Eiger BioPharmaceuticals Inc. started a rolling submission of a new drug application to the U.S. Food and Drug Administration for the approval of lonafarnib to treat rare genetic disorders that cause accelerated aging.
The Palo Alto, Calif.-based biopharmaceutical company is developing lonafarnib to treat Hutchinson-Gilford progeria syndrome, or progeria, and progeroid laminopathies.
Progeria causes accelerated aging in children and without treatment, children die of heart disease at an average age of 14.5. Progeroid laminopathies are genetic conditions of premature aging caused by certain gene mutations. Collectively, worldwide prevalence of Progeroid Laminopathies is likely greater than progeria.
Eiger said lonafarnib showed extended survival in children and young adults with progeria. Data from phase 1/2 and phase 2 studies showed the drug was associated with a reduction in risk of mortality by 88%.
The company plans to complete the rolling submission of the drug in the first quarter of 2020.
The rolling review program of the U.S. regulator allows companies to submit individual portions of the application instead of waiting to complete all sections for one-time full submission.
Lonafarnib was previously assigned as a breakthrough therapy by the FDA and granted orphan drug designations by the FDA and the European Medicines Agency. In addition, the drug also received breakthrough therapy status from the FDA and a priority medicine designation from the EMA to treat patients with hepatitis delta virus infection.
Eiger licensed lonafarnib from Merck & Co. Inc. to lead the regulatory development, commercialization and distribution of the medicine.