Altuviiio (antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl) / SOBI, Sanofi - LARVOL DELTA

Sobi's strength in haematology to be showcased at ASH 2024 (PRNewswire) - "New data from Sobi and partners will be presented at the 66th Annual Meeting of the American Society of Hematology (ASH) in San Diego, CA (USA) from the 7th - 10th of December 2024. During the meeting several analyses will be presented on haemophilia A...paroxysmal nocturnal haemoglobinuria (PNH)..."

Clinical data • P3 data • Genetic Disorders • Hemophilia • Hemophilia A • Paroxysmal Nocturnal Hemoglobinuria

Companies.- Sobi's 'Altuvoct' (efanesoctocog alfa) arrives in Spain for the treatment of haemophilia A [Google transltion] (Infosalus.com) - "Sobi announced on Tuesday that the Ministry of Health has approved 'Altuvoct' (efanesoctocog alfa) as an orphan drug for the treatment and prophylaxis of bleeding in patients with hemophilia A, making this drug the first ultra-extended half-life replacement therapy authorized for hemophilia."

Launch Europe • Genetic Disorders • Hemophilia • Hemophilia A

Clinical Outcomes over 2 Years of Once-Weekly Efanesoctocog Alfa Treatment in Children with Severe Hemophilia A: Second Interim Analysis from the Phase 3 XTEND-ed Long-Term Extension Study (ASH 2024) - P3 | "None of the TEAEs led to death or treatment discontinuation. There were no thrombotic events.ConclusionThe results from over 2 years in previously treated children with severe hemophilia A show that once-weekly efanesoctocog alfa continues to be well tolerated and provides highly effective bleed protection with no FVIII inhibitors reported."

Clinical • Clinical data • P3 data • P3 data: top line • CNS Disorders • Cough • Epilepsy • Hematological Disorders • Hemophilia • Hemophilia A • Musculoskeletal Pain • Pain • Rare Diseases • Respiratory Diseases

Real-World Use of Efanesoctocog Alfa in Adult Patients with Hemophilia a Including Those with a History of Factor VIII Inhibitor (ASH 2024) - "Eighteen PwHA used extended half-life recombinant Factor VIII products, one used standard half life products, and four used emicizumab-kxwh. Our cost analysis is based on estimates, actual costs may differ. Future studies should aim to evaluate long-term comprehensive outcomes in patients on Efa prophylaxis and its impact on health care system utilization."

Clinical • Real-world • Real-world evidence • Cardiovascular • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • Thrombosis

Real-World Patterns of Additional Factor Treatment Use Among Hemophilia a Patients on Regular Prophylaxis in the United States: Results from the Picnichealth Database (ASH 2024) - "Background The standard-of-care for hemophilia A (HA) involves prophylaxis (PPX) with factor replacement therapies (standard half-life [SHL] and extended half-life [EHL]), or emicizumab (Emi)...Patients with evidence of mild HA (6%−50%), any FVIII inhibitor (on index), or treated with efanesoctocog alfa were excluded...Among Emi PPX users, 62% on weekly regimen, 64% on biweekly regimen, and 36% on monthly regimen used additional factor treatment. Conclusion The majority of patients with moderate or severe HA on routine PPX use additional factor treatments to treat bleeds, whereas a smaller proportion of patients use additional factor treatment before physical activity or procedures to prevent bleeds."

Clinical • Real-world • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia A • Pain • Pediatrics • Rare Diseases

Indirect Comparison of the Efficacy and Therapy-Related Costs of a Pharmacokinetic and Individualized Prophylaxis Regimen with Simoctocog Alfa Versus Other Extended-Half Life Factor VIII Concentrates (ASH 2024) - "For comparison, aggregated data was obtained from the following trials with EHL concentrates : pathfinder2 (turoctocog alfa pegol, N = 175), A-LONG (efmoroctocog alfa, N = 117), PROTECT FVIII (damoctocog alfa pegol, N = 110), PROPEL (rurioctocog alfa pegol 1–3% and 8–12%; N = 57 and 58), and XTEND-1 (efanesoctocog alfa, Group A, N = 133). Conclusion : Albeit at a generally higher weekly dose, a PK-guided, individualized prophylaxis regimen with simoctocog alfa offered comparable or significantly improved zero bleed rates and significantly lower or comparable ABRs than prophylactic regimens with EHL rFVIII concentrates. Nevertheless, the estimated annual cost of a simoctocog alfa-based regimen is 20–55% lower than with the other concentrates."

