Lonafarnib filed with FDA for rolling review in progeria.- Eiger Biopharma

Eiger BioPharmaceuticals announced that it has initiated submission of a New Drug Application (NDA) for Lonafarnib for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and Progeroid Laminopathies to the FDA under the Rolling Review Process. Lonafarnib, an oral farnesyltransferase inhibitor (FTI), has demonstrated extended survival in children and young adults with Progeria, an ultra-rare and fatal disease that causes premature aging in children. Without treatment, children with Progeria die of heart disease at an average age of 14.5 years.

Lonafarnib has been granted Orphan Drug Designation for Progeria and Progeroid Laminopathies by the FDA and EMA and Breakthrough Therapy Designation and Rare Pediatric Disease Designation by the FDA. The FDA agreed that the Lonafarnib submission could be a rolling NDA, allowing completed portions of an NDA to be submitted and reviewed by the FDA on an ongoing basis. Eiger plans to complete the NDA rolling submission in first quarter of 2020.