Eiger Announces Positive Meeting with FDA on Progeria
September 5, 2018
Rare Daily Staff
Eiger BioPharmaceuticals said it plans to submit an application to the U.S. Food and Drug Administration for marketing approval of its experimental drug lonafarnib as a treatment of progeria, an ultra-rare and fatal genetic condition characterized by accelerated aging in children.
Progeria, also known as Hutchinson-Gilford progeria syndrome, is caused by a mutation in the lamin A gene, which results in the production of an aberrant protein, progerin. Researchers believe that defective protein makes the nucleus of the cell unstable. That cellular instability leads to the process of premature aging in progeria.
The manifestations of progeria include growth failure, loss of body fat and hair, aged-looking skin, stiff joints, hip dislocation, cardiovascular disease, and stroke. Children with the condition live on average to a little more than 14 and die from heart disease typically associated with old age. There are no approved drugs to treat the condition. An estimated 350 children worldwide have the condition.
Lonafarnib inhibits an enzyme that is involved in the biological pathway that leads to the production of the mutated protein that drives the condition. The FDA granted Orphan Drug designation to Lonafarnib for progeria.
Eiger said it discussed with the FDA the analysis methodology for the survival data that was published in April 2018 Journal of the American Medical Association as potential support for submission of an NDA filing. The clinical study, which compared children with progeria who received lonafarnib monotherapy with matched untreated children with Progeria, found that children taking lonafarnib monotherapy experienced a 77 percent reduction in the risk of mortality compared to a natural history, matched-control cohort of untreated children 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.
The Progeria Research Foundation funded the clinical studies with lonafarnib in progeria. 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 United States and plans to file the NDA in 2019 and anticipates scientific advice from European Medicines Agency by end of 2018.
“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,” David Cory, president and CEO of Eiger.
September 5, 2018
Photo: David Cory, president and CEO of Eiger BioPharmaceuticals
Sign up for updates straight to your inbox.