Clinical • PK/PD data • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Cost Comparison of Efanesoctocog Alfa with Existing Factor VIII Replacement Therapies for Major Surgeries in People with Severe Hemophilia a (ASH 2024) - P2/3, P3 | "Objective To estimate total costs associated with perioperative hemostatic management in patients with severe HA treated with SHL (octocog alfa), EHL (rurioctocog alfa pegol and efmoroctocog alfa), and high-sustained (efanesoctocog alfa) FVIII replacement therapies. This is attributed to its high-sustained factor activity and reduced factor consumption during the reported perioperative period. The major limitations of the study were : the types of major surgeries varied among studies; the perioperative period data of octocog alfa were not found."

HEOR • Reimbursement • Surgery • US reimbursement • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Association between Hemophilia Joint Health Score and Quality of Life Using Results from the Xtend-1 Efanesoctocog Alfa Phase 3 Trial (ASH 2024) - P3 | "LS mean difference (95% confidence interval) between QoL PROs CFB at Week 52 in "Maintenance or improvement" and "Worsening" HJHS subgroups were : PROMIS Pain Intensity worst score -0.61 (‑1.17 to -0.05; p=0.032), PROMIS Pain Intensity T-score -3.79 (-7.56 to -0.02; p=0.049), Haem-A-QoL total score -6.21 (-12.38 to -0.05; p=0.048), Haem-A-QoL PH score -2.65 (-4.63 to -0.67; p=0.009), and PROMIS PF T-score 3.34 (5.99 to 0.69; p=0.014). Conclusion Our findings indicate a stronger improvement in PROs across the three domains of pain, physical functioning, and overall QoL, in patients with joint improvement or maintenance compared with patients with joint worsening, thus highlighting the importance of improved or preserved joint health in patients with hemophilia A. Prospective validation is warranted to confirm these data."

HEOR • P3 data • Hematological Disorders • Hemophilia • Hemophilia A • Orthopedics • Pain • Rare Diseases

Clinical Outcomes over 3 Years of Once-Weekly Efanesoctocog Alfa Treatment in Adults and Adolescents with Severe Hemophilia A: Second Interim Analysis from the Phase 3 XTEND-ed Long-Term Extension Study (ASH 2024) - P3 | "Conclusion Long-term results in adults and adolescents in XTEND-ed shows that once-weekly efanesoctocog alfa continues to provide high efficacy and is well tolerated. No inhibitors were detected and ABRs remained low, with continued improvement observed for overall QoL."

Clinical • Clinical data • P3 data • P3 data: top line • Atrial Fibrillation • Cardiovascular • CNS Disorders • Hematological Disorders • Hemophilia • Hemophilia A • Infectious Disease • Musculoskeletal Diseases • Musculoskeletal Pain • Novel Coronavirus Disease • Orthopedics • Rare Diseases • Thrombosis

Real-World Experience of Switching to Prophylactic Efanesoctocog Alfa in Patients with Moderate and Severe Hemophilia a: An Analysis of the Adelphi Hemophilia Wave III Disease Specific Programme (ASH 2024) - "Patients in the United States with moderate or severe hemophilia A who had switched from prophylaxis with FVIII replacement therapy or non-factor therapy (emicizumab) to receiving efanesoctocog alfa prophylactically for ≥170 days were included in this analysis. The limitations of this study were the small sample size and the limited duration of efanesoctocog alfa treatment. These data indicate that in a real-world setting, efanesoctocog alfa provides an effective alternative to existing treatments for hemophilia A, with a once-weekly dosing schedule."

