Beroctocog alfa
- Generic Name
- Beroctocog alfa
- Brand Names
- Alphanate
- Drug Type
- Biotech
- CAS Number
- 9001-27-8
- Unique Ingredient Identifier
- 17RNR4H479
- Indication
Beroctocog alfa is indicated for the prevention and control of bleeding in patients with hemophilia A or acquired Factor VIII (FVIII) deficiency. It is also indicated for surgical/invasive procedures in adult and pediatric patients with von Willebrand Disease in who desmopression is either ineffective or contraindicated. It is not indicated for patients with severe (i.e. type 3) von Willebrand Disease whom are undergoing major surgery.
- Associated Conditions
- Bleeding
- Associated Therapies
- Perioperative management therapy
FDA Expands Jivi Approval for Hemophilia A Treatment in Children Ages 7-12
• The FDA has approved Bayer's Jivi (antihemophilic factor recombinant, PEGylated-aucl) for pediatric patients aged 7 to 12 years with hemophilia A, expanding treatment options for this age group.
• The approval was based on data from the Alfa-PROTECT and PROTECT Kids studies, which demonstrated the safety and efficacy of Jivi in pediatric patients with severe hemophilia A.
• Jivi was previously approved in 2018 for adults and adolescents aged 12 and older, and can be used for on-demand treatment, perioperative management, and routine prophylaxis to reduce bleeding episodes.
Acquired Hemophilia A Pipeline Expands with 10+ Novel Therapies in Development
• Global Acquired Hemophilia A pipeline comprises over 10 companies developing innovative therapies across various clinical stages, according to DelveInsight's 2025 report.
• Recent clinical advances include promising results for rituximab as a potential first-line therapy and Pfizer's positive Phase 3 AFFINE trial results for giroctocogene fitelparvovec gene therapy.
• Key pharmaceutical players including Novo Nordisk, Sanofi, Pfizer, and Belief Biomed are advancing treatments ranging from gene therapies to monoclonal antibodies targeting this rare autoimmune bleeding disorder.
Sobi Showcases Efanesoctocog Alfa Data from XTEND Phase 3 Program at WFH 2025 Summit
• Sobi is presenting new clinical outcomes from the XTEND phase 3 program for Altuvoct® (efanesoctocog alfa) at the WFH 2025 Comprehensive Care Summit in Dubai, focusing on joint health and surgical outcomes in hemophilia A patients.
• The data highlights efanesoctocog alfa's ability to maintain factor VIII levels in the non-hemophilia range with once-weekly dosing, potentially transforming treatment standards for people with hemophilia A.
• Efanesoctocog alfa utilizes innovative Fc-VWF-XTEN fusion protein technology that breaks through the von Willebrand factor ceiling, overcoming the half-life limitation affecting current factor VIII therapies.
CDMO Market Report: Key Regulatory Approvals and Clinical Advances in March-April 2025
• Multiple CDMOs secured significant contract manufacturing opportunities as regulatory bodies approved new indications for established drugs, particularly in oncology and rare diseases.
• AstraZeneca's portfolio saw substantial growth with expanded approvals for Imfinzi, Tagrisso, and Lynparza, strengthening partnerships with contract manufacturers including Lonza, Dottikon, and Samsung Biologics.
• Contract manufacturers supporting treatments for autoimmune conditions showed strong performance, with Argenx's Vyvgart Hytrulo receiving expanded indications for myasthenia gravis and CIDP.
New Study Highlights Benefits of Maintaining High Factor VIII Levels in Hemophilia A Patients
• Maintaining high Factor VIII (FVIII) levels in people with hemophilia A provides significant clinical benefits, including reduced bleeding risk and better joint health preservation.
• Despite advances in prophylactic treatment, standard-of-care prophylaxis does not completely prevent joint damage or chronic pain, with approximately 47% of hemophilia patients experiencing ongoing pain.
• Recent innovations in hemophilia treatment, including extended half-life therapies, non-factor replacement options, and gene therapy, offer new approaches to sustaining higher FVIII levels and improving patient outcomes.
NHS Approves Revolutionary Weekly Treatment for Severe Haemophilia A at £510,000 Per Year
• NICE has recommended efanesoctocog alfa, a groundbreaking weekly injection therapy, for NHS patients aged two and above with severe haemophilia A, affecting approximately 1,900 people in the UK.
• The innovative treatment, priced at £510,000 annually for an average adult, will be available at a confidential discounted rate through an agreement between NHS England and Swedish Orphan Biovitrum.
• This once-weekly dosing regimen represents a significant advancement over current factor VIII replacements, offering effective bleeding control while reducing treatment burden for patients with severe haemophilia A.
