EMA has recommended granting a marketing authorisation in the European Union (EU) under exceptional circumstances for Zokinvy (lonafarnib), a treatment for patients with a genetically confirmed diagnosis of Hutchinson-Gilford Progeria Syndrome or progeroid laminopathies. Zokinvy is indicated for use in children one year of age and older.
Hutchinson-Gilford Progeria Syndrome is an ultra-rare multi-systemic ‘premature aging’ disease. It can be diagnosed by genetic testing but also based on a consistent pattern of clinical features, including limited growth, characteristic facial features such as a receding chin and a narrow, pointed nose, loss of hair and body fat, crowded teeth, small and fragile bones, and stiffness of joints. Most children die in their early teens due to severe cardiovascular complications at an average age of 14.5 years…