Clinical • Real-world • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

LIBERTY: Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa (clinicaltrials.gov) - P3 | N=78 | Not yet recruiting | Sponsor: Swedish Orphan Biovitrum

New P3 trial • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

PROTECT-ALT: A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A in Taiwan (clinicaltrials.gov) - P=N/A | N=120 | Recruiting | Sponsor: Sanofi | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Efanesoctocog Alfa versus Standard and Extended Half-Life Factor VIII Prophylaxis in Adolescent and Adult Patients with Haemophilia A without Inhibitors. (PubMed, Adv Ther) - P3 | "Efanesoctocog alfa was associated with significantly lower ABRs (any, spontaneous and joint) compared with EHL or SHL prophylaxis therapies. Patients had, on average, 2.2 and 3.6 fewer bleeds per year versus EHL and SHL therapies, respectively."

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Efanesoctocog Alfa Versus Emicizumab in Adolescent and Adult Patients With Haemophilia A Without Inhibitors. (PubMed, Adv Ther) - P3 | "Efanesoctocog alfa prophylaxis was associated with significantly lower rates of any, treated, and joint bleeds and improved joint health compared with emicizumab in patients with severe haemophilia A."

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

PROTECT-ALT: A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A in Taiwan (clinicaltrials.gov) - P=N/A | N=120 | Not yet recruiting | Sponsor: Sanofi

New trial • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Press Release: Sanofi Q3: 15.7% sales growth boosted by earlier-than-anticipated vaccine sales; 2024 business EPS guidance raised due to strong business performance (GlobeNewswire) - "Dupixent sales up 23.8% to €3,476 million; full-year 2024 target of ~€13 billion confirmed; Pharma launches up 67.1% to €727 million, led by ALTUVIIIO, Nexviazyme and Rezurock...US sales were €6,886 million and increased by 23.6%. The strong performance was driven by new launches, including Beyfortus, and by Dupixent, insulins and all Vaccines businesses with sales of legacy medicines slightly offsetting growth. Europe sales were €2,886 million and increased by 6.6%. Growth was driven by new launches, Dupixent, and Vaccines....Rest of World sales were €3,666 million and increased by 10.3%. Performance was led by Dupixent and Vaccines, and partly by new launches and Toujeo. China sales were €757 million and increased by 3.6%, driven by strong performance by Dupixent mostly offset by Lovenox and Other medicines."

Sales • Atopic Dermatitis • Chronic Obstructive Pulmonary Disease • Chronic Rhinosinusitis With Nasal Polyps • Chronic Spontaneous Urticaria • Eosinophilic Esophagitis • Graft versus Host Disease • Hemophilia A • Prurigo Nodularis

Patient Experience With Efanesoctocog Alfa for Severe Hemophilia A: Results From the XTEND-1 Phase 3 Clinical Study Exit Interviews. (PubMed, Clin Ther) - P3 | "The exit interviews demonstrated that once-weekly efanesoctocog alfa prophylaxis resulted in patient-relevant and meaningful improvements in pain and physical functioning, consistent with the quantitative findings from XTEND-1. These results support the validity of the Haem-A-QoL PH and PROMIS Pain Intensity 3a assessed during XTEND-1, demonstrating the potential for change with efficacious treatment."

Interview • Journal • P3 data • Hematological Disorders • Hemophilia • Hemophilia A • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Osteoarthritis • Pain • Rare Diseases

Pharmacokinetic evaluation of efanesoctocog alfa: breakthrough factor VIII therapy for hemophilia A. (PubMed, Expert Opin Drug Metab Toxicol) - "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."

