Medicinal product | Active substance | Date of authorisation | Marketing authorisation holder | Approved indication | Orphan medicinal product# |
Cayston | Aztreonam lysine | Sept 21, 2009 | Gilead Sciences International Ltd, Cambridge, UK | Suppressive therapy of chronic pulmonary infections due to P. aeruginosa in patients with CF aged ≥6 years | Yes |
TOBI Podhaler | Tobramycin | July 20, 2011 | Novartis Europharm Ltd, Camberley, UK | Suppressive therapy of chronic pulmonary infection due to P. aeruginosa in adults and children with CF aged ≥6 years | Yes |
Colobreathe | Colistimethate sodium | Feb 13, 2012 | Teva BV, Haarlem, The Netherlands | Management of chronic pulmonary infections due to P. aeruginosa in patients with CF aged ≥6 years | No |
Bronchitol | Mannitol | April 13, 2012 | Pharmaxis Pharmaceuticals Ltd, London, UK | Treatment of CF in adults aged ≥18 years as an add-on therapy to best standard of care | Yes |
Kalydeco | Ivacaftor | July 23, 2012 | Vertex Pharmaceuticals (Europe) Ltd, London, UK | Treatment of CF in patients aged ≥2 years who have the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N and S549R; it is also indicated for the treatment of adult CF patients who have an R117H mutation in the CFTR gene | Yes |
Vantobra | Tobramycin | March 18, 2015 | PARI Pharma GmbH, Starnberg, Germany | Management of chronic pulmonary infection due to P. aeruginosa in patients with CF aged ≥6 years | No |
Quinsair | Levofloxacin | March 26, 2015 | Chiesi Orphan BV, Schiphol, The Netherlands | Management of chronic pulmonary infections due to P. aeruginosa in adult patients with CF | No |
Orkambi | Lumacaftor/ivacaftor | Nov 19, 2015 | Vertex Pharmaceuticals (Europe) Ltd, London, UK | Treatment of CF in patients aged ≥12 years who are homozygous for the F508del mutation in the CFTR gene | No |
Enzepi¶ | Pancreas powder | June 29, 2016 | Allergan Pharmaceuticals International Ltd, Dublin, Ireland | Pancreatic enzyme replacement treatment in exocrine pancreatic insufficiency due to CF or other conditions (e.g. chronic pancreatitis, post-pancreatectomy or pancreatic cancer); indicated in adults, infants, children and adolescents | No |
P. aeruginosa: Pseudomonas aeruginosa. #: for more details about the definition of orphan designation, see section “Trends in CF drug development”; ¶: Enzepi was withdrawn from the market in July 2017 for commercial reasons.