A prospective, single-center observational study in Buenos Aires, Argentina evaluated a generic formulation of elexacaftor/tezacaftor/ivacaftor (ETIgf) in 71 people with CF (≥6 years) with responsive variants over 12 months — split into modulator-naïve (Group 1, n=43) and prior lumacaftor/ivacaftor users (Group 2, n=28).
At 12 months, Group 1 / Group 2 showed: ppFEV1 +20.8% / +18.5%; LCI2.5 −13.3% / −11.2%; sweat chloride −53.1% / −58.2%; CFQ-R +66.1 / +30.4 points; BMI z-score +0.5 in Group 1 (non-significant in Group 2); and pulmonary exacerbations reduced by 95% in both groups. Adverse events were mild (5 elevated transaminases, 3 rash).
Single-center study with a small sample (n=71), limiting generalizability. No direct head-to-head comparison with the branded originator formulation. Fecal elastase did not change, suggesting exocrine pancreatic outcomes may need longer follow-up or different metrics.
A generic ETI formulation produced in Argentina showed efficacy and safety closely matching originator data, supporting the feasibility of affordable generic CFTR modulators for expanding access in resource-limited settings. Clinicians in lower-income regions may consider advocating for generic modulator pathways where patent barriers are absent.
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