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

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

INDIVIDUAL PHARMACOKINETIC EVALUATION OF FIXED-SEQUENCE SINGLE-DOSE OCTOCOG ALFA, RURIOCTOCOG ALFA PEGOL, AND EFANESOCTOCOG ALFA IN ADULTS WITH SEVERE HEMOPHILIA A (EHA 2024) - P1 | "Consistent superiority regarding time over 40% FVIII activity, half-life, clearance, and FVIII exposure wereobserved with efanesoctocog alfa compared with SHL and EHL FVIII in all patients. No serious TEAEs orinhibitor development were reported for efanesoctocog alfa. Study funded by Sanofi and Sobi."

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

ZIN Recommends Including Altuvoct, Alecensa, Rybrevant, & Truqap in Lock for Expensive Medicines (NAVLIN DAILY) - "ZIN has advised the VWS to include a number of drugs in the lock for expensive medicines following their approval by the EMA in April...First, ZIN noted that Swedish Orphan Biovitrum’s Altuvoct (efanesoctocog alfa), intended for the prevention and treatment of bleeding in patients with hemophilia A caused by factor VIII deficiency, should be placed in the lock as it is expected to cost more than EUR 20 million per year...The Institute found an indication extension to Roche’s Alecensa (alectinib) would exceed EUR 50,000 per patient and estimates the annual cost will exceed EUR 10,000,000...According to ZIN, the annual cost of Rybrevant would exceed EUR 20,000,000, including future extended indications expected in late 2024 and early 2025...Lastly, the Institute recommended including AstraZeneca’s Truqap (capivasertib) in the lock....ZIN underscores that AZ’s treatment would exceed annual costs of EUR 20,000,000."

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

Sobi to present new data across its haematology portfolio at the 2024 EHA congress (Cision) - "Sobi will present data at the EHA (European Haematology Association) hybrid congress, taking place in Madrid on the 13-16th June, highlighting the company’s dedication to advancing treatments for rare and debilitating haematological diseases. The congress will feature Sobi's latest advances in the treatment of immune thrombocytopenia (ITP), myelofibrosis, haemophilia A, and paroxysmal nocturnal haemoglobinuria (PNH)....'Our ongoing efforts to enhance care and address the unique challenges faced by young patients with ITP will be highlighted with the primary data from the avatrombopag phase 3 paediatric study.'"

Clinical data • Hematological Disorders • Hemophilia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Paroxysmal Nocturnal Hemoglobinuria • Thrombocytopenia

Indirect comparison of prophylaxis efficacy between simoctocog alfa and efanesoctocog alfa in severe hemophilia A and their cost in the United States (ISTH 2024) - "After matching, the percentage of patients with zero bleeds and mean total ABR were similar between the 2 regimens (Figure 1). The mean weekly dose was significantly higher in patients treated with simoctocog alfa versus efanesoctocog alfa (98.3 IU/kg vs 52.2 IU/kg; p< 0.001). The median dosing interval in the NuPreviq study was 3.5 days; patients in the XTEND-1 study were treated once-weekly."

Clinical • Hematological Disorders • Hemophilia • Rare Diseases

Interim analysis of joint outcomes in adult and adolescent patients with severe hemophilia A receiving efanesoctocog alfa during the phase 3 XTEND-ed long-term extension study (ISTH 2024) - P3 | "Arm A of XTEND-ed enrolled 146 participants from XTEND-1 (additional mean [standard deviation, SD] treatment duration 82.5 [14.3] weeks). By Month 12 in XTEND-ed, joint health had improved or been maintained in evaluable participants compared with XTEND-1 baseline, as measured by HJHS total score, total joint score, and subdomain scores (Figure, Table). The HJHS domain with the greatest mean (SD) change from baseline to Month 12 was flexion loss -0.6 (2.7)."

Clinical • P3 data • P3 data: top line • Cardiovascular • Hematological Disorders • Hemophilia • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Pain • Rare Diseases • Rheumatology • Thrombosis

Perioperative management with efanesoctocog alfa in adults, adolescents, and children with severe hemophilia A in the phase 3 XTEND clinical program (ISTH 2024) - P3 | "Forty-one participants ( < 18 years, n=9; ≥18 years, n=32) underwent 49 major surgeries (Table 1); 23 were orthopedic surgeries, with knee arthroplasty most common (n=12). All single 50 IU/kg pre-operative doses maintained hemostasis during major surgery. Median (range) number of doses per major surgery was 4.0 (1–7) during the perioperative period (Days −1 to 14)."

Clinical • P3 data • Hematological Disorders • Hemophilia • Musculoskeletal Diseases • Orthopedics • Pain • Rare Diseases

Achieving More Days Near Normal with ALTUVIIIO®: Clinical Trial Outcomes & Real-World Experience (ISTH 2024) - "Sponsored by Sanofi"

Clinical • Real-world • Real-world evidence • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Pediatric Perspective: A Clinical Discussion on ALTUVIIIO for Kids (ISTH 2024) - "Sponsored by Sanofi"

Clinical • Pediatrics

Long-term outcomes with efanesoctocog alfa prophylaxis for previously treated children with severe hemophilia A, an interim analysis of the phase 3 XTEND-ed study (ISTH 2024) - P3 | "Seventy-one of 74 males (96%) rolled over from XTEND-Kids to XTEND-ed. The mean (standard deviation [SD]) efficacy period was 35.8 (14.1) weeks. No FVIII inhibitors were detected."

