Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as BIVV001 (Low Dose), Efanesoctocog alfa, ALTUVIIIO
BIVV001 · 6 trials · 3 indications
The number of participants with the occurrence of inhibitor development (neuatralizing antibodies detected against factor VIII \[FVIII\]) as determined via the Nijmegen modified Bethesda assay.
Inhibitor development was defined as an inhibitor result of greater than or equal to (\>=0.6) Bethesda units (BU/mL) that was confirmed by a second test result from a separate sample, drawn 2 to 4 weeks following the date when the original sample was drawn. Both tests must have been performed by the central laboratory using the Nijmegen modified Bethesda assay.
ABR is annualized number of treated bleeding episodes (BE) per participant per year. Treated Bleeding episode: any occurrence of hemorrhage that required administration of BIVV001. It started from 1st sign of bleed and ended no more than 72 hours after last injection to treat bleeding episode, any subsequent bleeding at same location/injections administered less than or equal to (\<=) 72 hours apart from previous injection were considered same bleeding episode. ABR=number of treated BE during efficacy period (EP)/number of days during EP\*365.25. EP reflects the sum of all intervals of time during which participants were treated with BIVV001 according to the study arms and treatment regimens. This outcome measure (OM) presents estimated results (i.e., results estimated by fitting negative binomial \[NB\] regression model on data collected during EP).
ABR is annualized number of treated bleeding episodes per participant per year. Treated Bleeding episode: any occurrence of hemorrhage that required administration of BIVV001. It started from 1st sign of bleed and ended no more than 72 hours after last injection to treat bleeding episode, any subsequent bleeding at same location/injections administered \<=72 hours apart from previous injection were considered same bleeding episode. ABR=number of treated bleeding episodes during EP/number of days during EP\*365.25. EP reflects the sum of all intervals of time during which participants were treated with BIVV001 according to the study arms and treatment regimens. This OM presents observed results (i.e., descriptive statistics values based on the data which was collected during EP).
AE was defined as any untoward medical occurrence in a participant who received study drug and did not necessarily had a causal relationship with the treatment. TEAE is defined as any AE that begins on or after the single dose of Advate but before the single dose of BIVV001. Serious AE (SAE) was defined as any AE resulting in death, immediate risk of death, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity, or a congenital/anomaly/birth defect, or any other medically important event.
AE was defined as any untoward medical occurrence in a participant who received study drug and did not necessarily had a causal relationship with the treatment. TEAE is defined as any AE that begins on or after the study treatment (BIVV001) and within 28 days after BIVV001 administration. SAE was defined as any AE resulting in death, immediate risk of death, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity, or a congenital/anomaly/birth defect, or any other medically important event.
Number of Participants with Clinically Significant Abnormalities in Laboratory tests (including hematology, clinical chemistry, urinalysis, and coagulation and thrombosis markers) was reported.
Number of participants with clinically significant abnormalities (including hematology, clinical chemistry, urinalysis, and coagulation and thrombosis markers) was reported.
Development of an inhibitor was defined as a neutralizing antibody value of greater than or equal to (\>=) 0.6 Bethesda units per milliliter (BU/mL) identified and confirmed by a second test on an independent sample, collected within 2 to 4 weeks of the first positive sample, with both tests performed by the central laboratory using Nijmegen-modified Bethesda assay.
| Arm | Type | Description |
|---|---|---|
| Arm A: Previously treated in BIVV001 study | EXPERIMENTAL | This arm includes participants who have completed study EFC16293 or study EFC16295, participants who have completed Arm B or Arm C of this study (LTS16294) and roll over into Arm A, and participants who have completed any other potential BIVV001 study. Participants in this arm will continue receiving BIVV001 prophylaxis treatment once weekly (QW) for a total of 100 exposure days (EDs) cumulative from the parent study and this study. Participants will have the opportunity to continue in this study for up to 4 years, unless BIVV001 is commercially available in their applicable participating country. |
| Arm B: Newly initiated (China Only) in BIVV001 | EXPERIMENTAL | This arm includes Chinese participants of any age who will be newly initiated on BIVV001 prophylaxis treatment once-weekly (QW) for 52 weeks. After 52 weeks of treatment in this arm B, participants will be able to roll over into arm A. |
| Arm C: Newly initiated in BIVV001 with planned major surgery | EXPERIMENTAL | This arm includes participants of any age who will be newly initiated on BIVV001 prophylaxis treatment once-weekly (QW) and will undergo planned major surgery after at least 6 initial EDs with BIVV001, and within 26 weeks from Day 1. After 52 weeks of treatment in arm C, participants will be able to roll into arm A. |