Hemlibra Shows Significant Improvement in Joint Pain for Hemophilia A Patients in 78-Week Study
• A Phase 3 clinical trial involving 504 patients with severe hemophilia A demonstrated that Hemlibra treatment increased the proportion of patients reporting "no or little swelling pain" from 37% to 84% within 13 weeks.
• The bispecific antibody therapy showed sustained pain improvement benefits for up to 78 weeks, with significant increases in patients reporting reduced joint pain compared to conventional factor 8 prophylaxis.
• Approximately 25,000 hemophilia A patients globally are now receiving Hemlibra treatment, which offers a convenient once-weekly subcutaneous injection with prophylactic effects lasting up to four weeks.
UK MHRA Approves Sobi's Efanesoctocog Alfa for Haemophilia A Treatment
• The UK Medicines and Healthcare products Regulatory Agency has approved efanesoctocog alfa (Altuvoct) for treating and preventing bleeding in patients aged 2 years and above with severe or moderate haemophilia A.
• Clinical trials demonstrated significant efficacy, with 65% of patients aged 12 and above reporting zero bleeding episodes over a year-long study when receiving weekly prophylactic injections.
• NICE has recommended efanesoctocog alfa as a cost-effective treatment option specifically for patients with severe haemophilia A (factor VIII activity level less than 1%), subject to commercial arrangements.
Novo Nordisk's Mim8 Shows Promise in Phase III Pediatric Hemophilia A Trial
• Novo Nordisk's bispecific antibody Mim8 demonstrated strong safety and efficacy in Phase III FRONTIER3 trial, with 74.3% of children experiencing no treated bleeds over 26 weeks.
• The trial enrolled 70 children aged 1-11 with hemophilia A, showing particular success in patients with inhibitors, as none required coagulation factor treatment for bleeding events.
• With plans for US and EU regulatory submissions this year, Mim8 positions itself to compete with Roche's established Hemlibra in the hemophilia A market projected to exceed $28 million by 2034.
Fitusiran Shows Promise in Reducing Bleeding Episodes in Hemophilia A and B Patients
• Fitusiran, an RNA interference therapy, is highlighted as a potential breakthrough for hemophilia A and B treatment, regardless of inhibitor status.
• Phase 3 trials (NCT03417245) demonstrated that fitusiran significantly reduced annualized bleeding rates in hemophilia A and B patients compared to on-demand factor concentrates.
• Another study (NCT03417102) showed a 90.8% reduction in annualized bleeding rate in patients with inhibitors treated with fitusiran compared to on-demand treatment.
• Fitusiran's efficacy and convenient subcutaneous administration position it as a drug to watch in hematology, offering a more personalized approach to hemophilia management.
Hemgenix Gene Therapy Shows Sustained Efficacy in Hemophilia B Patients After Four Years
• Data from the HOPE-B trial demonstrates that Hemgenix (etranacogene dezaparvovec-drlb) provides long-term bleed protection for adults with hemophilia B.
• Patients treated with Hemgenix experienced a 90% reduction in adjusted annualized bleeding rate (ABR) at year four compared to the lead-in period.
• 94% of patients remained free of continuous prophylaxis treatment at year four, indicating sustained independence from regular infusions.
• The gene therapy maintained a favorable safety profile, with most adverse events occurring within the first six months post-treatment.
European Commission Approves BioMarin's Roctavian for Severe Hemophilia A
• The European Commission granted conditional marketing approval for BioMarin's Roctavian (valoctocogene roxaparvovec) for severe hemophilia A in adults without Factor VIII inhibitors.
• Roctavian, a one-time gene therapy, delivers a functional Factor VIII gene using an adeno-associated virus vector, potentially offering a curative treatment option.
• BioMarin plans to launch Roctavian in Europe with a 'net' price of approximately €1.5 million, starting in Germany and followed by France, contingent on reimbursement agreements.
• BioMarin intends to refile for Roctavian's approval in the US, anticipating a decision by late March 2023, with a potential price range of $2 to $3 million after discounts.
Pfizer Abandons Hemophilia A Gene Therapy, Jeopardizing Sangamo's Financial Stability
• Pfizer has terminated its partnership with Sangamo Therapeutics for the hemophilia A gene therapy, giroctocogene fitelparvovec, despite promising Phase III trial results.
• This decision by Pfizer casts doubt on the commercial viability of first-generation hemophilia A gene therapies, influenced by the slow market uptake of BioMarin's Roctavian.
• Sangamo's stock plummeted by 56% following the announcement, as the company loses potential milestone payments of up to $220 million from Pfizer.
