RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Australia), Priority Review (United States), Breakthrough Therapy (United States)

Structure/Sequence

Sequence Code 453117726

Source: *****

Sequence Code 453117729

Source: *****

Clinical Trials associated with Efanesoctocog alfa

NCT06941870

/ Not yet recruitingPhase 4

Prospective Interventional Study of Effectiveness of Efanesoctocog Alfa Prophylaxis on Synovial Hypertrophy in Patients With Hemophilia A

The objective of the study is to assess the improvement of synovial hypertrophy during the 12 months of efanesoctocog alfa prophylaxis once per week (QW) in joints with existing evidence of synovial hypertrophy in participants with hemophilia A.
The study duration for each participant is approximately 12 months.

Start Date30 May 2025

Sponsor / Collaborator

Sanofi

NCT06940830

/ Not yet recruitingPhase 4

A Low-interventional Study to Evaluate Long-term Effectiveness of Real-world Prophylactic Treatment With Efanesoctocog Alfa on Joint Health in People With Haemophilia A (ALTITUDE)

The rationale for this study is to further understand and describe the long-term prophylactic effectiveness of efanesoctocog alfa in preventing joint bleeds in a real-life setting.

Start Date01 May 2025

Sponsor / Collaborator

Swedish Orphan Biovitrum AB

[+1]

NCT06716814

/ RecruitingPhase 3

A Multinational, Prospective, Open-label, Roll-over Study (LIBERTY) to Provide Post-trial Access to Treatment for Patients With Severe Haemophilia A Who Have Completed a Previous Trial With Efanesoctocog Alfa

This is a multinational, prospective, open-label, roll-over study in patients with severe haemophilia A, ≥6 years of age, who have completed participation in any of the parental studies with efanesoctocog alfa; XTEND-ed study (LTS16294), FREEDOM study (Sobi.BIVV001-001), or PK comparison study (Sobi.BIVV001-003). The aim of the study is to provide patients with continuous benefit from efanesoctocog alfa treatment and to further continue clinical monitoring for safety and efficacy until efanesoctocog alfa is commercially available in each patient's respective country (or until March 2027, whichever comes first).
The study starts with the Baseline Visit, which will be done in connection to the End of Study visit (or equivalent) in the respective parent study. Subsequent study visits (on site or phone call) will be done approximately every 13 weeks until End of Treatment. An End of Study safety phone call will be done 14 (+7) days after the End of Treatment Visit.

Start Date06 Mar 2025

Sponsor / Collaborator

Swedish Orphan Biovitrum AB

[+1]

100 Clinical Results associated with Efanesoctocog alfa

100 Translational Medicine associated with Efanesoctocog alfa

100 Patents (Medical) associated with Efanesoctocog alfa

Literatures (Medical) associated with Efanesoctocog alfa

01 May 2025·AMERICAN JOURNAL OF HEMATOLOGY

Treatment of Bleeding Episodes With Efanesoctocog Alfa in Previously Treated Patients With Severe Hemophilia A in the Phase 3 XTEND‐1 Study

Article

Author: Tosetto, Alberto ; Meunier, Sandrine ; Weyand, Angela C. ; Bystrická, Linda ; Willemze, Annemieke ; Neill, Graham ; Suzuki, Nobuaki ; Abad‐Franch, Lydia

ABSTRACT:

Despite therapeutic advances, people with hemophilia experience bleeds. These may be life‐threatening, result in permanent joint damage, chronic pain, difficulties with mobility/daily activities, and impact quality of life. In the XTEND‐1 study (NCT04161495), once‐weekly efanesoctocog alfa (50 IU/kg) prophylaxis provided highly effective bleed prevention and high‐sustained factor levels for most of the week and was well‐tolerated. We report post hoc analysis of bleeding episodes and their treatment in previously treated patients (≥ 12 years old). Participants received 50 IU/kg efanesoctocog alfa either as once‐weekly prophylaxis (Arm A) or on‐demand followed by once‐weekly prophylaxis (Arm B) in XTEND‐1. Endpoints included treatment of bleeding episodes and response to treatment. During XTEND‐1, 422 bleeding episodes were reported among 159 participants; 362 were treated. Most treated bleeding episodes (74%; n = 268) occurred during the Arm B on‐demand period, of which 197 (74%) were spontaneous. Seventy‐five participants had no bleeding episodes in Arm A; all in Arm B had ≥ 1 bleeding episode while on‐demand. Most participants (n = 107, 81%) had zero treated spontaneous bleeding episodes and rates of treated bleeding episodes in Arm A (prophylaxis) were low (median [interquartile range] overall ABR: 0.00 [0.00–1.04]). A single injection was sufficient to resolve 97% (350/362) of treated bleeding episodes, no bleeding episodes required > 3 injections, and responses to 95% of evaluable injections were rated excellent/good. Median total dose was 50.9 IU/kg per bleeding episode. Results of this analysis further demonstrated that once‐weekly efanesoctocog alfa provides highly effective bleed protection and treatment of bleeding episodes in participants with severe hemophilia A.Trial Registration: NCT04161495

