Drugs / Efanesoctocog alfa
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed hemophilia B leyden 1 milestone | |||||
| Filed | EU (EMA) | — | hemophilia B leyden | 2023-05-19 | “Sobi today announced that the European Medicines Agency (EMA) has accepted and validated a marketing authorisation application for efanesoctocog alfa” sobi.com ↗ |
| Approved Indication not stated 1 milestone | |||||
| Approved | EU (EMA) | Altuvoct | — | 2024-06-17 | europa.eu ↗ |
Trials 10
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 12 trials | ||||||
| Phase 1 | NCT05042440 | Aug → Nov 2021 | hemophilia A | Sanofi | Completed | No outcome recorded |
| Phase 1 | NCT04770935 | May 2021 → Dec 2022 | von Willebrand disease (hereditary or acquired) | Bioverativ, a Sanofi company | Completed | No outcome recorded |
| Phase 35 trials · 2 met primary | ||||||
| Phase 3 | NCT06716814 | Mar 2025 → Mar 2027 expected | hemophilia A | Swedish Orphan Biovitrum | Recruiting | No outcome recorded |
| Phase 3 | NCT05817812 | Jul 2023 → Jun 2026 | severe hemophilia A | Swedish Orphan Biovitrum | Completed | No outcome recorded |
| Phase 3 | NCT04644575 | Feb 2021 → Jan 2027 expected | hemophilia A | Bioverativ, a Sanofi company | Active not recruiting | No outcome recorded |
| Phase 3 | NCT04759131 | Feb 2021 → Jan 2023 | hemophilia A | Bioverativ, a Sanofi company | Completed | Met primary |
| Phase 3 | NCT04161495 | Nov 2019 → Feb 2022 | hemophilia A | Bioverativ, a Sanofi company | Completed | Met primary |
| Phase 43 trials | ||||||
| Phase 4 | NCT06941870 | Sep 2025 → Oct 2027 expected | coagulation protein disease | Sanofi | Recruiting | No outcome recorded |
| Phase 4 | NCT06940830 | May 2025 → Dec 2028 expected | hemophilia A | Swedish Orphan Biovitrum | Active not recruiting | No outcome recorded |
| Phase 4 | NCT06752850 | Dec 2024 → Nov 2026 expected | hemophilia A | Swedish Orphan Biovitrum | Active not recruiting | No outcome recorded |
| Comparator or background therapy1 trial | ||||||
| Phase 1 | NCT06579144 Comparator | Feb 2025 → May 2026 | hemophilia A | Swedish Orphan Biovitrum | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 29
| Date | Issuer | Release |
|---|---|---|
| 2024-07-18 | Sobi, Inc. | Results NEJM publishes ALTUVOCT® phase 3 XTEND-Kids results highlighting treatment potential for children with severe haemophilia A sobi.com ↗
The study demonstrated that fixed-dose once-weekly prophylaxis with ALTUVOCT, a new class of high-sustained factor VIII therapy, provided highly effective bleed protection in children. |
| 2024-07-17 | Sanofi | Results Press Release: NEJM publishes ALTUVIIIO XTEND-Kids phase 3 data supporting its potential to transform the treatment landscape for children with severe hemophilia A sanofi.com ↗
Full results from the XTEND-Kids phase 3 study published in The New England Journal of Medicine ( NEJM ) highlights the efficacy, safety, and pharmacokinetic profile of ALTUVIIIO [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein]. |
| 2024-06-21 | Sanofi | Results Press Release: ISTH: Sanofi advances leadership in hemophilia with new data for ALTUVIIIO and fitusiran sanofi.com ↗
In previously treated patients (≥12 years old) who had the completed the XTEND-1 (Arm A/B) trial, the mean annualized bleed rate (ABR) with ALTUVIIIO was 0.72 (standard deviation [SD])=1.26) for arm A and 0.42 (SD=0.89) for arm B. |
| 2024-06-19 | Sobi, Inc. | Regulatory European Commission grants Sobi® Marketing Authorisation for ALTUVOCT™ for treatment of haemophilia A sobi.com ↗
Sobi® today announced the European Commission has granted Marketing Authorisation for ALTUVOCT™ (efanesoctocog alfa), for the treatment and prevention of bleeds and perioperative prophylaxis in haemophilia A. |
| 2024-04-26 | Sobi, Inc. | Regulatory Sobi® receives positive CHMP opinion recommending approval of efanesoctocog alfa for once-weekly treatment of haemophilia A sobi.com ↗
Sobi® today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a positive opinion recommending approval of efanesoctocog alfa, for the treatment and prevention of bleeds and perioperative prophylaxis in haemophilia A. |
| 2023-11-20 | Sobi, Inc. | Results Sobi to present new data at ASH 2023 Annual Meeting sobi.com ↗
Once-Weekly Efanesoctocog Alfa Prophylaxis Provided High Sustained Factor VIII Activity Levels, Independent of Blood Group, in Children < 12 Years of Age with Severe Hemophilia A |
| 2023-09-25 | Sanofi | Regulatory Press Release: Once-weekly ALTUVIIIO® approved in Japan as a new class of factor VIII therapy for hemophilia A sanofi.com ↗
