TABLE 2

Cystic fibrosis (CF) products that received a marketing authorisation within the centralised procedure until 2016

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more details about the definition of orphan designation, see section “Trends in CF drug development”; ^¶: Enzepi was withdrawn from the market in July 2017 for commercial reasons.