Real-Life Experience With a Generic Formulation of Elexacaftor/Tezacaftor/Ivacaftor in Patients With Cystic Fibrosis
Alejandro Teper1, Viviana Rodríguez1, Silvina Lubovich1
1Division of Respiratory, Hospital de Niños Ricardo Gutiérrez, Ciudad Autónoma de Buenos Aires, Argentina.
Introduction:
Elexacaftor/Tezacaftor/Ivacaftor (ETI) has revolutionized cystic fibrosis (CF) care. In Argentina, the absence of patent restrictions allowed the production of a generic ETI formulation (ETIgf), significantly reducing costs.
Objective:
This study evaluates the real-world clinical impact of ETIgf in people with CF (pwCF).
Methods:
This prospective, single-center observational study in Buenos Aires, Argentina, included 71 pwCF (≥ 6 years) with responsive variants. Participants were categorized as: Group 1 (modulator-naïve) or Group 2 (previously on lumacaftor/ivacaftor). Clinical outcomes, including ppFEV1, LCI2.5, BMI z-score, CFQ-R, sweat chloride concentration (SCC), fecal elastase levels, liver enzymes and pulmonary exacerbations (PEx), were assessed over 12 months following the initiation of ETIgf.
Results:
Group 1 included 43 patients, and Group 2 included 28 patients (median age: 10.9 and 10.6 years, respectively). The mean percentage change from baseline after 12 months of treatment for Group 1 and Group 2 was: ppFEV1 +20.8 (p < 0.01) and +18.5 (p < 0.01); LCI2.5 -13.3 (p < 0.01) and -11.2 (p < 0.05); CFQ-R + 66.1 (p < 0.01) and +30.4 (p < 0.01); and SCC -53.1 (p < 0.01) and -58.2 (p < 0.01), respectively. BMI z-score increased by 0.5 in Group 1 (p < 0.001), while Group 2 showed a non-significant increase. Pulmonary exacerbations were reduced by 95% in both groups. No changes were observed in fecal elastase levels. Adverse events were mild: five patients experienced elevated transaminase levels and three presented with a rash.
Conclusion:
ETIgf demonstrates robust clinical efficacy and safety, mirroring results reported for the originator. These findings highlight the viability of affordable generic modulators in expanding global access to life-changing therapy.
More Related Videos
07:04Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients
Published on: February 11, 2017
08:00Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study
Published on: April 11, 2018
Related Concept Videos
Cystic Fibrosis: Management
Sinus disease and chronic sinusitis...
Cystic Fibrosis: Pathogenesis
CF is primarily caused by a genetic mutation in a chromosome 7 gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The most common gene mutation leading to CF is the ΔF508 mutation, but...
Pharmaceutical Alternatives: Stability-Related Therapeutic Nonequivalence
Pharmacogenetics of Drug Targets: β₂-Adrenergic Receptors, Apo E, Thymidylate Synthase
Pharmaceutical Alternatives: Polymorphic Form-Related and Particle Size-Related Therapeutic Nonequivalence
Impact of Pharmacokinetic–Pharmacodynamic Models: Regulatory Decisions
