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))

PLoS One. 2021 Dec 23;16(12):e0261410. doi: 10.1371/journal.pone.0261410. eCollection 2021.

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.

Publication types

  • Clinical Trial, Phase III
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Biosimilar Pharmaceuticals / chemical synthesis
  • Biosimilar Pharmaceuticals / therapeutic use*
  • Cystic Fibrosis / complications
  • Cystic Fibrosis / drug therapy*
  • Cystic Fibrosis / physiopathology
  • Deoxyribonuclease I / chemistry
  • Deoxyribonuclease I / metabolism
  • Deoxyribonuclease I / therapeutic use*
  • Deoxyribonucleases / therapeutic use*
  • Expectorants / therapeutic use
  • Female
  • Forced Expiratory Volume
  • Humans
  • Lung / physiopathology
  • Lung Diseases / drug therapy
  • Lung Diseases / physiopathology
  • Male
  • Middle Aged
  • Mucociliary Clearance
  • Prospective Studies
  • Recombinant Proteins / chemistry
  • Recombinant Proteins / metabolism
  • Recombinant Proteins / pharmacology
  • Recombinant Proteins / therapeutic use

Substances

  • Biosimilar Pharmaceuticals
  • Expectorants
  • Recombinant Proteins
  • Deoxyribonucleases
  • Deoxyribonuclease I
  • dornase alfa

Associated data

  • ClinicalTrials.gov/NCT04468100

Grants and funding

The study was initiated and supported by the GENERIUM JSC. GENERIUM JSC had role in the study design, data collection and analysis, decision to publish, and preparation of the manuscript. 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 from the sponsor (GENERIUM JSC).