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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

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Durable responses to triplet immunotherapy targeting TGF-β, PD-L1, and tumor antigen, with an IL-15 receptor superagonist in mismatch repair proficient castration-resistant prostate cancer.

Redman Jason Mark JM, Madan Ravi A RA, Donahue Renee N RN, Toney Nicole J NJ et al.

Immune checkpoint blockade is minimally active in unselected castration-resistant prostate cancer (CRPC) and does not reproducibly yield durable decreases in prostate-specific antigen (PSA) levels. The Quick Efficacy Seeking Trial was designed to employ a combination of agents to initiate an immune response (with BN-Brachyury vaccine), potentiate that response (with nogapendekin-alfa inbakicept (NAI), an interleukin (IL)-15 receptor superagonist), and reduce or eliminate immunosuppressive entities in the tumor microenvironment (with bintrafusp alfa, a dual inhibitor of programmed death-ligand 1 and transforming growth factor beta). Epacadostat (an indoleamine 2,3-dioxygenase (IDO) inhibitor) was also employed in one cohort to reduce immune suppression induced by IDO's conversion of tryptophan to kynurenine. Patients with CRPC enrolled sequentially to receive vaccine + bintrafusp alfa (Arm 2.1), vaccine + bintrafusp alfa + NAI (Arm 2.2), and vaccine + bintrafusp alfa + NAI + epacadostat (Arm 2.3), with the primary objective to determine response rate. Adverse events in Arms 2.1 and 2.2 were manageable and consistent with the safety profiles of each agent individually, and notable for five individuals developing isolated adrenocorticotropic hormone deficiency. Arm 2.3 was closed early due to skin toxicity. Sustained declines in PSA were seen in 1/13 (8%) patients in Arm 2.1, 7/24 (29%) patients in Arm 2.2, including six with proficient mismatch repair/microsatellite stable tumors, and 0/6 (0%) patients in Arm 2.3. Analyses of peripheral immune profiles provided evidence of a multifaceted antitumor immune response, including IL-15 receptor superagonist NAI-dependent expansion and activation of natural killer cells and CD8+ T cells, increased effector-to-suppressor immune cell ratios, and induction of cytotoxic immune gene programs. NCT03493945.

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PubMedHaemophilia : the official journal of the World Federation of Hemophilia2026-07-24

Extended Half-Life Recombinant Factor VIII Conjugated with Modifying Substances Does Not Affect Fibrin Clot Formation or Stability in Haemophilia A Blood Samples.

Shimonishi Naruto N, Nogami Keiji K

Various extended half-life recombinant factor VIII (EHL-FVIII) products have been designed to improve the pharmacokinetic properties of FVIII, allowing prolonged haemostatic coverage and reducing the injection burden in people with haemophilia A. Nevertheless, the influence of direct molecular attachment on fibrin clot formation and stability remains to be investigated. To investigate the stability and architecture of fibrin clots in the presence of various types of EHL-FVIII. Three EHL-FVIII products with direct attachment modifications (damoctocog alfa pegol, efraloctocog alfa, and rurioctocog alfa pegol) and two standard FVIII (turoctocog alfa and rurioctocog alfa) were added to FVIII-deficient whole blood and plasma at various concentrations for functional comparison. Under whole-blood conditions, functional assays were performed using rotational thromboelastometry (ROTEM) and a microchip flow-chamber system (T-TAS). Under plasma-based conditions, fibrin fibres were directly observed by electron microscopy and coagulation function was assessed using clot waveform analysis (CWA). Anticoagulant and fibrinolytic activities were evaluated by CWA with the addition of activated protein C and tissue plasminogen activator, respectively. At equivalent activity levels, none of the assays revealed significant differences among the three EHL products or the two standard products. All contributed comparably to fibrin clot formation and stability, as well as to anticoagulation and fibrinolysis functions. Direct modification by PEGylation or IgG-Fc fusion to impart EHL characteristics preserves the functional properties of native FVIII. People with haemophilia A require treatment with factor VIII (FVIII) to prevent or control bleeding. Some FVIII products are designed to remain active in the body for a longer time, which can reduce the number of injections needed. This extended half-life is achieved by chemically or biologically modifying FVIII, for example by attaching polyethylene glycol (PEG) or the Fc portion of immunoglobulin G. These treatments are known as extended half-life FVIII (EHL-FVIII) products. However, it has not been fully established whether these modifications affect how blood clots form and remain stable. In this study, we compared three EHL-FVIII products with two standard FVIII products using FVIII-deficient blood and plasma. We evaluated clot formation and stability using several laboratory techniques, including whole-blood assays and scanning electron microscopy. We also examined potential differences in anticoagulant and fibrinolytic properties. At comparable FVIII activity levels, we observed no major differences between the EHL-FVIII products and the standard FVIII products in any of the assays performed. These findings suggest that PEGylation or Fc fusion, which are used to extend the half-life of FVIII, do not impair its functional properties related to fibrin clot formation and stability.

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PubMedMolecular genetics and metabolism reports2026-07-24

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Guimas Arlindo A, Martins Esmeralda E

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PubMedBMJ case reports2026-07-24

Spontaneous CSF rhinorrhoea in a patient with factor VII deficiency: surgical and haematological considerations.