Clinical • P3 data • P3 data: top line • Hematological Disorders • Hemophilia • Rare Diseases

First interim analysis of clinical outcomes in adults and adolescents with severe hemophilia A receiving efanesoctocog alfa prophylaxis in XTEND-ed, a phase 3 long-term extension study (ISTH 2024) - P3 | "A total of 146 patients (including 1 female) rolled over from XTEND-1 to XTEND-ed (12–17 years, n=21; 18–64 years, n=120; ≥65 years, n=5). Mean (range) treatment duration in XTEND-ed was 82.5 (14.1–103.6) weeks. FVIII inhibitors were not detected."

Clinical • Clinical data • P3 data • P3 data: top line • Hematological Disorders • Hemophilia • Rare Diseases

FREEDOM: A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Weekly With Efanesoctocog Alfa (clinicaltrials.gov) - P3 | N=93 | Active, not recruiting | Sponsor: Swedish Orphan Biovitrum | Recruiting ➔ Active, not recruiting

Enrollment closed • Hematological Disorders • Hemophilia • Rare Diseases

Efanesoctocog alfa prophylaxis outcomes in European patients from the XTEND-1 trial (BSH 2024) - P3 | "In European patients, efanesoctocog alfa improved bleed protection versus prestudy prophylaxis. Results were consistent with the XTEND-1 primary analysis. Funded by Sobi and Sanofi."

Clinical • Hematological Disorders • Hemophilia • Pain • Rare Diseases

Practical challenges associated with efanesoctocog alfa (ALTUVIIIO) prophylaxis in a 19-month-old male with severe hemophilia A. (PubMed, Pediatr Blood Cancer) - No abstract available

Journal • Hematological Disorders • Hemophilia • Rare Diseases

"Ultra-Long FVIII: A Major Step Forward to a Hemophilia-Free Mind". (PubMed, J Thromb Haemost) - "Leveraging expertise gained with fusion to immunoglobulin Fc fragments, disconnecting FVIII from endogenous von Willebrand factor (via a D'-D3 fragment), and benefiting from the pharmacokinetic prolongation provided by the addition of hydrophilic polypeptides, efanesoctocog alfa opens a new era in the treatment of hemophilia A. The term Ultra-Long (UL) FVIII has been proposed to designate it and differentiate it from EHL FVIII. The level of FVIII correction within the normal range for several days provided by this molecule should allow an increasing number of patients to mentally and physically free themselves from the physical and psychological constraints of hemophilia A. Certainly, the constraint of weekly intravenous infusions persists, but is compensated by a correction of hemostasis whose amplitude and duration remain unmatched by other therapeutic options currently available."

Journal • Review • Hematological Disorders • Hemophilia • Rare Diseases

Sobi receives positive CHMP opinion recommending approval of efanesoctocog alfa for once-weekly treatment of haemophilia A (PRNewswire) - "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. Efanesoctocog alfa is a once-weekly and high-sustained factor VIII replacement therapy for patients of all ages and any disease severity....The recommendation from the CHMP is based on the results from the pivotal phase 3 studies: XTEND-1 in adults and adolescents and XTEND-Kids in children, which evaluated the efficacy and safety of efanesoctocog alfa."

European regulatory • Genetic Disorders • Hemophilia

REAL-WORLD EXPERIENCES WITH ALTUVIIIO [ANTIHEMOPHILIC FACTOR (RECOMBINANT), FC- VON WILLEBRAND FACTOR XTEN FUSION PROTEIN]: A FIRST-IN-CLASS TREATMENT OPTION (SPONSORED BY SANOFI) (THSNA 2024) - "Sponsored by SANOFI Join us for a conversation about the mechanism of extension, pharmacokinetics, efficacy, dosing and safety information from the XTEND-1 clinical trial, as well as real-world clinical cases with ALTUVIIIO. This presentation will feature a live patient perspective."

Clinical • Real-world • Real-world evidence

DESIGN OF A PROSPECTIVE, OBSERVATIONAL, MULTICENTER STUDY OF THE EFFECTIVENESS OF EFANESOCTOCOG ALFA ON LONG-TERM JOINT HEALTH IN PATIENTS WITH HEMOPHILIA A IN THE UNITED STATES AND JAPAN (THSNA 2024) - P | "Perioperative use–specific outcomes will be recorded across cohorts A and B.ConclusionsThe real-world experience of participants receiving efanesoctocog alfa in this study will provide insight into the long-term clinical impact of high-sustained factor levels including joint health. This study will also provide data on participants previously underrepresented in clinical trials."

Clinical • Observational data • Hematological Disorders • Hemophilia • Musculoskeletal Diseases • Rare Diseases • Rheumatology