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Also known as Alprolix, recombinant factor IX fusion protein, BIIB029, FIXFc, recombinant coagulation factor IX Fc fusion protein, coagulation factor IX (recombinant) Fc fusion protein
rFIXFc · 4 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 ≥ 0.6 Bethesda units (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. Incidences were summarized for any positive inhibitor for participants with ≥ 50 exposure days (EDs) to rFIXFc. In addition, the incidence for all participants, regardless of their EDs to rFIXFc, was also summarized. An exact 95% CI for the proportion of participants with a confirmed inhibitor was calculated using the Clopper-Pearson exact method for a binomial proportion.
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. |
| rFIXFc Prophylaxis | EXPERIMENTAL | At Baseline and at Day 1, participants receive a single intravenous (IV) injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg will be administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated. |
| 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). |
| FIX | DRUG | Vials of prestudy FIX (provided by the participants) were combined as needed, based on the nominal labeled potency (e.g., 250 IU, 500 IU, and 1000 IU), to achieve the participant's calculated dose. |
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...
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 |
rFIXFc is used to prevent and treat bleeding episodes in people with hemophilia B, including severe hemophilia B. It is a recombinant factor IX therapy developed for males with this inherited bleeding disorder, studied in previously untreated and previously treated patients across pediatric and adult populations.
rFIXFc is developed by Sanofi, which trades under the ticker SNY. The company conducted the clinical development program for the therapy across multiple Phase 3 studies in hemophilia B.
rFIXFc has completed Phase 3 clinical development. Three Phase 3 trials have been completed, along with an earlier Phase 1/2a study. It is an investigational therapy that has been studied in hemophilia B and severe hemophilia B.
rFIXFc has been studied in completed trials including NCT02234310 in previously untreated males with severe hemophilia B, NCT01440946 in pediatric patients with hemophilia B, NCT01425723 for long-term safety and efficacy in previously treated participants, and NCT00716716, a Phase 1/2a study.
Yes, rFIXFc is also known as BIIB029, a recombinant coagulation factor IX Fc fusion protein. The name BIIB029 appears in the pediatric Phase 3 trial NCT01440946, which studied the same therapy in previously treated pediatric participants with hemophilia B.