ABBV-119

ABBV-119 is an investigational small molecule drug developed by AbbVie as a corrector of cystic fibrosis transmembrane conductance regulator (CFTR) protein mutations, specifically classified as a C2 corrector. Its primary mechanism of action is to modulate the CFTR protein, aiming to restore its function in patients with cystic fibrosis who have at least one F508del mutation. ABBV-119 has been studied in combination with other CFTR modulators such as galicaftor (ABBV-2222) and navocaftor (ABBV-3067), forming part of triple combination therapies intended to enhance the restoration of CFTR expression and function beyond dual therapy regimens. Clinical development reached Phase II for cystic fibrosis; however, trials were terminated due to strategic considerations and efficacy results[1][6]. Preclinical studies identified reversible hepatobiliary effects and elevated liver enzymes as potential risks[3][6].