• Sangamo now intends to explore all options for advancing the gene therapy, including seeking a new collaboration partner, while facing financial challenges with limited cash reserves.
FDA Approves Novo Nordisk's Alhemo for Hemophilia A and B with Inhibitors
• The FDA has approved Alhemo (concizumab-mtci) as a once-daily prophylactic treatment for hemophilia A and B with inhibitors in patients 12 years and older.
• Alhemo, a subcutaneous injection, is the first of its kind for this patient population, offering an alternative to intravenous infusions.
• Clinical trials showed an 86% reduction in treated bleeding episodes with Alhemo compared to no prophylaxis, significantly improving patient outcomes.
• Alhemo works by blocking tissue factor pathway inhibitor (TFPI), enhancing thrombin production to facilitate blood clotting in the presence of inhibitors.
Fitusiran Demonstrates Efficacy in Hemophilia A and B with Reduced Factor Consumption
• Fitusiran, an siRNA therapeutic targeting antithrombin, shows high efficacy as a prophylactic agent for hemophilia A and B in the ATLAS-OLE trial.
• The antithrombin-guided dosing regimen of fitusiran led to a 70% reduction in breakthrough treated bleeds compared to bypassing agent prophylaxis.
• Patients on fitusiran achieved effective bleed control with significantly lower doses of factor VIII/IX or bypassing agents, often with a single infusion.
• The revised dosing strategy, targeting antithrombin levels between 15% and 35%, optimizes the benefit-risk profile of fitusiran prophylaxis.
Roche Discontinues Hemophilia A Gene Therapy Development, Shifts Focus to Enhanced Function FVIII Variant
Roche has decided to discontinue the development of its hemophilia A gene therapy, SPK-8011, following a strategic decision. The company is now focusing on introducing an enhanced function factor VIII (FVIII) variant to its gene therapy pipeline, aiming to address unmet needs and reduce treatment burden for patients.
Hemophilia Treatment Advances: Gene Therapy, Prophylaxis, and On-Demand Therapies Highlighted at ASH 2024
• ASH 2024 highlighted significant progress in hemophilia care since Hemlibra's approval, including gene therapies for hemophilia A and B, offering curative potential.
• Despite advances, unmet needs persist, including spontaneous bleeds in prophylaxis patients, limited gene therapy access, and concerns about long-term risks like liver toxicity.
• Emerging therapies like Pfizer's giroctocogene fitelparvovec and Sanofi's fitusiran show promise, with ongoing trials and regulatory reviews potentially expanding treatment options.
• Novel on-demand treatments like Staidson's bemiltenase alfa offer alternatives, particularly in price-sensitive markets, demonstrating a 12-hour bleed clearance rate of 83%.
Indian Scientists Report Success with Novel Gene Therapy for Severe Hemophilia A
• A novel gene therapy developed in India has shown promising results in treating severe Hemophilia A, with a zero bleeding rate observed in treated patients.
• The gene therapy uses a lentiviral vector to introduce a corrected gene into stem cells, enabling the body to produce Factor VIII autonomously.
• This one-time treatment offers a potential lifelong solution, reducing the need for frequent and expensive clotting factor infusions.
• The initial trial, involving five patients, suggests this therapy could be a more affordable and accessible option for Hemophilia A patients in resource-constrained settings.
Novo Nordisk Presents Promising Data on Sickle Cell Disease and Hemophilia Treatments at ASH 2024
• Novo Nordisk presented Phase 2 results from the HIBISCUS trial, showing etavopivat reduced vaso-occlusive crises in sickle cell disease patients.
• Interim Phase 3 data from the FRONTIER4 trial highlighted the safety and efficacy of Mim8 prophylaxis in hemophilia A patients with or without inhibitors.
• Phase 3 explorer7 study results demonstrated the efficacy of concizumab in hemophilia A or B patients with inhibitors, regardless of target joints at baseline.
EU Approves Pfizer's Hympavzi as First Weekly Subcutaneous Treatment for Severe Hemophilia B
• The European Commission has approved Pfizer's Hympavzi, a once-weekly subcutaneous treatment, for severe hemophilia B in adults and adolescents without inhibitors.
• Hympavzi is the first anti-TFPI antibody approved in Europe for hemophilia A or B without inhibitors, offering convenient at-home administration via autoinjector.
• The approval was based on the Phase 3 BASIS study, which demonstrated a 35% reduction in annualized bleeding rate compared to routine clotting factor injections.
• Hympavzi provides a new treatment option with simplified, weight-independent dosing, potentially reducing the treatment burden for hemophilia B patients.
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