Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as turoctocog alfa pegol (N8-GP), NNC 0129-0000-1003, N8-GP
turoctocog alfa pegol · 9 trials · 2 indications
Number of bleeding episodes per year (Annualised Bleeding Rate) data is reported. Annualised bleeding rate (ABR) is the number of bleeding episodes per year.
An adverse event (AE) was defined as any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease temporally associated with the use of a product, whether or not considered related to the product. All AEs mentioned here are treatment emergent adverse events (TEAEs). The TEAE is defined as an event reported from date of first trial product administration until end of the treatment visit (week 104) or follow-up visit if relevant (1 month after end of the treatment).
Number of participants with inhibitory antibodies against coagulation factor VIII (FVIII) was reported during the main and extension phase of the trial.
The number of participants with inhibitory antibodies against coagulation factor VIII (FVIII) ≥0.6 Bethesda units was presented.
Haemostatic effect during surgery was evaluated on a four-point response scale as 'none', 'moderate', 'good' and 'excellent'. This was assessed after completion of surgery (defined as "last stitch"). Excellent: Better than expected/predicted in this type of procedure. Good: As expected in this type of procedure. Moderate: Less than optimal for the type of procedure but haemostatic response maintained without change of treatment regimen. None: Bleeding due to inadequate therapeutic response with adequate dosing, change of regimen required.
All participants with neutralizing antibodies were included in the numerator and any participant with a minimum 50 exposure days plus any participant with inhibitory inhibitors was included in the denominator. A positive inhibitor test was defined as ≥0.6 bethesda unit (BU). Estimates are based on exact calculations for a binomial distribution. For the calculation of the 'inhibitor rate' the nominator included all participants with neutralising antibodies while the denominator included all participants with a minimum of 50 exposures plus any participant with less than 50 exposures but with neutralising inhibitors.
Annualised bleeding rate (ABR) is the number of bleeding episodes per year. This was assessed only for the prophylaxis treatment with N8-GP.
All participants with neutralizing antibodies were included in the numerator and any participant with a minimum 50 exposure days plus any participant with inhibitory inhibitors was included in the denominator. A positive inhibitor test was defined as ≥0.6 bethesda unit (BU). Estimates are based on exact calculations for a binomial distribution. For the calculation of the 'inhibitor rate' the nominator included all participants with neutralising antibodies while the denominator included all participants with a minimum of 50 exposures plus any participant with less than 50 exposures but with neutralising inhibitors.
ABR is the number of bleeding episodes per year. This was assessed only for the prophylaxis treatment with N8-GP.
All participants with neutralizing antibodies were included in the numerator and any participant with a minimum 50 exposure days plus any participant with inhibitory inhibitors was included in the denominator. A positive inhibitor test was defined as ≥0.6 bethesda unit (BU). Estimates are based on exact calculations for a binomial distribution. For the calculation of the 'inhibitor rate' the nominator included all participants with neutralising antibodies while the denominator included all participants with a minimum of 50 exposures plus any participant with less than 50 exposures but with neutralising inhibitors.
Annualised bleeding rate (ABR) is the number of bleeding episodes per year reported during the prophylactic treatment with N8-GP.
Count and % of Adverse events
| Arm | Type | Description |
|---|---|---|
| N8-GP prophylaxis | EXPERIMENTAL | All patients will receive prophylaxis with 50 IU/kg N8-GP every 4 days for a treatment period of at least 28 weeks (with the possibility of switching to twice-weekly dosing during the treatment period at the discretion of the investigator). |
| N8-GP, once weekly | EXPERIMENTAL | All participants will receive turoctocog alfa pegol (N8-GP) once weekly. |
| N8-GP, twice weekly | EXPERIMENTAL | All participants will receive N8-GP twice weekly. |
| N8-GP, three times weekly | EXPERIMENTAL | All participants will receive N8-GP three times weekly. |
| 50 EDs (exposure days) | EXPERIMENTAL | - |
| N8-GP | EXPERIMENTAL | - |
| Surgery | EXPERIMENTAL | - |
| Prophylaxis | EXPERIMENTAL | - |
| On-demand | EXPERIMENTAL | - |
| N8-GP s.c. | EXPERIMENTAL | - |
| N8-GP pivotal | EXPERIMENTAL | - |
| N8-GP commercial | ACTIVE_COMPARATOR | - |
| A | EXPERIMENTAL | - |
| B | EXPERIMENTAL | - |
| C | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| turoctocog alfa pegol (N8-GP) | DRUG | N8-GP will be injected into a vein (intravenous injections) every 4 days in at least 28 weeks |
| Turoctocog alfa pegol | DRUG | Turoctocog alfa pegol 75 IU/kg body weight will be administered once weekly as intravenous injections for a duration of 2 years. |
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 severe congenital haemophilia A with a...
Turoctocog alfa pegol is used for the prophylaxis and treatment of bleeding episodes in people with haemophilia A, a congenital bleeding disorder. It is a factor VIII medicine given to patients with this inherited clotting condition, including previously treated patients aged 12 years and older.
Turoctocog alfa pegol targets factor VIII, encoded by the F8 gene. It is an exogenous protein that supplies factor VIII activity, the clotting factor that is missing or deficient in haemophilia A, thereby supporting blood coagulation and helping to prevent or control bleeding episodes.
Turoctocog alfa 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 A and congenital bleeding disorder.
Turoctocog alfa pegol is in Phase 3 clinical development. It is an investigational agent and is not described as approved. Eight trials have been completed, including Phase 1 pharmacokinetic and safety studies and Phase 3 studies in haemophilia A, with no trials currently active.
Completed trials include NCT05082116, a Phase 3 study of prophylaxis and bleeding episode treatment in previously treated Chinese patients with haemophilia A, and NCT03528551, a Phase 3 study in haemophilia A. Phase 1 studies include NCT02994407, evaluating subcutaneous injection, and NCT02920398, a pharmacokinetic comparison.
Yes, turoctocog alfa pegol is also known as N8-GP. N8-GP is the development code used for this factor VIII therapy in Novo Nordisk's clinical program, so searches for either name refer to the same investigational haemophilia A treatment.