Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as NNC-0156-0000-0009, 40K PEG-rFIX
nonacog beta pegol · 7 trials · 2 indications
Haemostatic effect of N9-GP for treatment of bleeding episodes was assessed by 4-point response scale: none, moderate, good or excellent. Evaluation during trial was done by participant and/or parent(s)/caregiver within approximately 8 hours after a single injection as follows: Excellent: Abrupt pain relief and/or clear improvement in objective signs of bleeding within approximately 8 hrs after a single injection; Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hrs after a single injection, but possibly requiring more than one injection for complete resolution; Moderate: Probable or slight beneficial effect within approximately 8 hours after the first injection, but usually requiring more than one injection; None: No improvement, or worsening of symptoms.
Number of participants with incidence of inihibitory antibodies against FIX after 50 ED is presented and defined as an inhibitory antibody titre greater than equal to 0.6 Bethesda unit (BU) at two consecutive tests performed at the central laboratory and also tested positive for nonacog beta pegol binding antibodies.
Number of participants with incidence of inihibitory antibodies against FIX after 100 ED is presented and defined as an inhibitory antibody titre greater than equal to 0.6 Bethesda unit (BU) at two consecutive tests performed at the central laboratory and also tested positive for nonacog beta pegol binding antibodies.
Number of participants with incidence of inihibitory antibodies against FIX at end of trial is presented and defined as an inhibitory antibody titre greater than equal to 0.6 Bethesda unit (BU) at two consecutive tests performed at the central laboratory and also tested positive for nonacog beta pegol binding antibodies.
Haemostatic effect during surgery was evaluated immediately after surgery (last stitch) using a fourpoint response scale: \- Four-point response scale: Excellent, good, moderate, poor. The evaluation was done by the surgeon, anaesthesiologist and/or investigator based on experience as follows: 1. Excellent: Better than expected/predicted in this type of procedure. 2. Good: As expected in this type of procedure. 3. Moderate: Less than optimal for the type of procedure but haemostatic response maintained without change of treatment regimen. 4. Poor: Bleeding due to inadequate therapeutic response with adequate dosing, change of regimen required.
Inhibitors were analysed with either the Nijmegen modified factor IX Bethesda assay or a heat/cold Nijmegen modified factor IX Bethesda assay. Number of participants who developed inhibitory antibodies against factor IX are reported.
The primary endpoint was incidence of inhibitors against coagulation factor nine (FIX) defined as titre ≥0.6 Bethesda unit (BU). Number of subjects who developed inhibitors against FIX are reported.
Inhibitors were analysed with either the Nijmegen modified factor IX Bethesda assay or a heat/cold Nijmegen modified factor IX Bethesda assay. Number of subjects who developed inhibitory antibodies against factor IX are reported.
| Arm | Type | Description |
|---|---|---|
| Arm A - Nonacog beta pegol (On-demand/Prophylaxis) | EXPERIMENTAL | Participants on on-demand treatment for 28 weeks, thereafter prophylactic treatment |
| Arm B - Nonacog beta pegol (Prophylaxis) | EXPERIMENTAL | Participants on prophylactic treatment only |
| 50 EDs (exposure days) | EXPERIMENTAL | - |
| Patients enrolled in trial | EXPERIMENTAL | New patients will be included as well as transferred patients from Paradigm™ 2 or Paradigm™ 4 trial |
| NNC-0156-000-0009 | EXPERIMENTAL | - |
| Prophylaxis, high dose (once weekly) | EXPERIMENTAL | - |
| Prophylaxis, low dose (once weekly) | EXPERIMENTAL | - |
| On-demand | EXPERIMENTAL | - |
| Prophylaxis, high dose (every second week) | EXPERIMENTAL | - |
| Prophylaxis, high dose (trial duration 52 weeks) | EXPERIMENTAL | - |
| Prophylaxis, low dose (trial duration 52 weeks) | EXPERIMENTAL | - |
| On-demand (trial duration 28 weeks) | EXPERIMENTAL | - |
| 25U/kg | EXPERIMENTAL | - |
| 50U/kg | EXPERIMENTAL | - |
| 100U/kg | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| Nonacog beta pegol | DRUG | Nonacog beta pegol is administered as intravenous injections. Participants will receive nonacog beta pegol as prophylaxis, as on-demand for treatment of bleeding episodes and in relation to surgery. |
Inclusion Criteria: * Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial. * Male Chinese patient with moderate to severe congenital haemophi...
Nonacog beta pegol is used for the treatment and prevention of bleeding in people with haemophilia B, a congenital bleeding disorder. It is being developed for both previously treated and previously untreated patients, including children, and has been studied in a Chinese patient population as well.
Nonacog beta pegol targets coagulation factor IX, encoded by the F9 gene. It is an exogenous protein designed to supply the clotting factor that is missing or deficient in haemophilia B, thereby supporting blood coagulation and helping to control or prevent bleeding episodes.
Nonacog beta pegol is developed by Novo Nordisk A/S, which trades under the ticker NVO. Novo Nordisk is the sponsor of the clinical program studying the drug in haemophilia B and related congenital bleeding disorders.
Nonacog beta pegol is in Phase 3 clinical development. It is an investigational agent and has been evaluated across six completed Phase 3 trials, with no active trials currently listed. It has not been established as approved for marketing in the information available.
Six completed Phase 3 trials have studied nonacog beta pegol, including NCT05365217 in Chinese patients with haemophilia B, NCT02141074 in previously untreated patients, NCT01467427 in previously treated children, and NCT01395810, a long-term extension study. Total enrollment across the program is 255 participants.
Yes, nonacog beta pegol is also known as N9-GP and has been studied under the code NNC-0156-0000-0009. These names refer to the same investigational factor IX therapy developed by Novo Nordisk for haemophilia B.