ivacaftor + ataluren

Ivacaftor + ataluren is a combination drug investigated for the treatment of cystic fibrosis (CF) patients with nonsense mutations in the CFTR gene. Ivacaftor is a CFTR potentiator that increases chloride ion transport by enhancing the gating function of the CFTR protein at the cell surface. Ataluren is a readthrough agent designed to enable ribosomes to bypass premature stop codons caused by nonsense mutations, allowing production of full-length functional CFTR protein. This combination aims to address both defective protein production and function in certain genetic subsets of CF patients. The combination has been studied primarily in clinical trials but has not demonstrated consistent meaningful clinical benefit; development for cystic fibrosis has been discontinued by PTC Therapeutics after failed Phase 3 trials, though some ongoing small studies continue to explore its effects in combination with ivacaftor alone[1][2][3][4][10].

Other names

PTC124 + ivacaftor