not only for Progeria but also for broader implications in understanding the general aging process and common heart diseases.