Comparison of biosimilar Tigerase and Pulmozyme in long-term symptomatic therapy of patients with cystic fibrosis and severe pulmonary impairment (subgroup analysis of a Phase III randomized open-label clinical trial (NCT04468100))

Elena L Amelina, Stanislav A Krasovsky, Nina E Akhtyamova-Givirovskaya, Nataliya Yu Kashirskaya, Diana I Abdulganieva, Irina K Asherova, Ilya E Zilber, Liliya S Kozyreva, Lubov M Kudelya, Natalya D Ponomareva, Nataliya P Revel-Muroz, Elena M Reutskaya, Tatiana A Stepanenko, Gulnara N Seitova, Olga P Ukhanova, Olga V Magnitskaya, Dmitry A Kudlay, Oksana A Markova, Elena V Gapchenko, Elena L Amelina, Stanislav A Krasovsky, Nina E Akhtyamova-Givirovskaya, Nataliya Yu Kashirskaya, Diana I Abdulganieva, Irina K Asherova, Ilya E Zilber, Liliya S Kozyreva, Lubov M Kudelya, Natalya D Ponomareva, Nataliya P Revel-Muroz, Elena M Reutskaya, Tatiana A Stepanenko, Gulnara N Seitova, Olga P Ukhanova, Olga V Magnitskaya, Dmitry A Kudlay, Oksana A Markova, Elena V Gapchenko

Abstract

Background: Patients with cystic fibrosis (CF) need costly medical care and adequate therapy with expensive medicinal products. Tigerase® is the first biosimilar of dornase alfa, developed by the lead Russian biotechnology company GENERIUM. The aim of the manuscript to present post hoc sub-analysis of patients' data with cystic fibrosis and severe pulmonary impairment of a larger comparative study (phase III open label, prospective, multi-centre, randomized study (NCT04468100)) of a generic version of recombinant human DNase Tigerase® to the only comparable drug, Pulmozyme®.

Methods: In the analyses included subgroup of 46 severe pulmonary impairment patients with baseline FEV1 level 40-60% of predicted (23 patients in each treatment group) out of 100 patients registered in the study phase III open label, prospective, multi-center, randomized study (NCT04468100), and compared efficacy endpoints (FEV1, FVC, number and time of exacerbations, body weight, St.George's Respiratory Questionnaire) as well as safety parameters (AEs, SAEs, anti-drug antibody) within 24 treatment weeks.

Results: All outcomes were comparable among the studied groups. In the efficacy dataset, the similar mean FEV1 and mean FVC changes for 24 weeks of both treatment groups were observed. The groups were also comparable in safety, all the secondary efficacy parameters and immunogenicity.

Conclusions: The findings from this study support the clinical Tigerase® biosimilarity to Pulmozyme® administered in CF patients with severe impairment of pulmonary function.

Conflict of interest statement

Elena L. Amelina, Stanislav A. Krasovsky, Nataliya Yu. Kashirskaya, Diana I. Abdulganieva, Irina K. Asherova, Ilya E. Zilber, Liliya S. Kozyreva, Lubov M. Kudelya, Natalya D. Ponomareva, Nataliya P. Revel-Muroz, Elena M. Reutskaya, Tatiana A. Stepanenko, Gulnara N. Seitova, Olga P. Ukhanova, Olga V. Magnitskaya received payment for the above-mentioned clinical trial. Dmitry A. Kudlay, Nina E. Akhtyamova-Givirovskaya, Oksana A. Markova, Elena V. Gapchenko are the employees of JSC GENERIUM. This does not alter our adherence to PLOS ONE policies on sharing data and materials.

Figures

Fig 1. Patients’ distribution.
Fig 1. Patients’ distribution.
Fig 2. Kaplan–Meier curves for the time…
Fig 2. Kaplan–Meier curves for the time to the first chronic pulmonary disease exacerbation (FAS and PP populations).

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