01 Mar 2025·HAEMOPHILIA

In vitro combined haemostatic efficacy of emicizumab and extended half‐life factor VIII compounds

Article

Author: Désage, Stéphanie ; Leuci, Alexandre ; Lienhart, Anne ; Dargaud, Yesim ; Nougier, Christophe ; Josset, Laurie ; Rezigue, Hamdi

Abstract:

Introduction:

Early prophylaxis is the gold standard of care for severe haemophilia. The development of subcutaneous Factor VIII (FVIII) mimetics, such as emicizumab, has significantly reduced the disease burden and improved protection against bleeding episodes. Despite its benefits, emicizumab does not fully normalize haemostasis, requiring additional FVIII treatment for surgical procedures and management of breakthrough bleeding. In these cases, extended or ultra‐extended half‐life FVIII products are most commonly used. However, laboratory monitoring of these combinations can be challenging.

Aim:

This study investigates the in vitro combined haemostatic activity of emicizumab with efmoroctocog alfa and efanesoctocog alfa using a thrombin generation assay (TGA).

Results and Conclusion:

TGA can be used to monitor combined treatment with emicizumab and either efmoroctocog alfa or efanesoctocog alfa, which is not possible with currently available FVIII reagents for the latter. As expected, there is no synergistic effect between the mimetic and FVIII at therapeutical doses. Both efmoroctocog alfa and efanesoctocog alfa show similar in vitro procoagulant activity in terms of thrombin generation.

02 Jan 2025·Expert Opinion on Drug Metabolism & Toxicology

Pharmacokinetic evaluation of efanesoctocog alfa: breakthrough factor VIII therapy for hemophilia A

Review

Author: Yada, Koji ; Nogami, Keiji

INTRODUCTION:

Blood coagulation factor (F)VIII functions as a cofactor in the tenase complex responsible for phospholipid-dependent FIXa-mediated activation of FX in plasma. Congenital defect of FVIII causes severe bleeding disorder, hemophilia (H) A. Intravenous FVIII replacement therapy is the gold standard therapy in patients with HA (PwHA) but requirement for frequent dosing of FVIII owing to pharmacokinetics burdens PwHA a lot. Efanesoctocog alfa is a new class of recombinant FVIII and has the ability to overcome conceivable unmet needs in treatment for PwHA.

AREAS COVERED:

Efanesoctocog alfa is a B domain-deleted single-chain fusion FVIII connected to the Fc-region of human immunoglobulin G1, D'D3-fragment of von Willebrand factor (VWF), and unstructured hydrophilic recombinant polypeptides (XTEN). Owing to its novel design, it can function independently of endogenous VWF and elicits 2 to 4 times longer half-life compared to other existing FVIII products. The prolonged half-life contributes to maintain high level of FVIII activity for most of the week and has led to excellent hemostatic effect by once-weekly administration in phase 3 clinical trials.

EXPERT OPINION:

Efanesoctocog alfa with outstanding pharmacological properties, well tolerated in the clinical trials, is a promising FVIII therapy for PwHA. Future studies should include long-term safety, especially in previously untreated patients.

