Eiger BioPharmaceuticals has received minutes from a pre-investigational new drug (pre-IND) meeting with the Division of Gastroenterology and Inborn Errors Products of the U.S. Food and Drug Administration (FDA) for lonafarnib in the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria). Eiger plans to submit a new drug application (NDA) in 2019. There is no approved treatment for Progeria, an ultra-rare and fatal genetic condition characterized by accelerated aging in children.
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Eiger and the FDA engaged in a collaborative discussion regarding the analysis methodology for the survival data that was published in April 2018 Journal of the American Medical Association (JAMA) as potential support for submission of an NDA filing. This clinical study, which compared children with Progeria who received lonafarnib monotherapy with matched untreated children with Progeria, reported a primary outcome of mortality. The study found that children taking lonafarnib monotherapy (n=63) experienced a 77 percent reduction in the risk of mortality compared to a natural history, matched-control cohort of untreated children (n=63) after two years of study. Based on this meeting with the FDA, Eiger does not anticipate conducting additional clinical efficacy studies to complete the filing for registration.
Clinical studies with lonafarnib in Progeria were funded by The Progeria Research Foundation. Eiger has supplied lonafarnib for investigational use in Progeria clinical studies since 2015, and is now responsible for development, regulatory, commercialization and distribution activities for lonafarnib in Progeria.
"Eiger is preparing a regulatory submission for lonafarnib in Progeria in the U.S. with plans to file the NDA in 2019 and anticipates scientific advice from European Medicines Agency by end of 2018," said David Cory, President and CEO of Eiger. "We are committed to ensuring access to lonafarnib for all children with Progeria, and plan to launch a global expanded access program by end of year."