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A paediatric treatment for 2 to 5 year olds has been approved by the European Commission to treat children with the most common form of cystic fibrosis…
Orkambi was initially approved in Europe in 2015 for patients with CF ages 12 years and older who have two copies of the F508del mutation and was later expanded to include children ages six to 11 years.
The FDA Office of Orphan Products Development grants Orphan Drug Designation to novel drugs or biologics that are intended for the treatment of rare diseases or conditions affecting fewer than 200,000 patients in the United States.