| BIVV001: Participants aged <6 Years | EXPERIMENTAL | Participants aged less than (\<) 6 years received BIVV001 at a dose of 50 international units per kilogram (IU/kg) intravenous (IV) injection once-weekly (QW) prophylaxis for 52 weeks. |
| BIVV001: Participants aged 6 to <12 Years | EXPERIMENTAL | Participants aged 6 to \<12 years received BIVV001 at a dose of 50 IU/kg IV injection QW prophylaxis for 52 weeks. |
| Arm A: Prophylaxis | EXPERIMENTAL | Participants who were on a prophylaxis treatment with a FVIII product prior to study EFC16293 including participants who rolled over from study OBS16221, received BIVV001 50 international units per kilogram (IU/kg) intravenous (IV) injection once-weekly (QW) for 52 weeks in the current study. Study OBS16221 participants with 6 months historical data on prophylaxis treatment with a marketed FVIII product prior to enrollment were analyzed as a subgroup (named as: Arm A: Historical Prophylaxis \[OBS16221\]) in the outcome measure analysis. |
| Arm B: On-Demand Then Prophylaxis | EXPERIMENTAL | Participants who were on an on-demand treatment regimen with a FVIII product prior to study EFC16293, including participants who rolled over from study OBS16221, received BIVV001 50 IU/kg IV injection as an on-demand treatment (as needed for the treatment of bleeding episodes) from Week 1 to Week 26 in current study. At Week 26, participants in Arm B were switched to prophylaxis treatment, and received BIVV001 50 IU/kg, IV injection QW until Week 52. |
| efanesoctocog alfa (BIVV001) | EXPERIMENTAL | Each participant will be sequentially dosed with three single intravenous (IV) doses of first rFVIII (Advate®), second Polyethylene Glycol (PEG)-rFVIII (Adynovi® or Adynovate®), and lastly, BIVV001 |
| Low Dose Cohort: Advate 25 IU/kg Then BIVV001 25 IU/kg | EXPERIMENTAL | Participants received a single intravenous (IV) dose of Advate 25 international units per kilogram (IU/kg) on Day 1 of Advate treatment period (3 days) followed by a single IV dose of BIVV001 25 IU/kg in BIVV001 treatment period (BTP) (28 days). Advate treatment period (ATP) consisted of a washout of at least 72 hours which was started from the time of Advate dosing. |
| High Dose Cohort: Advate 65 IU/kg Then BIVV001 65 IU/kg | EXPERIMENTAL | Participants received a single IV dose of Advate 65 IU/kg on Day 1 of ATP (4 days) followed by a single IV dose of BIVV001 65 IU/kg in BTP (28 days). ATP consisted of a washout of at least 96 hours which was started from the time of Advate dosing. |
| Name | Type | Description |
|---|---|---|
| efanesoctocog alfa (BIVV001) | DRUG | Pharmaceutical form:Solution for Injection Route of administration: Intravenous |
| Efanesoctocog alfa | DRUG | Solution for injection Intravenous |
| Octocog alfa | DRUG | Solution for injection Intravenous |
| Rurioctocog alfa pegol | DRUG | Solution for injection Intravenous |
| Advate (Low Dose) | BIOLOGICAL | Participants received a single IV low dose of Advate 25 IU/kg. |
| Advate (High Dose) | BIOLOGICAL | Participants received a single IV high dose of Advate 65 IU/kg. |
| BIVV001 (Low Dose) | BIOLOGICAL | Participants received single IV low dose of BIVV001 25 IU/kg. |
| BIVV001 (High Dose) | BIOLOGICAL | Participants received single IV high dose of BIVV001 65 IU/kg. |
Inclusion criteria : * For participants rolling over into Arm A * Participants who have completed the studies EFC16923, EFC16925, Arm B or Arm C of the current study, or any other potential BIVV001 study. * Male or Female * For participants new to BIVV001 (Arm B and C) * Participants who ha...
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 |
BIVV001 is an investigational therapy being developed for the treatment of Hemophilia A, a bleeding disorder caused by deficient or defective clotting factor VIII. It is designed to address the underlying factor VIII deficiency in previously treated adults with severe Hemophilia A. It has been studied in a Phase 1 clinical trial.
BIVV001 targets factor VIII, the clotting protein that is deficient or dysfunctional in Hemophilia A. The molecule is a recombinant factor VIII Fc - Von Willebrand Factor - XTEN fusion protein, meaning it combines factor VIII with Fc, von Willebrand factor, and XTEN components to extend its presence in the body.
BIVV001 is being developed by Sanofi, which trades on the New York Stock Exchange under the ticker SNY. Sanofi is the sponsor of the clinical program evaluating BIVV001 in Hemophilia A.
BIVV001 is in Phase 1 clinical development. It is an investigational agent and is not approved for commercial use. The Phase 1 program has been completed, and the drug remains in early-stage clinical evaluation for Hemophilia A.
BIVV001 has been evaluated in the Phase 1 EXTEN-A trial, registered as NCT03205163. That study assessed the safety, tolerability, and pharmacokinetics of a single intravenous injection of BIVV001 in previously treated adults with severe Hemophilia A. It enrolled 16 participants across the United States and Japan and has been completed.
BIVV001 (Low Dose) is an alternative name for BIVV001, so both names refer to the same investigational agent. The low dose designation reflects a dose level studied within the clinical program rather than a separate drug candidate. BIVV001 is a recombinant factor VIII Fc - Von Willebrand Factor - XTEN fusion protein.