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dornase alfa (DNase, IBC Generium / Tigerase)

✓ Approved

Generium Pharmaceutical · DNASE1 · Recombinant Proteins

What is dornase alfa?

dornase alfa is a recombinant proteins developed by Generium Pharmaceutical. It is approved for therapeutic indications via inhaled.

Drug Profile

Brand NamesDNase, IBC Generium, Tigerase
CompanyGenerium Pharmaceutical
Drug ClassRecombinant Proteins
Molecular TargetDNASE1
RouteInhaled
StatusApproved
Sources: ClinicalTrials.gov, Generium Pharmaceutical

Mechanism of Action

Molecular Targets

dornase alfa acts on 1 molecular target:

DNASE1deoxyribonuclease 1 (DNL1, DRNI)
Want deeper analysis?Noah AI can explain complex mechanisms and compare to similar drugs.

Therapeutic Indications

dornase alfa is developed for 1 unique indication across 1 therapeutic area.

Therapeutic AreaConditionPhase
Congenital, familial and genetic disordersCystic fibrosis✓ Approved

Related Research Articles

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Efficacy and Safety of Insulin Efsitora Versus Degludec in Adults with Type 2 Diabetes Who Are Insulin-Naïve: Japan Subgroup Analysis of QWINT-2.

Kiyosue Arihiro A, Inoue Mariko M, Takita Yasushi Y, Nasu Risa R et al.

The phase 3 QWINT-2 study demonstrated that once-weekly insulin efsitora alfa (efsitora) was noninferior to once-daily insulin degludec (degludec) in reducing glycated hemoglobin (HbA1c) at week 52 when added to existing noninsulin glucose-lowering agents in adults with type 2 diabetes who were insulin-naïve. A Japan subgroup analysis of QWINT-2 using two dosing algorithms is presented here. Participants from Japan were randomized 1:1 to efsitora or degludec and followed one of two dosing algorithms: a general dosing algorithm or an optional alternative dosing algorithm available for participants anticipated to require less insulin, characterized by a body weight ≤ 60 kg or HbA1c level ≤ 7.5% at baseline. Assessments included changes in HbA1c and fasting blood glucose from weeks 0-52, time spent in target glucose range (TIR) from weeks 48-52, and hypoglycemia from weeks 0-52. In total, 144 participants from Japan were included (efsitora, n = 71; degludec, n = 73). Demographic and baseline characteristics were generally balanced between treatment groups. From weeks 0-52, mean HbA1c decreased from 8.04% to 6.63% with efsitora and from 8.00% to 6.64% with degludec (estimated treatment difference, -0.01%). TIR was similar between efsitora and degludec from weeks 48-52. Rates of combined level 2 or 3 hypoglycemia were low overall (weeks 0-52) and during the initial dosing period (weeks 0-12). Level 3 hypoglycemia was not reported in any participants with efsitora and two participants with degludec. The incidence of adverse events was similar between efsitora and degludec. The efficacy and safety of efsitora were comparable with degludec using the general and alternative dosing algorithms in Japanese participants. Once-weekly efsitora was comparable to once-daily degludec in reducing HbA1c in Japanese participants who were insulin-naïve. The efficacy and safety of efsitora in Japanese participants were consistent with the overall QWINT-2 study population. NCT05362058.

PMID 42177728
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PubMedJournal of inherited metabolic disease2026-05-23

Mapping Clinical Progression to Brain Atrophy in CLN2 Patients Under Cerliponase Alfa Treatment: A Prospective Neuroimaging Study.

Petersen Marvin M, Westermann Lena Marie LM, Hagenah Luca L, Nickel Miriam M et al.

Neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a lysosomal storage disorder, causes early childhood psychomotor regression, vision loss, seizures, and rapid progressive gray matter loss. However, the link between neurodegenerative processes induced by lysosomal pathophysiology and the clinical phenotype remains unclear. This study investigated the longitudinal association of gray matter atrophy on MRI with in-depth clinical phenotyping in 27 patients receiving intraventricular enzyme therapy (ntimepoints = 170; biannual clinical assessments and MRIs). Longitudinal changes in cortical thickness and subcortical volumes were modeled via linear mixed effects regression. We used linear regression to correlate 24-week (Δ24) changes in clinical assessments with global cortical thickness and applied multivariate data-driven statistics to model how specific brain regions are associated with clinical domains. Our analysis revealed a significant reduction in the mean cortical thickness over time (β = -0.002, p = 0.021), corresponding to an annual loss of 4.2%, compared to natural history controls with 12.5%, respectively. Regional analysis revealed a widespread pattern of cortical and subcortical gray matter atrophy. Global cortical thickness reductions over 24 weeks (Δ24) were significantly associated with changes in the Hamburg motor and language scale Δ24, Weill Cornell scale Δ24, and Movement Disorder Inventory Δ24. Multivariate statistics identified a significant latent dimension relating regional morphometric abnormalities to worse clinical outcomes, accounting for 82% of the shared variance. Leveraging connectome data, we demonstrated that atrophy was linked to brain network architecture. Given their strong associations with clinical outcomes, MRI-based brain morphometric measures are promising CLN2 disease biomarkers to aid diagnosis, monitor disease progression, and guide therapy.

PMID 42175674
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PubMedLeukemia & lymphoma2026-05-22

Long-term outcomes of ropeginterferon alfa-2b-njft in polycythemia vera: a review of safety, efficacy, and potential disease modification.

Abu-Zeinah Ghaith G, Reeves Brandi B

Polycythemia vera (PV) is a chronic myeloproliferative neoplasm characterized by erythrocytosis, thrombotic risk, and possible progression to aggressive myeloid neoplasms. Optimal management should achieve durable hematologic response, tolerability, and potential for long-term disease modification. Interferon alpha (IFN-α) is unique among current PV treatments for its disease-modifying potential; however, long-term tolerability concerns remain. Ropeginterferon alfa-2b-njft (ropeg), a novel monopegylated IFN-α, is the only IFN-α approved in the US for PV treatment. Phase III data from PROUD-PV (NCT01949805; EudraCT, 2012-005259-18) and its phase IIIb extension, CONTINUATION-PV (NCT02218047; EudraCT, 2014-001357-17), demonstrate that ropeg, compared with hydroxyurea, achieves hematologic response more slowly but provides greater, more durable responses after ∼18 months, underscoring the importance of long-term adherence. Early adverse events can challenge adherence, emphasizing the need for proactive symptom management. This review offers evidence- and experience-based perspectives on long-term ropeg treatment and adverse event management to support adherence and maximize therapeutic benefit in PV.

PMID 42169665
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PubMedScientific reports2026-05-21

Genome editing-based refinement of GPCR visualization in mice using the oxytocin receptor as a model.

Inoue Yukiko U YU, Kurumiya Eon E, Tany Ryosuke R, Koike Eriko E et al.

G protein-coupled receptors (GPCRs) mediate diverse physiological functions and are major drug targets, yet their in vivo visualization remains challenging due to poor antigenicity. In this study, we integrate structural prediction, functional assay, and genome editing to systematically refine epitope-tagging strategies for GPCRs in mouse models. Using the oxytocin receptor (Oxtr) as a representative GPCR, we compare conventional triple-HA tagging with two recently developed high-sensitivity tags, Spaghetti Monster fluorescent protein (smFP) and the ALFA tag. Each tag is fused to the Oxtr C-terminus, and in vitro G protein activation assays show that all three variants retain ~ 70% of wild-type activity, consistent with structural predictions from AlphaFold 3. We then generate knock-in mice employing genome editing to evaluate in vivo performance. smFP markedly improves the signal-to-noise ratio in tissue staining, whereas the ALFA tag disrupts trafficking without enhancing sensitivity. Primary cultured neurons derived from smFP knock-in mice clearly visualize the distribution of Oxtr along dendrites down to their terminals at subcellular resolution. Collectively, the refined animal models advance GPCR biology and inform the design of future genome editing approaches.

