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European approval for paediatric cystic fibrosis drug
January 24, 2019A 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… -
EU label for Vertex's cystic fibrosis drug Orkambi expanded again to include even younger children
January 22, 2019Orkambi 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. -
Proteostasis Therapeutics receives FDA approval for Cystic Fibrosis drug
March 19, 2018The 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.
Pharma Sources Insight January 2025
