Eiger BioPharmaceuticals (NASDAQ:EIGR) initiated a rolling NDA submission to the FDA for lonafarnib for the treatment of Hutchinson-Gilford progeria syndrome and progeroid laminopathies.
Progeria is an extremely rare and fatal disease caused by a mutation in the gene encoding for lamin A protein, which is a key structural component of cellular nuclei.
Progeria causes premature aging in children, causing patients to die of heart disease at an average age of 14.5 years. The disease is estimated to affect some 400 children worldwide.
Lonafarnib is an oral farnesyltransferase inhibitor that has demonstrated extended survival in children and young adults with progeria. Eiger plans to complete the NDA rolling submission in first quarter of 2020.
“Our discussions with FDA have been constructive and collaborative at each step, and agency agreement to accept our NDA on a rolling basis marks an important milestone to begin the regulatory review process for lonafarnib in progeria and progeroid laminopathies,” David Cory, Eiger’s president and CEO, said in a statement.