Maniyankode Krishnamohan Goutham G, Chakravarthy Priya P

A middle-aged woman presented with a 4-month history of watery nasal discharge and was diagnosed with spontaneous cerebrospinal fluid (CSF) rhinorrhoea. Routine blood tests revealed a prolonged prothrombin time (PT) leading to further evaluation and the incidental diagnosis of factor VII deficiency. To optimise safety before imaging, the patient received intravenous recombinant factor VII (NovoSeven, Eptacog alfa), after which CT cisternography demonstrated a persistent CSF leak from the cribriform plate. To avoid repeated recombinant factor VII administration, endoscopic endonasal repair of the defect was undertaken immediately, with meticulous intraoperative haemostasis. Her PT and international normalised ratio were closely monitored in the postoperative period. During more than 1 year of follow-up, she remained well with no recurrence. This case underscores the challenges of balancing haemostatic control during surgical intervention in patients with rare bleeding disorders. Notably, no prior reports in the literature describe CSF rhinorrhoea occurring in the setting of factor VII deficiency, making this presentation unique.

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PubMedJournal of nephrology2026-07-23

Haemodialysis in haemophilia B: a case report. Challenges and strategies from a nephrology perspective.

Terzo Chiara C, Maccarrone Rosario R, La Rosa Sandra S, Giuffrida Gaetano G et al.

Haemophilia B, the result of factor IX deficiency, is a complex clinical condition that is particularly challenging when associated with kidney failure. This is primarily due to the high bleeding risk associated with kidney replacement therapies. The increase in life expectancy in haemophilia patients has resulted in a greater prevalence of age-related comorbidities, including chronic kidney disease. Nevertheless, a consensus has yet to be reached regarding the most suitable dialysis modality for this population. In this report, we present a case study of a 68-year-old male patient suffering from severe haemophilia B, who was administered prophylactic Idelvion® (albutrepenonacog alfa) and subsequently underwent arteriovenous fistula creation and haemodialysis initiation without encountering any bleeding complications. This narrative review, from the perspective of a nephrologist, addresses the epidemiology and main clinical considerations regarding dialysis in haemophilia B. These considerations include the choice between haemodialysis and peritoneal dialysis, vascular access planning, haemostasis management with factor IX replacement, and anticoagulation strategies aimed at minimising bleeding risk. A review of the extant literature and clinical reports published between 2020 and 2025 was conducted, integrating documented experiences and available guideline recommendations. In addition, we put forward a series of pragmatic recommendations that are -underpinned by a systematic decision-making framework. Through meticulous multidisciplinary planning incorporating the utilisation of factor IX concentrates, meticulous surgical techniques, and tailored anticoagulation protocols, patients with haemophilia B can be deemed eligible for chronic haemodialysis with a reasonable degree of safety.

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PubMedOncology2026-07-21

Practical Guide for Managing Philadelphia-Negative Myeloproliferative Neoplasms in Patients with Organ Dysfunction.

Abdulsalam Shams S, Al-Shibly Rafal R, Kaddoura Rasha R, Ismail Omar Mohammad OM et al.

Classical Philadelphia-negative myeloproliferative neoplasms (MPNs), including polycythaemia vera, essential thrombocythaemia, and myelofibrosis, frequently occur in patients with renal, hepatic, cardiovascular, or hematologic comorbidities. These vulnerabilities complicate treatment selection, dose adjustment, and toxicity monitoring. However, patients with advanced kidney disease, dialysis dependence, decompensated cirrhosis, severe cytopenias, or overlapping organ impairment are often underrepresented in prospective trials. This narrative review presents a practical, organ-adapted framework for selecting MPN therapy in clinically complex patients. We define renal and hepatic impairment, compare commonly used cytoreductive and targeted therapies-including hydroxyurea, busulfan, anagrelide, ruxolitinib, fedratinib, momelotinib, pacritinib, ropeginterferon alfa-2b, and pegylated interferon alfa-2a-and discuss their use in dialysis, decompensated liver disease, cytopenic myelofibrosis, anemia-dominant myelofibrosis, and combined organ vulnerability. The proposed approach prioritizes label-based dose modification when available, cautious dose initiation when evidence is extrapolated, structured early monitoring, and multidisciplinary review for high-risk patients. Management of Philadelphia-negative myeloproliferative neoplasms (MPNs) in organ dysfunction should be individualized according to disease phenotype, treatment goal, organ reserve, hematologic tolerance, frailty, and label-specific safety restrictions. In renal impairment, hydroxyurea and ruxolitinib may remain feasible in selected patients when dose-adjusted and monitored closely, whereas dialysis and severe renal impairment require conservative initiation and multidisciplinary review because direct evidence remains limited. In myelofibrosis, JAK inhibitor choice should be phenotype-adapted: ruxolitinib remains a common standard when blood counts and organ function permit, momelotinib is attractive in anemia-dominant disease, pacritinib is particularly relevant in severe thrombocytopenia, and fedratinib is useful in selected settings but requires thiamine-focused safety management. In hepatic dysfunction or overlapping organ vulnerability, therapy should prioritize safety, reversible contributors, conservative starting strategies, explicit stopping rules, and early reassessment.

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