Tretten (catridecacog) / Novo Nordisk - LARVOL DELTA

A Study of Safety and Effectiveness of NovoThirteen® (rFXIII) During Treatment of Congenital FXIII Deficiency in Japan (clinicaltrials.gov) - P=N/A | N=8 | Completed | Sponsor: Novo Nordisk A/S | Enrolling by invitation ➔ Completed

Trial completion • Hematological Disorders

A multicenter, real-world experience with recombinant FXIII for the treatment of patients with FXIII deficiency: from pharmacokinetics to clinical practice. The Italian FXIII Study. (PubMed, Blood Transfus) - "Efficacy and safety of rFXIII were proven in all patients. The dosage and infusion timing for the treated patients sometimes differed to those reported in the MENTOR pivotal studies, thus underlying the importance of tailored management in a real-world scenario."

Journal • PK/PD data • Real-world • Real-world evidence • Cerebral Hemorrhage • CNS Disorders • Hematological Disorders • Hemophilia • Immunology • Rare Diseases

The pharmacokinetics of recombinant FXIII (catridecacog) from the MENTOR2 trial to a real-world study: a head-to-head comparison. (PubMed, J Thromb Thrombolysis) - "In the RW 55.6% of patients were females, 26.2% in the MENTOR2 (p < 0.05). The mean dosage of drug used for the PK assessment was 35 IU/kg in the MENTOR2, and 33.9 IU/kg in the RW study."

Head-to-Head • Journal • PK/PD data • Real-world evidence • Hematological Disorders • Hemophilia • Rare Diseases

"Lass ihn wenigstens antretten bevor du von rücktritt sprichst. Deppata gehts eh nimmer oder?" (@Xaver09010876)

Safety and effectiveness of recombinant factor XIII-A in congenital factor XIII deficiency: Real-world evidence. (PubMed, Res Pract Thromb Haemost) - "These data confirm that rFXIII-A prophylaxis is well tolerated as long-term care. There were no spontaneous bleeds, ABR was low, and rFXIII-A successfully treated bleeds in patients receiving rFXIII-A prophylaxis."

HEOR • Journal • Real-world evidence • Cardiovascular • Hematological Disorders • Mood Disorders • Thrombosis • Venous Thromboembolism

Catridecacog in the treatment of an Italian population of patients with FXIII deficiency from pharmacokinetics to clinical outcomes (the Italian rFXIII study) (BIC 2021) - "Efficacy and safety of prophylaxis with catridecacog was proven in all patients, also in preventing severe bleeding. The cumulative PK profile was similar to that reported in the MENTOR studies, but dosage and infusion timing for each patient were in some cases very different."

Clinical • Clinical data • PK/PD data • Cerebral Hemorrhage • CNS Disorders • Hematological Disorders • Hemophilia • Immunology • Rare Diseases

[VIRTUAL] Population Parmacokinetics of rFXIII (Catridecacog) and Clinical Outcomes of Prophylaxis in an Italian Population of Patients with Factor XIII Deficiency (ISTH 2021) - "Efficacy and safety of rFXIII was proven in all patients. The role of prophylaxis to prevent severe hemorrhagic events was also highlighted."

Clinical • Clinical data • Cerebral Hemorrhage • CNS Disorders • Hematological Disorders • Hemophilia • Immunology • Rare Diseases

[VIRTUAL] A Novel Assay Using Enzyme Capture – ELISA for Accurate Determination of Factor XIII Activity (ASH 2020) - "Methods : A standard curve was established by diluting known concentrations of recombinant factor XIII-A (rFXIII-A; Tretten) into (1) fXIII-A congenitally deficient and (2) fXIII immunodepleted plasma to determine if our assay is able to detect variable fXIII concentrations from 1 to 200% (0.01 - 2 IU/mL)... We conclude that Enzyme Capture-ELISA is a promising novel method for accurate detection of FXIII activity and is expected to improve sensitivity at low levels (<20%; <0.02 IU/mL), which are most clinically relevant. Further experiments are needed to refine the upper limit of assay linearity and thereby refine the dynamic range of the assay. With further optimization and validation, the EC-ELISA method may provide an improved diagnostic assay."

"Die meisten hatten sicher Corona aber haben nichts gemerkt. bei manchen tauchen keine Symptome auf. und bei manche tretten Symptome auf" (@ardit_aj)

[VIRTUAL] Results from a Phase 4, Real-World Study Confirm the Safety and Effectiveness of rFXIII-A2 for the Treatment of Congenital FXIII A-Subunit Deficiency (ISTH 2020) - "These real-world, long-term data confirm the safety and effectiveness of rFXIII-A2 for prophylaxis, treatment of bleeding episodes and minor surgeries in patients with FXIII-A CD."

Clinical • P4 data • Real-World Evidence • Allergy • Hematological Disorders

[VIRTUAL] An Italian Survey on the Real-world Use of rFXIII (Catridecacog) in Patients with Factor XIII Deficiency (ISTH 2020) - "This is a first survey on real-world use of catridecacog performed in patients with FXIII deficiency. The efficacy and safety of rFXIII, used in most cases at the recommended dosage and frequency, was proven in all treated patients. The role of prophylaxis to prevent recurrences was also highlighted."

Clinical • Real-World Evidence • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

[VIRTUAL] The real-life use of rFXIII (catridecacog) in patients with severe Factor XIII deficiency: an Italian survey (WFH 2020) - "This is a first survey on real-life use of catridecacog performed in an Italian population of FXIII deficient patients. The efficacy and safety of rFXIII, used in most cases at the recommended dosage and frequency, was proven in all treated patients. The role of prophylaxis to prevent haemorrhagic recurrences in patient presenting severe disorder was also highlighted."