The Japanese Ministry of Health, Labor, and Welfare (MHLW) has granted marketing authorization for ALTUVIIIO ® [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein], a first-in-class, high-sustained factor VIII replacement therapy. |
| 2023-06-25 | Sobi, Inc. | Results Late-breaking efanesoctocog alfa data presented at ISTH demonstrates highly effective bleed protection in children with severe haemophilia A with once-weekly dosing sobi.com ↗
The oral presentation detailed results from the XTEND-Kids study and confirmed that efanesoctocog alfa met the primary endpoint, occurrence of inhibitor development, and secondary endpoints including annualised bleeding rate (ABR) |
| 2023-06-25 | Sanofi | Results Press Release: ALTUVIIIO® late-breaking data at ISTH demonstrates highly effective bleed protection in children with severe hemophilia A with once-weekly dosing sanofi.com ↗
These data confirm that a once-weekly 50 IU/kg dose of ALTUVIIIO provides highly effective bleed protection in both children and adults and can be used across clinical scenarios. |
| 2023-05-19 | Sobi, Inc. | Regulatory EMA validates marketing authorisation application for efanesoctocog alfa for treatment of haemophilia A sobi.com ↗
Sobi today announced that the European Medicines Agency (EMA) has accepted and validated a marketing authorisation application for efanesoctocog alfa, a new class of high-sustained FVIII developed for the treatment of people with haemophilia A of all age groups. |
| 2023-03-02 | Sobi, Inc. | Results Positive topline results from pivotal XTEND-Kids phase 3 study of efanesoctocog alfa in children under 12 years of age with haemophilia A sobi.com ↗
Sobi® and Sanofi today announced that the XTEND-Kids phase 3 pivotal study evaluating the safety, efficacy and pharmacokinetics of efanesoctocog alfa as once-weekly prophylaxis in previously treated patients under 12 years of age with severe haemophilia A met its primary endpoint. |
| 2023-03-02 | Sanofi | Results Press Release: Completed XTEND-Kids Phase 3 study strengthens potential of ALTUVIIIOTM to redefine expectations for treatment of children <12 years of age with hemophilia A sanofi.com ↗
No inhibitors were detected in this study. |
| 2023-02-23 | Sobi, Inc. | Regulatory FDA approves once-weekly efanesoctocog alfa, a new class of high-sustained factor VIII therapy for haemophilia A sobi.com ↗
Sobi® today announced that the US Food and Drug Administration (FDA) has approved efanesoctocog alfa [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl], a first-in-class, high-sustained factor VIII replacement therapy for adults and children with haemophilia A. |
| 2023-02-23 | Sanofi | Regulatory Press Release: FDA approves once-weekly ALTUVIIIO™, a new class of factor VIII therapy for hemophilia A that offers significant bleed protection sanofi.com ↗
The U.S. Food and Drug Administration (FDA) has approved ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl], previously referred to as efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy. |
| 2023-01-26 | Sobi, Inc. | Results NEJM publishes data demonstrating clinically meaningful prevention of bleeds and superior bleed protection for efanesoctocog alfa sobi.com ↗
These data demonstrate the clinical benefits of normal to near-normal factor activity levels (>40%) for the majority of the week achieved by once weekly efanesoctocog alfa dosing. |
| 2023-01-25 | Sanofi | Results Press Release: NEJM publishes once-weekly efanesoctocog alfa Phase 3 data demonstrating its potential to transform the treatment landscape for people with hemophilia A sanofi.com ↗ |
| 2022-08-30 | Sobi, Inc. | Regulatory FDA grants priority review to efanesoctocog alfa for people with haemophilia A sobi.com ↗
The US Food and Drug Administration (FDA) has accepted for priority review the Biologics License Application (BLA) for efanesoctocog alfa (BIVV001) for the treatment of haemophilia A, a rare and life-threatening bleeding disorder. |
| 2022-08-30 | Sanofi | Regulatory Press Release: FDA grants priority review to efanesoctocog alfa for people with hemophilia A sanofi.com ↗
The U.S. Food and Drug Administration (FDA) has accepted for priority review the Biologics License Application (BLA) for efanesoctocog alfa (BIVV001) for the treatment of hemophilia A, a rare and life-threatening bleeding disorder. |
| 2022-07-28 | Sanofi | Regulatory Press Release: Strong execution in Q2 drives full-year 2022 guidance upgrade and delivers rich R&D news flow in Immunology and Rare Disease sanofi.com ↗
