Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
rFIXFc · 3 trials · 2 indications
Development of an inhibitor was defined as an inhibitor test result of \>= 0.60 Bethesda units per milliliter (BU/mL) that was confirmed by a second test result of \>=0.60 BU/mL from a separate sample, drawn 2 to 4 weeks after the date when the original sample was drawn, with both tests performed by the central laboratory using Nijmegen-modified Bethesda assay.
An inhibitor test result greater than or equal to (\>=)0.6 Bethesda units per milliliter (BU/mL), confirmed on 2 separate samples drawn 2 to 4 weeks apart, was considered positive. Both tests were to be performed by the central laboratory using the Nijmegen-modified Bethesda Assay. Data was summarized by treatment regimen for participants from Study 998HB102 and by age cohort (\<6 years and 6 to \<12 years old) and treatment regimen for participants from Study 9HB02PED per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
| Arm | Type | Description |
|---|---|---|
| Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) | EXPERIMENTAL | Participants received rFIXFc intravenous (IV) injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 International Units per kilogram (IU/kg) weekly until a participant reached at least 50 exposure days (ED=24-hour period in which greater than or equal to (\>=1) injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX (FIX) levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR. |
| On-Demand | EXPERIMENTAL | The individual dose of rFIXFc to treat bleeding episodes will be based on participant's clinical condition, type and severity of the bleeding event, and if indicated, Factor IX peak (recovery) levels. |
| Prophylaxis | EXPERIMENTAL | Weekly prophylaxis, individualized prophylaxis or personalized prophylaxis available. |
| rFIXFc | EXPERIMENTAL | Six intravenous (IV) dose levels, 1, 5, 12.5, 25, 50, and 100 IU/kg |
| Name | Type | Description |
|---|---|---|
| rFIXFc | BIOLOGICAL | Adjustments to the dose and interval of rFIXFc was made in this study based on investigator discretion using available pharmacokinetic (PK) data, subsequent FIX trough and peak levels, level of physical activity, and bleeding pattern, in accordance with local standards of care for a prophylactic regimen. There was an option to start study dosing as episodic treatment (on-demand). |
Key Inclusion Criteria: * Weight \>=3.5 kilogram at the time of informed consent. * Severe hemophilia B was defined as less than or equal to (\<=)2 International Units per deciliter (IU/dL) (\<=2 percent \[%\]) endogenous FIX documented in the medical record or as tested during the Screening Period...
rFIXFc is an investigational therapy for hemophilia B, including severe hemophilia B. It is being developed to prevent and treat bleeding episodes in patients with this condition. The drug is currently in clinical development and has not been approved by regulatory authorities.
rFIXFc is being developed by Sanofi, a global biopharmaceutical company traded on the stock exchange under the ticker SNY. Sanofi is conducting clinical trials to evaluate the safety and efficacy of rFIXFc in patients with hemophilia B.
rFIXFc is in Phase 3 clinical development. It has completed Phase 3 trials, including studies in previously treated patients and previously untreated patients with severe hemophilia B. The drug remains investigational and is not yet approved for commercial use.
rFIXFc has been studied in clinical trials including NCT00716716, a Phase 1/2a study in hemophilia B patients, and NCT01425723, a Phase 3 long-term safety and efficacy study in previously treated patients with severe hemophilia B. Another Phase 3 trial, NCT02234310, evaluated rFIXFc in previously untreated males with severe hemophilia B.
rFIXFc is also referred to as FIXFc in clinical trial documentation. The drug is a recombinant factor IX Fc fusion protein being investigated for the treatment of hemophilia B. Both names refer to the same investigational therapy developed by Sanofi.