114

News (Medical) associated with Efanesoctocog alfa

24 Apr 2025

·endpts.com

Sanofi's Altuviiio closing in on blockbuster status, Dupixent sales surge

Sanofi posted a strong set of first-quarter results Thursday morning, with its recombinant factor drug Altuviiio on track to become a blockbuster drug and its crown jewel Dupixent continuing to draw sales. Sales of the French drugmaker’s hemophilia A treatment Altuviiio doubled in the first quarter to €251 million ($286 million), according to a company earnings report . Almost 90% of that number was driven by the US, where patients continued to switch over from older plasma-derived and recombinant factor medicines. The remaining €33 million came from other markets, boosted by a recent launch in Japan. Altuviiio has been steadily gaining market share since it secured FDA approval in 2023, according to Sanofi. If it can replicate its Q1 sales for the rest of the year, the product should reach blockbuster status in 2025, a company spokesperson told Endpoints News in an email. Altuviiio is administered weekly, giving it a significant convenience edge over older recombinant factor drugs, which can require up to thrice weekly dosing. Sanofi’s older recombinant factor medicine, Eloctate, saw sales drop around 21% to €70 million as patients switched to Altuviiio. But its broader hemophilia A franchise fared positively, with sales rising by 50% to €321 million. That number formed part of Sanofi’s total first-quarter sales of €9.9 billion, which were up almost 10% from the same time last year. The figure came in 3% ahead of Wall Street consensus forecasts and was driven by continued growth of the company’s blockbuster inflammatory disease drug Dupixent, Jefferies analysts said in a Thursday note. Dupixent sales were up around 20% in the quarter to €3.5 billion. The growth was driven by rising use in all established indications, as well as “emerging use” in its latest approval for chronic obstructive pulmonary disorder, Sanofi said. Dupixent is expected to reach peak sales of $3.3 billion in COPD alone, according to Wall Street consensus estimates. Sanofi’s hemophilia offering was further bolstered by an FDA approval for siRNA medicine Qfitlia in hemophilia A and B last month. Qfitlia will be sold in the US at an average cost of $642,000 per year, but comes with a boxed warning for thrombotic events and gallbladder disease. Sanofi also reported a Phase 2 disappointment for its oral TNF inhibitor, balinatunfib, in psoriasis. The drug did not hit statistical significance in the primary endpoint of PASI-75, which measures the proportion of patients with a minimum 75% improvement in their psoriasis area and severity index score. Sanofi said the failure was driven by “the limited nature” of the trial, adding it would share full data at a future medical meeting. For now, the company is considering potential combination approaches for balinatunfib. Elsewhere, Sanofi made several cuts to its cancer pipeline. The drugmaker terminated a Phase 2 trial for NK cell engager SAR443579 in acute myeloid leukemia and a Phase 1 study of monoclonal antibody SAR444881 in solid tumors. The cuts are part of a broader pipeline prioritization that should help the company focus on its goal of becoming a world leader in immunology, the spokesperson said. On a call with the media on Thursday, CFO François Roger said the company is continuing to assess how the looming pharma tariffs would impact its business. Editor’s Note: This article was updated to add information about the balinatunfib trial failure and the company’s comments on potential pharma tariffs.

Drug ApprovalPhase 2Phase 1

24 Apr 2025

·www.globenewswire.com

Press Release: Sanofi: strong Q1 performance and 2025 guidance confirmed

Sanofi: strong Q1 performance and 2025 guidance confirmed Paris, April 24, 2025 Q1 sales growth of 9.7% at CER1 and business EPS2 of €1.79 Pharma launches reached sales of €0.8 billion, up 43.8%, driven by ALTUVIIIODupixent sales were €3.5 billion, up 20.3%Vaccines sales were €1.3 billion, up 11.4%, driven by favorable Beyfortus phasingResearch and Development expenses reached €1.8 billion, up 6.9%Selling, general and administrative expenses were €2.2 billion, up 3.8%Business EPS was €1.79, up 15.7% at CER and up 17.0% reported; IFRS EPS was €1.52 Pipeline continued to deliver Six regulatory approvals across medicines in immunology, rare diseases, and oncologyRecent new data from the mid-stage pipeline unlocks late-stage studies in asthma and skin diseases, broadening and deepening the scope in immunology Sustainability strategy Updated sustainability strategy focuses on access to healthcare, environmental impact, and resilience of healthcare systems. 70% of the portfolio and >75% of the pipeline target diseases that are impacted by climate and environmental challenges Capital allocation The closing of the sale of a controlling stake in Opella to CD&R is anticipated in Q23Sanofi has agreed to acquire DR-0201, a targeted bispecific myeloid cell engager4 Guidance confirmed In 2025, sales are anticipated to grow by a mid-to-high single-digit percentage at CER5. Sanofi confirms the expectation of a strong rebound in business EPS with growth at a low double-digit percentage at CER (before share buyback)6Sanofi intends to complete a share buyback program in 2025 of €5 billion of which 72% was already repurchased Paul Hudson, Chief Executive Officer: “We had a strong start in 2025 with sales growth of 9.7%, benefiting from investments in innovation and a favorable base of comparison. Our focus on pipeline value delivered further growth for Sanofi with sales from launches of new medicines and vaccines growing by 46.5%. We obtained approval for Qfitlia (fitusiran), a new treatment for patients with hemophilia, one of three potential launches this year. ALTUVIIIO is continuing to gain market share and is on track to become our next blockbuster in the full year. Business EPS was €1.79, confirming the expected strong rebound in 2025. Our redeployment of capital continued with significant progress on our share buyback program, the upcoming closing of the sale of a majority stake in Opella and the recent acquisition of a bispecific myeloid cell engager for deep B-cell depletion in immunology, strengthening our early pipeline and providing a potential treatment option in difficult-to-treat diseases like lupus. We achieved significant advances in the pipeline with six approvals. We also delivered steady progress across many new medicines in respiratory diseases and dermatology, including clinically meaningful efficacy for amlitelimab in asthma, allowing us to enter phase 3 development to create future value for patients, society, and our company. Underpinned by the growth in sales and business EPS, we confirm our 2025 guidance with the knowledge of the external environment we have today.” Q1 2025 Change Change at CER IFRS net sales reported €9,895m +10.8% +9.7% IFRS net income reported €1,872m +65.2% — IFRS EPS reported €1.52 +67.0% — Free cash flow7 €1,029m — — Business operating income €2,902m +20.1% +18.7 % Business net income €2,212m +15.9% +14.5 % Business EPS €1.79 +17.0% +15.7% Attachment Press_Release

