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Kalydeco, ivacaftor (VX-770)

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Company Vertex Pharmaceuticals Inc.
DescriptionSmall molecule potentiator of cystic fibrosis transmembrane conductance regulator (CFTR)
Molecular Target Cystic fibrosis transmembrane conductance regulator (CFTR)
Mechanism of ActionCFTR stabilizer
Therapeutic ModalitySmall molecule
Latest Stage of DevelopmentMarketed
Standard IndicationCystic fibrosis (CF)
Indication DetailsTreat cystic fibrosis (CF); Treat cystic fibrosis (CF) in patients ages >=6 with 1 the mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D; Treat cystic fibrosis (CF) in patients ages 6 and older who have the G551D mutation; Treat cystic fibrosis (CF) in patients carrying the G551D mutation in the CFTR gene on at least 1 allele; Treat cystic fibrosis (CF) in patients with a CFTR gating mutation; Treat cystic fibrosis (CF) in patients with a non-G551D CFTR gating mutation; Treat cystic fibrosis (CF) in patients with a R117H mutation in the CFTR gene; Treat cystic fibrosis (CF) in patients with clinical evidence of residual CFTR function; Treat cystic fibrosis (CF) patients >=6 years with the R117H mutation in the CFTR gene; Treat cystic fibrosis (CF) patients 2-5 years with 1 of the following 9 mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R
Regulatory Designation

U.S. - Breakthrough Therapy (Treat cystic fibrosis (CF) in patients ages 6 and older who have the G551D mutation);
U.S. - Orphan Drug (Treat cystic fibrosis (CF) in patients ages 6 and older who have the G551D mutation);
U.S. - Priority Review (Treat cystic fibrosis (CF) in patients carrying the G551D mutation in the CFTR gene on at least 1 allele);
EU - Accelerated Assessment (Treat cystic fibrosis (CF) in patients carrying the G551D mutation in the CFTR gene on at least 1 allele);
EU - Orphan Drug (Treat cystic fibrosis (CF) in patients ages 6 and older who have the G551D mutation);
Switzerland - Orphan Drug (Treat cystic fibrosis (CF))

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