Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
BENEFIX · 3 trials · 1 indication
Investigator Hemostatic Efficacy Assessment was based on response of bleeding episodes to BeneFIX treatment on 4-point rating scale: Excellent(1): definite pain relief or improvement in signs of bleeding starting within 8 hrs after infusion, with no additional infusion; Good(2): definite pain relief or improvement in signs of bleeding starting within 8 hrs or following infusion; Moderate(3): probable or slight improvement starting after 8 hours following infusion; No Response(4): no improvement at all between infusions or during 24 hour interval following an infusion, or condition worsens.
Investigator Hemostatic Efficacy Assessment was based on response of bleeding episodes to BeneFIX treatment on 4-point rating scale: Excellent(1): definite pain relief or improvement in signs of bleeding starting within 8 hrs after infusion, with no additional infusion; Good(2): definite pain relief or improvement in signs of bleeding starting within 8 hrs or following infusion; Moderate(3): probable or slight improvement starting after 8 hours following infusion; No Response(4): no improvement at all between infusions or during 24 hour interval following an infusion, or condition worsens.
Incidence of FIX inhibitor was defined as any result determined as positive at local laboratory, and confirmed at central laboratory with Nijmegen assay result \>=0.6 Bethesda Unit (BU). Incidence was stratified by participant exposure history - Minimally Treated Patients (MTPs): those who had received at least one prior FIX infusion, and \<= 100 documented Exposure Days (EDs); while Previously Treated Patients (PTPs): those who had received \>100 documented prior EDs. When number of prior EDs for an individual was not known to be at least 100, participants were included in the MTP population.
Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration of a drug. Vss is the apparent volume of distribution at steady-state.
Linear regression of the log linear concentration time curve. Only those data points judged to describe the terminal log linear decline were used in the regression.
AUMCinf/AUCinf, where AUMCinf is the area under the first moment curve from time 0 extrapolated to infinite time, calculated using the linear/log trapezoidal method.
Plasma decay half-life is the time measured for the plasma concentration to decrease by one half.
CL is a quantitative measure of the rate at which a drug substance is removed from the body.
Incremental recovery: Increase in circulating increase in FIX activity for every IU of BeneFIX administered per kg of body weight.
| Arm | Type | Description |
|---|---|---|
| Benefix | EXPERIMENTAL | Subjects received on-demand treatments with BeneFIX over a 6-month (calendar day) period. |
| Name | Type | Description |
|---|---|---|
| Benefix | BIOLOGICAL | BeneFIX for on-demand treatment of bleeding episodes were according to investigator prescription. FIX recovery was assessed by determining the FIX activity (FIX:C) levels in individual subjects. BeneFIX dosage for recovery assessments: single 75 IU/kg (±5 IU/kg) IV bolus infusion. |
Inclusion Criteria: * Subjects equal or more than 6 years of age with mild, moderate or severe hemophilia B (FIX activity: more than 5%, 1-5%, or less than 1%, respectively) * Subjects with previous exposure to FIX replacement therapy * If HIV positive, documented CD4 count more than 200/µL within ...
BENEFIX is used for hemophilia B, a bleeding disorder caused by a deficiency in clotting factor IX. It is a recombinant factor IX product developed by Pfizer, Inc. (NYSE: PFE) for the treatment of this condition. The drug is administered to help control and prevent bleeding episodes in patients with hemophilia B.
BENEFIX targets factor IX, a clotting protein that is deficient or defective in patients with hemophilia B. By providing recombinant factor IX, BENEFIX replaces the missing protein, helping to restore normal blood clotting. This mechanism addresses the underlying cause of the bleeding disorder.
BENEFIX is developed and manufactured by Pfizer, Inc., a global biopharmaceutical company traded on the New York Stock Exchange under the ticker symbol PFE. Pfizer is responsible for the clinical development and commercialization of this recombinant factor IX therapy for hemophilia B.
BENEFIX has completed Phase 3 clinical trials for hemophilia B. Three trials have been completed, including two Phase 3 studies and one Phase 1 study. The drug is not currently in active clinical development, as all trials are completed. It is an investigational product that has undergone clinical evaluation.
BENEFIX has been studied in three completed clinical trials. NCT00037557 was a Phase 3 study in severe hemophilia B in the United States with 20 participants. NCT00866606 was a Phase 3 on-demand treatment study in Chinese subjects with 35 participants. NCT02213250 was a Phase 1 pharmacokinetic study in male Chinese subjects with hemophilia B.
BENEFIX is a brand name for recombinant factor IX, a clotting factor used to treat hemophilia B. It is a genetically engineered version of the naturally occurring factor IX protein. The drug is designed to replace the missing or defective factor IX in patients with this bleeding disorder.