307 Cystic Fibrosis Patient Registry of Moscow and Moscow Region (CFPRMMO) in 2010–2012

Amelina, E.; Krasovsky, A.; Voronkova, A.; Черняк, А. В.; Gorinova, Y.; Kashirskaya, N.; Kondratieva, E.; Nikonova, V.; Shabalova, L.; Shelepneva, N.; Sherman, V.D.; Simonova, O.; Usacheva, M.; Kapranov, N.; Чучалин, А. Г.

doi:10.1016/s1569-1993(15)30481-1

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“…According to the Research Centre for Medical Genetics and neonatal screening, the frequency of CF in Russia is 1:8,000–12,000 newborns with significant differences by regions [ 2 – 5 ]. The Russian CF Patients Registry, created to assess the prevalence, genetic and clinical polymorphism, approaches to the treatment of the disease, included data of 3,142 patients by 2018 (mean age is 12.8±9.6 years, median age is 10.4 (12.4) years, proportion of patients aged ≥ 18 years is 24.7%) [ 6 , 7 ]. Since 2011 the Registry patients’ number increased from 1,026 in 2011 to 3,142 in 2018.…”

Section: Introductionmentioning

confidence: 99%

“…Mean FEV 1 (forced expiratory volume in 1 second) and FVC (forced vital capacity) were 77.6 ± 26.1% and 84.6 ± 21.8% of predicted, respectively. The median life expectancy for patients born in 2014–2018 was 33.8 years [ 6 ].…”

Section: Introductionmentioning

confidence: 99%

“…Dornase alfa is one of the most used medicinal products in CF patients [ 12 , 13 ]. According to the Russian CF Patients Registry, in 2018 95.7% of CF patients were administered dornase alfa [ 6 ].…”

Section: Introductionmentioning

confidence: 99%

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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))

Amelina

Krasovsky

Akhtyamova-Givirovskaya

et al. 2021

PLoS ONE

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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.

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%