PMID 42161997
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PubMedJournal of cystic fibrosis : official journal of the European Cystic Fibrosis Society2026-05-21

Effect of discontinuation and reduction of respiratory cotherapies on the effectiveness of CFTR modulators.

Reynaud Q Q, Payet C C, Fernandez V V, Eid E E et al.

We evaluated whether the initiation of CFTR modulators led to a concomitant reduction in or discontinuation of respiratory cotherapies in French pwCF treated with IVA or LUM/IVA and determined whether the reduction in or discontinuation of respiratory cotherapies led to decreased effectiveness of CFTR modulators on clinical status. We conducted a national retrospective cohort study using data from the French CF Registry (FCFR) linked to the French National Health Data System (SNDS), including all French pwCF aged 6 years and older who initiated IVA or LUM/IVA between 2012 and 2020 and who were treated for at least six months. Exposure to respiratory cotherapies was defined as at least one dispensing event of azithromycin, RhDNase, or inhaled antibiotics (aztreonam, colistimethate, or tobramycin). In total, 1378 pwCF were included in the study, with >90% of the population treated with LUM/IVA. Dornase alpha was discontinued in 25% of adults and 17% of children. Azithromycin was discontinued in 19% of adults and children. For inhaled antibiotics, discontinuation of the three treatments was observed in almost 40% of adults. No effect of the discontinuation of respiratory cotherapy was detected on the FEV1%, number of intravenous antibiotic courses or number of total antibiotic courses (0.13, 95% CI (-1.44; 1.70)); the values were -0.03 (-0.18; 0.13) and 0.04 (-0.25; 0.32), respectively. This real-world study revealed a high rate of respiratory cotherapy discontinuation or reduction during the year following LUM/IVA or IVA initiation, without measurable clinical impact.

PMID 42161713
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PubMedJournal of atherosclerosis and thrombosis2026-05-21

Hospital Course and Outcomes After Major Bleeding in Patients on Factor Xa Inhibitors in Japan: A Nationwide Observational Study.

Kohsaka Shun S, Yasaka Masahiro M, Hoshino Mitsuru M, Ide Ryotaro R et al.

To evaluate the clinical characteristics, management, and in-hospital outcomes of patients hospitalized with major bleeding while receiving direct factor Xa inhibitors (FXa-I) using a large-scale, nationwide administrative claims database. This retrospective cohort study analyzed an administrative hospital claims database to identify patients hospitalized with major bleeding while receiving FXa-I between August 2011 and May 2022 (before the launch of andexanet alfa), resulting in 8011 patients (median age, 80.0 years) being eligible for the analysis. The data collected encompassed patient demographics, hospitalization details, bleeding sites, and outcomes of interest, such as in-hospital mortality and the resumption of OACs. The most common bleeding sites were upper gastrointestinal bleeding (GIB; 30.3%), intracranial hemorrhage (ICH; 17.6%), lower GIB (11.7%), and trauma-related bleeding (9.1%). Patients had a median hospital stay of 11.0 days (range, 1-911 days). The overall in-hospital mortality rate was 7.1% (95% CI: 6.6-7.7%). In-hospital mortality rates by bleeding site were 17.8% for ICH, 5.1% for upper GIB, 1.4% for lower GIB, 10.2% for trauma-related bleeding, and 4.1% for other bleeding sites. The most common cause of death was hemorrhage-related (70.1%). OACs were resumed in 67.9% of patients, with a median time to resumption of 3.0 days. In this nationwide study, the in-hospital mortality rate for patients hospitalized for FXa-I-related major bleeding was approximately 7%. Although most patients resumed oral anticoagulant therapy during hospitalization, the significant in-hospital mortality rates observed, especially in cases of ICH, highlight the need for strategies to further improve the outcomes in patients experiencing FXa-I-related complications.

PMID 42161526
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