Journal • PK/PD data • Review • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A (clinicaltrials.gov) - P1 | N=24 | Not yet recruiting | Sponsor: Swedish Orphan Biovitrum

New P1 trial • Hematological Disorders • Hemophilia • Rare Diseases

Factor VIII stimulants and other novel therapies for the treatment of von Willebrand disease: what's new on the horizon? (PubMed, Expert Opin Pharmacother) - "The FVIII mimetic, emicizumab, has demonstrated early success in patients with severe VWD and is a promising treatment option for those who require prophylaxis. Furthermore, products like efanesoctocog alfa (Altuviiio®) and BT200 have achieved enhanced VWF/FVIII half-life extension could expand the current treatment landscape while concurrently minimizing treatment burden."

Journal • Hematological Disorders • Hemophilia

Altuvoct® - Schutz bei Hämophilie A. (PubMed, MMW Fortschr Med) - No abstract available

Journal

ACTIVIIITY: Goal Attainment and Physical Activity in People With Hemophilia A (clinicaltrials.gov) - P=N/A | N=35 | Not yet recruiting | Sponsor: Sanofi

New trial • Hematological Disorders • Hemophilia • Rare Diseases

Quarterly sales of Sanofi’s Dupixent cross €3B for the first time (Firstwordpharma Press Release) - "Sales of Sanofi’s Dupixent soared 29.2% in the second quarter to €3.3 billion ($3.6 billion), setting a new three-month high for the product, which the company said Thursday remains on course to generate revenue this year of around €13 billion...The company noted that new launches totalling €707 million made up 7% of biopharma sales in the second quarter, led by Altuviiio (+772.2% to €158 million), Nexviazyme/Nexviadyme (+66% to €168 million), Rezurock (+52.7% to €114 million)....Another product for which Sanofi has high hopes, its AstraZeneca-partnered respiratory syncytial virus vaccine. Beyfortus, only brought in €18 million in the quarter due to the 'seasonality towards the second half-year.'"

Sales • Atopic Dermatitis • Chronic Obstructive Pulmonary Disease • Dermatitis • Hemophilia • Idiopathic Pulmonary Fibrosis • Immunology • Inflammatory Bowel Disease • Pompe Disease • Pulmonary Disease • Respiratory Diseases • Respiratory Syncytial Virus Infections • Rheumatoid Arthritis • Scleroderma • Systemic Sclerosis

Efanesoctocog Alfa Prophylaxis for Children with Severe Hemophilia A. (PubMed, N Engl J Med) - P3 | "In children with severe hemophilia A, once-weekly prophylaxis with efanesoctocog alfa provided high sustained factor VIII activity in the normal to near-normal range (>40 IU per deciliter) for 3 days and more than 10 IU per deciliter for almost 7 days after administration, leading to effective bleeding prevention. Efanesoctocog alfa was associated with mainly nonserious adverse events. (Funded by Sanofi and Sobi; XTEND-Kids ClinicalTrials.gov number, NCT04759131.)."

Journal • Hematological Disorders • Hemophilia • Rare Diseases

Netherlands Includes Alecensa, Altuvoct, Truqap & Rybrevant in Lock (NAVLIN DAILY) - "A government gazette has outlined that Alecensa (alectinib), Altuvoct (efanesoctocog alfa), Truqap...and Rybrevant...will be included in the lock for expensive medicines...The National Health Care Institute (ZIN) expects the cost of Alecensa to be EUR 71,598.40 per patient annually, with an estimated maximum macro budget impact of EUR 11,978,412 per year by the third year...Meanwhile, the annual cost of Altuvoct is projected at EUR 924,300 per patient...A maximum of 939 patients per year are expected to be eligible for Truqap. Based on the price of Truqap, the total cost is EUR 1,420 per patient per year. Given the expected maximum number of patients...the maximum macro cost of the medicinal product shall be estimated at EUR 38,893,380 per year....For the indication involving Rybrevant and lazertinib, the treatment is projected to involve up to 522 patients, with a cumulative cost impact reaching up to EUR 91,045,152 by the third year."

Reimbursement • Breast Cancer • Hemophilia • HER2 Breast Cancer • HER2 Negative Breast Cancer • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor