Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins · 1 trial · 1 indication
Percentage of treated participants who developed factor VIII inhibitors
| Arm | Type | Description |
|---|---|---|
| Single Arm - All Participants | EXPERIMENTAL | All subjects enrolled in the study who meet the eligibility criteria. |
| Name | Type | Description |
|---|---|---|
| Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM) | BIOLOGICAL | Treatment regimens were determined by the investigator, and may have been any combination of standard prophylaxis (25 to 50 IU/kg body weight, 3 to 4 times per week), investigator-determined prophylaxis, and/or on-demand treatment (dose selected by investigator). The treatment of bleeding episodes and perioperative management was at the discretion of the investigator and consistent with the institution's standard of care. For incremental recovery assessments, a single infusion at 50 +/- 5 IU/kg was to be given. Immune tolerance induction (ITI) therapy for subjects who developed factor VIII inhibitors was at the discretion of the investigator, based on the institution's guidelines or described in peer-reviewed literature, and was to be approved by the sponsor's medical director. rAHF-PFM was to be administered intravenously via bolus infusion, except for perioperative management when it may have been given either by continuous or bolus infusion. |
Inclusion Criteria: * The subject has severe or moderately severe hemophilia A as defined by a baseline factor VIII level \<= 2% of normal, as documented at screening * The subject is \< 6 years of age * The subject's legally authorized representative has provided written informed consent Exclusio...
Top 8 of 9 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 15 | PHASE3 | Concizumab, Mim8, Denecimig, NNC0442-0344 A, turoctocog alfa pegol |
| Sanofi SA Sponsored ADR | SNY | 5 | PHASE3 | Fitusiran, efanesoctocog alfa |
| Pfizer Inc. | PFE | 8 | PHASE3 | Fidanacogene elaparvovec, Marstacimab |
| BioMarin Pharmaceutical Inc. | BMRN | 2 | PHASE3 | Valoctocogene roxaparvovec |
| Hoffmann-La Roche | RHHBY | 8 | PHASE3 | Emicizumab, NXT007, SPK-8011QQ, SPK-8011, SPK-8016 |
| Regeneron Pharmaceuticals, Inc. | REGN | 2 | PHASE1 | REGV131, LNP1265 |
| Takeda Pharmaceutical Co. Ltd. Sponsored ADR | TAK | 2 | PHASE1 | AskBio009 |
| Bayer | BAYRY | 6 | - | Damoctocog-alfa-pegol |
It is used to treat hemophilia A, a bleeding disorder caused by a deficiency in clotting factor VIII. The product is a recombinant factor VIII therapy developed for patients with hemophilia A, including previously untreated patients. It is manufactured and formulated without added human or animal proteins.
It targets F9, also known as coagulation factor IX, and is classified as an exogenous protein. By supplying recombinant clotting factor activity, it aims to support the coagulation cascade in patients with hemophilia A, whose bleeding episodes result from deficient factor VIII activity.
It is developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. Takeda is the company responsible for the development of this recombinant factor VIII product for hemophilia A.
It is in Phase 3 clinical development for hemophilia A. The product is investigational and is not described as approved. One Phase 3 trial has been completed, and no trials are currently listed as active.
It has been studied in the completed Phase 3 trial NCT00157157, titled Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients. That trial enrolled 66 participants across the United States, Austria, Canada, France, Germany, Italy, Puerto Rico, Spain, Sweden, and the United Kingdom.
Yes. Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins is also known as rAHF-PFM. The abbreviation rAHF-PFM refers to the same recombinant factor VIII product developed by Takeda for hemophilia A, so searches using either name refer to the same asset.