Clinical • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Treatment with Recombinant Factor XIII (Tretten) in a Pregnant Woman with Factor XIII Deficiency. (PubMed) - Am J Case Rep - "CONCLUSIONS Tretten, which is not indicated in pregnancy, offered a safe, effective treatment for miscarriages secondary to factor XIII-A deficiency in our patient. Further research is required to confirm this finding."

Journal • Biosimilar

Safety and efficacy of monthly replacement therapy with recombinant factor XIII (rFXIII) in paediatric subjects with congenital factor XIII A-subunit deficiency: An extension to trial F13CD-3760/mentor 4 (mentor 5) (clinicaltrials.gov) - P3, N=6; Sponsor: Novo Nordisk; Active, not recruiting; Completion date: Dec 2013 -> Jun 2014.

Trial completion date • Hemophilia

Safety of monthly recombinant factor XIII replacement therapy in subjects with congenital factor XIII deficiency: An extension to trial F13CD-1725 (mentor 2) (clinicaltrials.gov) - P3, N=60; Sponsor: Novo Nordisk; Completion Date: May 2013 -> Dec 2015; Recruiting -> Active, not recruiting.

Enrollment closed • Trial completion date • Hemophilia

New data on the safety and efficacy of recombinant FXIII in patients with congenital FXIII A-subunit deficiency (ASH 2014) - Presentation time: Saturday, December 6, 2014, 5:30 PM-7:30 PM; Abstract #1520; P3, N=60; NCT00978380; Sponsor: Novo Nordisk; "Prophylaxis of bleeding of patients with congenital FXIII A-subunit deficiency with rFXIII in the mentor™ trials program has demonstrated very effective bleed control, with an excellent safety profile. The ABR in the ongoing mentorTM2 safety extension trial was 0.042, which is lower than the rate of 0.138 seen in the mentor™1 pivotal study. A bleeding rate of 0.042 corresponds to an average patient having 1 bleed approximately every 24 years."

P3 data • Hemophilia

Novo Nordisk: London FY 2012 Investor Presentation (Novo Nordisk) - "NovoThirteen was effective in preventing bleeding after once-monthly dosing for 12 months; Annual frequency of bleeds requiring treatment was 0.138 (vs. 2.91 for historical controls); Treatment with NovoThirteen was generally safe with only transient, non-neutralising antibody formation being reported in few patients"

P3 data • Hemophilia

Novo Nordisk: Annual Report 2014 (Novo Nordisk) - Anticipated expiry of active ingredient patent in US in 2021; Anticipated data protection in US until 2025; Anticipated expiry of active ingredient formulation patent in Germany in 2016; Anticipated expiry of active ingredient formulation patent in China in 2016; Anticipated expiry of active ingredient formulation patent in Japan in 2016

Anticipated patent expiry • Hemophilia

FDA rejects Novo hemophilia treatment again due to manufacturing issues (WCG’s FDAnews) - "Novo Nordisk struck out again with the FDA, which for the second time declined the Danish drugmaker’s BLA for a recombinant factor XIII (rFXIII) compound to treat hemophilia. According to Novo, manufacturing issues at one of its plants are to blame."

BLA • Hemophilia

FDA approves Tretten to treat rare genetic clotting disorder (FDA) - "The U.S. Food and Drug Administration...approved Tretten, Coagulation Factor XIII A-Subunit (Recombinant), the first recombinant product for use in the routine prevention of bleeding in adults and children who have a rare clotting disorder, known as congenital Factor XIII A-subunit deficiency"

BLA • Hemophilia

European Medicines Agency (EMA) approves catridecacog (NovoThirteen) for congenital factor XIII A-subunit deficiency (Nelm.nhs.uk) - EMA has approved catridecacog (NovoThirteen) for use within the EU for the long term prophylactic treatment of bleeding in pts 6 years & above with congenital factor XIII A-subunit deficiency

European regulatory • Hemophilia

Pharmacokinetics of recombinant factor XIII in young children with congenital FXIII deficiency and comparison with older patients (Haemophilia) - PMID: 23834599; P3, N=6; Sponsor: Novo Nordisk; mentor 4 (NCT01230021); PMID: 23834599; “…single dose of 35 IU kg-1 rFXIII maintained plasma FXIII levels above 0.1 IU mL-1 over a 30-day period in young children with congenital FXIII deficiency, and is, therefore, likely to provide adequate prophylaxis in this age group…”

P3 data • Hemophilia

Tailored prophylaxis with rFXIII (NovoThirteen ) in a young girl with severe FXIII deficiency and previous cephalohaematoma. (PubMed, Haemophilia) - No abstract available

Journal

mentor™6: Use of rFXIII in Treatment of Congenital FXIII Deficiency, a Prospective Multi-centre Observational Study (clinicaltrials.gov) - P=N/A; N=30; Completed; Sponsor: Novo Nordisk A/S; Active, not recruiting ➔ Completed

Clinical • Trial completion

mentor™6: Use of rFXIII in Treatment of Congenital FXIII Deficiency, a Prospective Multi-centre Observational Study (clinicaltrials.gov) - P=N/A; N=30; Active, not recruiting; Sponsor: Novo Nordisk A/S; Enrolling by invitation ➔ Active, not recruiting

Clinical • Enrollment closed