Drug ApprovalAcquisitionVaccinePhase 3Clinical Result

23 Apr 2025

·www.prnewswire.com

Sobi presents clinical data at WFH 2025 Comprehensive Care Summit

STOCKHOLM, April 23, 2025 /PRNewswire/ -- Sobi® (STO: SOBI) presents clinical data aimed at updating the global haemophilia community attending this year's WFH 2025 Comprehensive Care Summit in Dubai from the 23 – 25 April. New and updated outcomes and analyses will be presented from the XTEND phase 3clinical program testing the effectiveness of Altuvoct® (efanesoctocog alfa) treatment for haemophilia A, including updates on patients' joint health and surgical outcomes in patients. "At WFH 2025 CCS, Sobi will present highlights from the XTEND phase 3 program and discuss the new treatment paradigm with FVIII levels in the non-haemophilia range. Sharing these clinical outcomes with the wider haemophilia community will help to ensure the latest treatment approaches are widely known and understood," said Lydia Abad-Franch, MD, MBA, Head of Research, Development, and Medical Affairs (RDMA), and Chief Medical Officer at Sobi. Key data to be presented at WFH 2025 Comprehensive Care Summit, Dubai, UAE Altuvoct® (efanesoctocog alfa) About efanesoctocog alfa Efanesoctocog alfa [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] (formerly BIVV001) is a novel and significant part of the week factor VIII therapy. It has the potential to deliver near-normal factor activity levels for significant parts of the week, improving bleed protection in a once-weekly dose for people with haemophilia A. Efanesoctocog alfa builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN® polypeptides to extend its time in circulation. It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies. It was approved as ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] by Sanofi in the US in February 2023. About the Sobi and Sanofi collaboration Sobi and Sanofi collaborate on the development and commercialisation of Alprolix® and Elocta®/Eloctate®. The companies also collaborate on the development and commercialisation of efanesoctocog alfa, referred to as ALTUVIIIO™ in the USA and ALTOVUCT in Europe. An investigational factor VIII therapy with the potential to provide high sustained factor activity levels with once-weekly dosing for people with haemophilia A. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia and most Middle Eastern markets). Sanofi has final development and commercialisation rights in North America and all other regions in the world excluding the Sobi territory. Sobi® Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Providing sustainable access to innovative medicines in the areas of haematology, immunology and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East and Asia. In 2022, revenue amounted to SEK 18.8 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com, LinkedIn and YouTube. Contact For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Drug ApprovalBiosimilar

100 Deals associated with Efanesoctocog alfa

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hemophilia A	United States	Bioverativ, Inc.	22 Feb 2023

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hemorrhage	Phase 3	United States	Sanofi (China) Investment Co. Ltd.	23 Feb 2021
Hemorrhage	Phase 3	United States	Bioverativ Therapeutics, Inc.	23 Feb 2021
Hemorrhage	Phase 3	United States	Biogen, Inc.	23 Feb 2021
Hemorrhage	Phase 3	Belgium	Biogen, Inc.	23 Feb 2021
Hemorrhage	Phase 3	Belgium	Sanofi (China) Investment Co. Ltd.	23 Feb 2021
Hemorrhage	Phase 3	Belgium	Bioverativ Therapeutics, Inc.	23 Feb 2021
Hemorrhage	Phase 3	Bulgaria	Sanofi (China) Investment Co. Ltd.	23 Feb 2021
Hemorrhage	Phase 3	Bulgaria	Biogen, Inc.	23 Feb 2021
Hemorrhage	Phase 3	Bulgaria	Bioverativ Therapeutics, Inc.	23 Feb 2021
Hemorrhage	Phase 3	Canada	Sanofi (China) Investment Co. Ltd.	23 Feb 2021

