EU/3/18/2116 - orphan designation for treatment of cystic fibrosis
Ivacaftor, N-(1,3-dimethyl-1H-pyrazole-4-sulfonyl)-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1H-pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide, tezacaftor (ivacaftor / tezacaftor / elexacaftor)
Orphan
Human