Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Recombinant Factor VIII · 5 trials · 3 indications
Annualized number (mean +/- standard deviation) of total bleeds that occurred within 48 hours after all prophylaxis infusions (Part A: 6 months and at least 50 exposure days \[EDs\]; Part B: at least 50 EDs or until inhibitor development) was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds (only treated bleeds were classified as spontaneous or trauma), untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other').
To examine the Pharmacokinetic (PK) characteristics of BAY 81-8973 and ensure that the new drug is similar to Kogenate FS. All results are based on the chromogenic assay.
To examine the PK characteristics of BAY81-8973 and ensure that the new drug is similar to Kogenate FS. All results are based on the chromogenic assay.
The annualized number of bleeds experienced by participants
| Arm | Type | Description |
|---|---|---|
| PTPs 0-12 years | EXPERIMENTAL | Previously treated patients (PTPs) aged below 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (EDs) in main study - Part A. Participants having reached at least 50 EDs in main study - Part A were offered participation in an open label extension study (optional). Participants who transitioned from main study - Part A to the extension study received BAY81-8973, 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part A and extension study). |
| PUPs/MTPs 0-<6 years | EXPERIMENTAL | Previously untreated patients (PUPs) or minimally treated patients (MTPs, patients who had no more than 3 exposure days (EDs) with any FVIII product) received BAY81-8973 15-50 IU/kg at least 1x/week for at least 50 EDs or until inhibitor development in main study - Part B. Participants having reached at least 50 EDs in main study - Part B were offered participation in an open label extension study and received BAY81-8973 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part B and extension study); participants who developed an inhibitor in main study - Part B were offered participation in open label extension study and received Immune Tolerance Induction (ITI) treatment with BAY81-8973 until successful eradication of the inhibitor, or until failure, for approximately 18 months. |
| Arm 1: Recombinant Factor VIII (BAY81-8973) then Kogenate FS | EXPERIMENTAL | Part A - Arm 1: Participants first received one single intravenous (IV) injection of BAY81-8973 50 IU/kg, then 1 single IV injection of Kogenate FS (BAY14-2222) 50 IU/kg with a wash-out period of at least 2-3 days in between |
| Arm 2: Kogenate FS then Recombinant Factor VIII (BAY81-8973) | EXPERIMENTAL | Part A - Arm 2: Participants first received one single intravenous (IV) injection of Kogenate FS (BAY14-2222) 50 IU/kg, then 1 single IV injection of BAY81-8973 50 IU/kg with a wash-out period of at least 2-3 days in between |
| Arm 3: Recombinant Factor VIII by CS/EP then by CS/ADJ | EXPERIMENTAL | Part B - Arm 3: Participants received IV injection of BAY81-8973 at 20-50 IU/kg 2-3 times per week with BAY81-8973 measured by Chromogenic Substrate Assay Potency Per European Pharmacopeia for 6 months and then crossed over to study drug measured by Chromogenic Substrate Assay/Adjusted to Label Potency for 6 months |
| Arm 4: Recombinant Factor VIII by CS/ADJ then by CS/EP | EXPERIMENTAL | Part B - Arm 4:. Participants received IV injection of BAY81-8973 at 20-50 IU/kg 2-3 times per week with BAY81-8973 measured by Chromogenic Substrate Assay/Adjusted to Label Potency for 6 months and then crossed over to study drug measured by Chromogenic Substrate Assay Per European Pharmacopeia for 6 months |
| Arm 5: Recombinant Factor VIII by CS/EP | EXPERIMENTAL | Part C - Arm 5: Participants received a loading dose of approximately 50 IU/kg of BAY 81-8973 before the first surgical incision followed by further treatment with BAY 81-8973 according to surgical requirements for up to 3 weeks |
| Recombinant Factor VIII prophylaxis treatment | EXPERIMENTAL | Participants received 25 IU/kg of Recombinant Factor VIII (Kogenate FS, BAY14-2222) intravenously (IV), 3 times per week. Dose escalation steps by 5 IU/kg (to 30 IU/kg or 35 IU/kg maximum) for patients exhibiting a bleeding frequency of 12 bleeding episodes per year or greater. |
| Recombinant Factor VIII on-demand treatment | EXPERIMENTAL | Participants received Recombinant Factor VIII (Kogenate FS, BAY14-2222) IV for bleeds in accordance with package insert instructions and study physician recommendations. |
| Arm 1 | EXPERIMENTAL | - |
| Arm 2 | EXPERIMENTAL | - |
| Arm 3 | ACTIVE_COMPARATOR | - |
| Arm 4 | ACTIVE_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| Recombinant Factor VIII (Kovaltry, BAY81-8973) | BIOLOGICAL | Main study: 25-50 IU/kg at least 2x/week for 6 months and at least 50 EDs, IV infusion; Extension study: 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part A and extension study), IV infusion. Exposure day (ED): An ED is a unit of time (1 day) in which replacement treatment of Hemophilia is given to a patient. |
| Recombinant Factor VIII (BAY81-8973) | BIOLOGICAL | Single dose of BAY81-8973 crossed over to single dose of Kogenate FS |
| Recombinant Factor VIII (Kogenate FS, BAY14-2222) | BIOLOGICAL | Single dose of Kogenate FS crossed over to Single dose of BAY81-8973 |
| Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980) | BIOLOGICAL | Low dose of BAY 79-4980 \[13mg of liposomes/kg\] then cross over to rFVIII-FS (35 IU/kg reconstituted in 2.5 mL WFI / 1000 IU). |
Inclusion Criteria: * Male * PTPs (previously treated patients): aged \<= 12 years * PUPs (previously untreated patients) / MTPs (minimally treated patients): aged \< 6 years * Severe hemophilia A defined as \< 1% FVIII concentration (FVIII:C) * PTPs: \>= 50 exposure days (EDs) with any FVIII conce...
Recombinant Factor VIII is used for hemophilia A, a bleeding disorder in which the blood does not clot properly. It is also indicated for blood coagulation disorders. The drug is being developed by Bayer AG and is currently in Phase 3 clinical trials for these conditions.
Recombinant Factor VIII targets the missing or defective clotting factor VIII in patients with hemophilia A. By replacing this deficient protein, the drug helps restore normal blood clotting and reduces bleeding episodes. It is a recombinant form of the human factor VIII protein.
Recombinant Factor VIII is being developed by Bayer AG, a German pharmaceutical company traded on the OTC market under the ticker BAYRY. The drug is currently in Phase 3 clinical development for the treatment of hemophilia A and related blood coagulation disorders.
Recombinant Factor VIII is in Phase 3 clinical development. It is an investigational drug and has not yet been approved by regulatory authorities. The company has completed three clinical trials, including two Phase 3 studies and two Phase 1 studies, with a total enrollment of 129 participants.
Recombinant Factor VIII has been studied in several completed clinical trials. NCT00623480 evaluated secondary prophylaxis in severe hemophilia A patients. NCT01029340 assessed efficacy and safety of a new full-length recombinant human FVIII. NCT00629837 and NCT01653639 were Phase 1 pharmacokinetic studies. All trials enrolled male patients aged 12 years or older.
Recombinant Factor VIII is the same drug as BAY 79-4980 and BAY14-2222, which are code names used in clinical trials. NCT00629837 studied BAY 79-4980, and NCT01653639 studied BAY14-2222. These names refer to the same recombinant factor VIII product developed by Bayer.