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


XTEND-1 and XTEND-Kids (Pubmed \| Haemophilia)	Phase 3	Hemophilia A factor VIII (FVIII) activity levels	-	Efanesoctocog alfa 50 IU/kg	pkvezxqfzw(xbaldaghbe) = icskrcskfa ghqqawwhjc (piimvhgvwa, 45 - 361)	Positive	18 Mar 2025
NCT04161495 (XTEND-1, Pubmed \| Clin Ther)	Phase 3	Hemophilia A	159	Efanesoctocog alfa prophylaxis 50 IU/kg for 52 weeks	yqncwlgdlu(xrwmwwgmju) = lrbxerpxtt myihqiqmnu (ksuppxjogr ) View more	Positive	01 Dec 2024
NCT04759131 (XTEND-Kids, PRNewswire) Manual	Phase 3	Hemophilia A	73	ALTUVOCT	rmnolbsogy(kwwgffqkwe) = lfcenzgolx hwqfidarce (qflsjcqvrk, 0.42–0.90) View more	Positive	18 Jul 2024
NCT04759131 (XTEND-Kids, Pubmed)	Phase 3	Hemophilia A	74	Efanesoctocog alfa	jrrkeflvuj(dhjwqkcdpk) = Most adverse events were nonserious. No serious adverse events that were assessed by the investigator as being related to efanesoctocog alfa were reported. nxulibfiqf (vcornjvjdr )	Positive	18 Jul 2024
NCT05042440 (Phase 1 sequential pharmacokinetic study, EHA2024) Manual	Phase 1	Hemophilia A FVIII	13	Octocog alfa	yggjlayiau(grfsacfdyr) = zumlvxnoui nmyltnltnb (pgbwqdxgwa ) View more	Positive	14 May 2024
	Phase 1	Hemophilia A FVIII	13	Rurioctocog alfa pegol	yggjlayiau(grfsacfdyr) = sylcnuabhk nmyltnltnb (pgbwqdxgwa ) View more	Positive	14 May 2024
NCT04759131 (XTEND-Kids, CTgov)	Phase 3	Hemophilia A	74	efanesoctocog alfa (BIVV001) (BIVV001: Participants Aged <6 Years)	qjhdvowkjy = qscepderqh xnpfyubkdj (cissysfujz, qpffegkrua - dligafoctl) View more	-	13 Feb 2024
NCT04759131 (XTEND-Kids, CTgov)	Phase 3	Hemophilia A	74	efanesoctocog alfa (BIVV001) (BIVV001: Participants Aged 6 to <12 Years)	qjhdvowkjy = zmbuhkiiqa xnpfyubkdj (cissysfujz, kcrcxrfckm - mzqobekcuv) View more	-	13 Feb 2024
NCT04161495 (XTEND-1, ISTH2023)	Phase 3	Hemophilia A	78	Standard-of-care FVIII prophylaxis	cvsnieeydh(hftrggqzvz) = dmbubortyz ydtkwwzmcg (sbopwwrhsq )	-	24 Jun 2023
NCT04161495 (XTEND-1, ISTH2023)	Phase 3	Hemophilia A	78	Efanesoctocog alfa 50 IU/kg prophylaxis	cvsnieeydh(hftrggqzvz) = dfxxcjjjrd ydtkwwzmcg (sbopwwrhsq )	-	24 Jun 2023
NCT04161495 (XTEND-1, ASH 12022 \| ASH 22022) Manual	Phase 3	Hemophilia A	159	Efanesoctocog alfa	griwbilunz(nydwwuqqnl) = wipihjiwdj xykktqhvnv (pndgxvxwkn ) View more	Positive	15 Nov 2022
NCT04161495 (XTEND-1, ASH 12022 \| ASH 22022) Manual	Phase 3	Pain \| Hemophilia A	159	Efanesoctocog alfa (Arm A)	wyqxieagjj(oqxtxgjmek) = was improved or maintained (change from baseline≤0) for most patients (81.5% [97/119]) at Week 52 vaogcyqcmk (ekavkspsng )	Positive	15 Nov 2022
NCT04161495 (XTEND-1, ASH 12022 \| ASH 22022) Manual	Phase 3	Pain \| Hemophilia A	159	Efanesoctocog alfa (Arm B)		Positive	15 Nov 2022
NCT04161495 (XTEND-1, Corporate Publications) Manual	Phase 3	Hemorrhage	150	Efanesoctocog alfa	ghxcbrgcbk(zxdobuyogi) = dnvrksovxz mirxeceyva (vgefxymohv )	Positive	11 Jul 2022

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