Catridecacog 2,500unit powder and solvent for solution for injection vials

Circulating factor XIII (FXIII) consists of two active (A) and two carrier (B) subunits in tetrameric form. Congenital FXIII deficiency is a rare autosomal-recessive trait that mostly results from an FXIII A-subunit deficiency. Classic coagulation assays, such as prothrombin time or activated partial thromboplastin time, are not sensitive to FXIII; therefore, specific FXIII assays are necessary to detect the deficiency. The clinical picture of congenital FXIII deficiency comprises abortions, umbilical cord bleeding, increased surgical bleeding, intracerebral hemorrhage (which can, unfortunately, be the very first sign of severe FXIII deficiency), menorrhagia, and wound-healing disorders. Given the risk of intracranial hemorrhage, continued prophylaxis is to be recommended in severe deficiency, even in the actual absence of bleeding symptoms. Functional FXIII half-life decreases in consumptive processes (eg, surgery), explaining why increased dosing is needed in such situations. A recombinant FXIII (rFXIII) subunit-A molecule, which is expressed in Saccharomyces cerevisiae, has been evaluated for replacement therapy in congenital FXIII deficiency. The bleeding frequency under continued rFXIII prophylaxis during a year-long treatment period was significantly lower compared to on-demand treatment. Importantly, no severe spontaneous bleedings occurred, and bleeding requiring additional intervention only occurred after relevant trauma. Treatment with rFXIII proved to be safe: antibodies against rFXIII detected in four patients were not considered clinically relevant. No allergic reactions were observed. These data show that rFXIII can be used safely and effectively for continued prophylaxis in congenital FXIII deficiency; it is conceivable that this also holds true for treatment of acute bleeding, but clinical proof of this is pending.

Abstract licence: CC BY-NC 3.0

Despite many articles regarding the antihemorrhagic treatment and prophylaxis, there is a lack of experience about how to best conduct major surgical procedures in patients with congenital factor XIII (FXIII) deficiency. Here we report a case of surgery (right inguinal hernia, complicated by heaviness and pain) performed in a patient with FXIII deficiency, receiving recombinant FXIII prophylaxis (Catridecacog 35 UI/kg every 28±2 days). Our experience shows that Catridecacog can be used safely and effectively not only for continued prophylaxis but also in surgery and adds to the very limited body of evidence currently available on surgery in this bleeding disorder.

Abstract licence: CC BY 4.0

This trial was conducted in Europe. The aim of this trial was to investigate safety and pharmacokinetics of multiple doses of catridecacog (recombinant factor XIII, rFXIII) in healthy volunteers. Conditions: Congenital Bleeding Disorder, Congenital FXIII Deficiency, Healthy. Interventions: catridecacog, placebo.

Source: ClinicalTrials.gov (public domain)

This trial is conducted in Europe and the United States of America (USA). The aim of this trial is to evaluate the safety of escalating single doses of rFXIII (recombinant factor XIII, catridecacog) administered following first time myocardial revascularization requiring cardiopulmonary bypass (CPB). Conditions: Acquired Bleeding Disorder, Cardiac Surgery Requiring Cardiopulmonary Bypass. Interventions: catridecacog, placebo.

Source: ClinicalTrials.gov (public domain)

This trial is conducted in Japan. The aim of this trial is to investigate the safety profile of recombinant factor XIII (rFXIII) assessed by the occurrence of adverse events in healthy Japanese subjects. In addition pharmacokinetic parameters will be investigated. Conditions: Acquired Bleeding Disorder, Cardiac Surgery Requiring Cardiopulmonary Bypass, Healthy. Interventions: catridecacog, placebo, catridecacog, placebo.

Source: ClinicalTrials.gov (public domain)

This study is conducted globally. The aim of this observational study is to investigate the incidence of specific adverse drug reactions associated with the use of recombinant factor XIII (NovoThirteen®) in patients with congenital FXIII A-subunit deficiency (congenital FXIII deficiency), comprising FXIII antibodies, allergic reactions, embolic and thrombotic events and lack of therapeutic effect. The study will aim at observing all patients exposed to NovoThirteen® in the EU, and additional patients from selected non-EU countries. Recombinant FXIII (rFXIII) is registered in EU and Switzerland as NovoThirteen® and in Canada as Tretten®. Conditions: Congenital Bleeding Disorder, Congenital FXIII Deficiency. Interventions: catridecacog.

Source: ClinicalTrials.gov (public domain)