Efanesoctocog alfa, the first factor VIII therapy to be granted FDA Breakthrough Therapy Designation |
| 2022-07-10 | Sobi, Inc. | Results Pivotal data demonstrate once-weekly efanesoctocog alfa provides superior bleed protection compared to prior factor prophylaxis sobi.com ↗
The study met the primary efficacy endpoint, with once-weekly efanesoctocog alfa prophylaxis providing clinically meaningful bleed protection for people with severe haemophilia A. |
| 2022-07-10 | Sanofi | Results Press Release: Pivotal data demonstrate once-weekly efanesoctocog alfa provides superior bleed protection compared to prior factor prophylaxis sanofi.com ↗
The study met the primary efficacy endpoint, with once-weekly efanesoctocog alfa prophylaxis providing clinically meaningful bleed protection for people with severe hemophilia A. |
| 2022-06-01 | Sobi, Inc. | Regulatory FDA grants efanesoctocog alfa Breakthrough Therapy designation for haemophilia A sobi.com ↗ |
| 2022-06-01 | Sanofi | Regulatory Press Release: FDA grants efanesoctocog alfa Breakthrough Therapy designation for hemophilia A sanofi.com ↗ |
| 2022-03-09 | Sobi, Inc. | Results Efanesoctocog alfa met primary and key secondary endpoints in pivotal study in haemophilia A, demonstrating superiority to prior factor prophylaxis treatment sobi.com ↗
Swedish Orphan Biovitrum AB (publ) (Sobi®) (STO:SOBI) and Sanofi (EURONEXT: SAN and NASDAQ: SNY) today announced positive top-line results from the pivotal XTEND-1 phase 3 study evaluating the safety, efficacy and pharmacokinetics of efanesoctocog alfa (BIVV001) in previously treated patients ≥12 years of age with severe haemophilia A. |
| 2022-03-09 | Sanofi | Results Press Release: Efanesoctocog alfa met primary and key secondary endpoints in pivotal study in hemophilia A, demonstrating superiority to prior factor prophylaxis treatment sanofi.com ↗ |
| 2021-11-04 | Sanofi | Results New data in blood cancers, hemophilia, and other hematological disorders to be presented at ASH 2021 sanofi.com ↗ |
| 2021-02-18 | Sanofi | Regulatory Efanesoctocog alfa granted FDA Fast Track Designation for treatment of hemophilia A sanofi.com ↗
The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) for efanesoctocog alfa, previously known as BIVV001 (rFVIIIFc-VWF-XTEN), in patients with hemophilia A. |
| 2020-09-10 | Sobi, Inc. | Results New England Journal of Medicine publishes positive final results from phase 1/2a study of BIVV001 in people with severe haemophilia A sobi.com ↗
The New England Journal of Medicine today published positive final results from the phase 1/2a trial evaluating the safety, tolerability and pharmacokinetics of BIVV001 (rFVIIIFc-VWF-XTEN) in adult patients with severe haemophilia A. |
| 2020-09-10 | Sanofi | Results New England Journal of Medicine publishes positive final results from Phase 1/2a study of BIVV001 in people with severe hemophilia A sanofi.com ↗
The New England Journal of Medicine today published positive final results from the Phase 1/2a trial evaluating the safety, tolerability and pharmacokinetics of BIVV001 (rFVIIIFc-VWF-XTEN) in adult patients with severe hemophilia A. |
All press releases naming this drug 32 releases
Evidence & citations 10 cited values
Every value below carries the sentence it was read from. 13 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | efanesoctocog alfa | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04770935 ↗ |
| Known as | Altuviiio | ChEMBL registry synonym — accepted as the source's own label CHEMBL5095275 ↗ |
| Known as | Altuvoct | ChEMBL registry synonym — accepted as the source's own label CHEMBL5095275 ↗ |
| Known as | BIVV001 | “Drug: efanesoctocog alfa (BIVV001)” NCT04759131 ↗3“efanesoctocog alfa (BIVV001)” NCT04161495 ↗ “efanesoctocog alfa (BIVV001)” NCT04644575 ↗ “efanesoctocog alfa (BIVV001)” NCT04770935 ↗ |
| Known as | Efanesoctocog alfa-ehtl | ChEMBL registry synonym — accepted as the source's own label CHEMBL5095275 ↗ |
| Known as | rFVIIIFc-VWF-XTEN | “Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001)” NCT04161495 ↗1“Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001)” NCT04759131 ↗ |
| Action | Restore | “first-in-class high-sustained factor VIII (FVIII) replacement therapy” PMID 41583536 ↗ Jan 2026 |
| Modality | Protein / enzyme biologic | “Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein” NCT04161495 ↗ |
| Route | Intravenous | “Route of administration: IV injection” NCT04161495 ↗ |
| Target | F8 | “Coagulation factor VIII exogenous protein” CHEMBL5095275 ↗ |