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))

• Published in: PloS one 2021-12, Vol.16 (12), p.e0261410
• Main Authors: Amelina, Elena L, Krasovsky, Stanislav A, Akhtyamova-Givirovskaya, Nina E, Kashirskaya, Nataliya Yu, Abdulganieva, Diana I, Asherova, Irina K, Zilber, Ilya E, Kozyreva, Liliya S, Kudelya, Lubov M, Ponomareva, Natalya D, Revel-Muroz, Nataliya P, Reutskaya, Elena M, Stepanenko, Tatiana A, Seitova, Gulnara N, Ukhanova, Olga P, Magnitskaya, Olga V, Kudlay, Dmitry A, Markova, Oksana A, Gapchenko, Elena V
• Format: Article
• Language: English
• Subjects: Adult; Antibodies; Biological products; Biology and Life Sciences; Biosimilar pharmaceuticals; Biosimilar Pharmaceuticals - chemical synthesis; Biosimilar Pharmaceuticals - therapeutic use; Biotechnology; Body weight; Clinical trials; Comparative analysis; Comparative studies; Cystic fibrosis; Cystic Fibrosis - complications; Cystic Fibrosis - drug therapy; Cystic Fibrosis - physiopathology; Deoxyribonuclease; Deoxyribonuclease I - chemistry; Deoxyribonuclease I - metabolism; Deoxyribonuclease I - therapeutic use; Deoxyribonucleases - therapeutic use; Dornase alfa; Drug therapy; Expectorants - therapeutic use; Female; Forced Expiratory Volume; Genetics; Health care; Health facilities; Health services; Humans; Immunogenicity; Impairment; Limited liability companies; Lung - physiopathology; Lung diseases; Lung Diseases - drug therapy; Lung Diseases - physiopathology; Male; Medicine and Health Sciences; Middle Aged; Mucociliary Clearance; Parameters; Patient outcomes; Patients; People and Places; Prospective Studies; Pulmonary functions; Recombinant Proteins - chemistry; Recombinant Proteins - metabolism; Recombinant Proteins - pharmacology; Recombinant Proteins - therapeutic use; Research and analysis methods; Respiratory function; Respiratory insufficiency; Safety; Subgroups; Testing
• ISSN: 1932-6203; 1932-6203
• DOI: 10.1371/journal.pone.0261410

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