Year in Review: Cystic Fibrosis Modulator Therapy—Efficacy, Access, and Evolution

The introduction of cystic fibrosis transmembrane conductance regulator (CFTR) modulators has transformed the therapeutic landscape of cystic fibrosis. CFTR modulators target specific functional defects in the CFTR protein, improving its folding, trafficking, gating, or conductance depending on mutation class. As CFTR modulators become the standard of care, emerging data highlight not only their transformative benefits but also knowledge gaps regarding long-term outcomes, variability in therapeutic response, effects on early disease, and extra-pulmonary implications for people with cystic fibrosis (CF) complications. Additionally, access, tolerability, and the needs of individuals with rare or non-modulator-responsive mutations remain important challenges. This article aims to provide a summary of recent literature pertaining to CFTR modulators. Through this summary, we seek to enhance the knowledge of the growing body of evidence guiding the next era of CF care, in which disease modification at the molecular level is increasingly achievable.