Idelvion (albutrepenonacog alfa) / CSL Behring - LARVOL DELTA

Cost-Savings Analysis of Fitusiran Prophylaxis: Reducing Breakthrough Bleeding Treatment Expenditure in the Kingdom of Saudi Arabia (ISPOR-EU 2025) - P3 | "For people with haemophilia (PwH) A without inhibitors, episodic treatments comprised octocog alfa, efmoroctocog alfa and rurioctocog alfa pegol for PwH A and nonacog alfa, albutrepenonacog alfa and eftrenonacog alfa for PwH B. Treatments included for PwH with inhibitors were factor VIII inhibitor bypassing activity (FEIBA) and eptacog alfa. In the KSA, fitusiran AT-DR prophylaxis may considerably reduce breakthrough bleed management costs in PwH versus CFC/BPA prophylaxis. Cost savings are predicted to be more substantial in PwH with inhibitors than in those without inhibitors."

HEOR • Hematological Disorders • Hemophilia • Rare Diseases

Cost-Savings Assessment of Fitusiran Prophylaxis in Minimizing Breakthrough Bleeding Treatment Expenses in the United Arab Emirates (ISPOR-EU 2025) - P3 | "The episodic treatments included were efmoroctocog alfa, octocog alfa and rurioctocog alfa pegol for people with haemophilia (PwH) A without inhibitors and albutrepenonacog alfa, nonacog alfa and eftrenonacog alfa for PwH B without inhibitors...A scenario analysis examined the impact of vial sharing. Among PwH without inhibitors, fitusiran AT-DR enabled per-bleed savings ranging from UAE Dirham (AED) 4,625 (efmoroctocog alfa) to AED 11,521 (rurioctocog alfa pegol) in PwH A and from AED 8,935 (nonacog alfa) to AED 30,053 (albutrepenonacog alfa) in PwH B. In PwH with inhibitors, fitusiran AT-DR usage generated per-bleed savings of AED 71,846 (FEIBA) to AED 90,761 (eptacog alfa). In the UAE, fitusiran AT-DR prophylaxis may considerably reduce costs associated with episodic treatments for breakthrough bleeds in PwH compared with CFC/BPA prophylaxis. PwH with inhibitors might have larger cost savings than those without inhibitors."

HEOR • Hematological Disorders • Hemophilia • Rare Diseases

Safety, Efficacy and Treatment Patterns of rIX-FP in Previously Untreated Paediatric Haemophilia B Patients: A Retrospective Chart Review in Japan. (PubMed, Haemophilia) - "This study provides real-world insights into rIX-FP use in paediatric PUPs, demonstrating its favourable safety, effective prophylaxis, and lower IVR in infants."

Journal • Retrospective data • Hematological Disorders • Hemophilia • Hemophilia B • Pediatrics • Rare Diseases

Comparing Real-World Outcomes of Prophylaxis with Extended Half-life Factor IX (rIX-FP vs. rFIXFc and N9-GP) for Haemophilia B: An Analysis of Medical Chart Data from Germany. (PubMed, Adv Ther) - "rIX-FP prophylaxis was associated with significantly lower FIX consumption and numerically (but not significantly) lower bleeding rates compared with rFIXFc. Compared to N9-GP, prophylaxis with rIX-FP was associated with similar FIX consumption and significantly lower bleeding rates."

Journal • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Real-World Prophylaxis Outcomes with rIX-FP and rFIXFc for Males with Hemophilia B: Pooled Analysis of Medical Chart Data from Germany and Italy. (PubMed, Adv Ther) - "rIX-FP prophylaxis was associated with reduced FIX consumption versus rFIXFc and offered equally effective or potentially improved bleed protection. Additionally, PwHB who switched to rIX-FP achieved significant decreases in FIX consumption, ABR, and AjBR compared with their prior FIX product."

Journal • Real-world evidence • Retrospective data • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Relationship between Dose, Factor IX Activity Levels and Bleeding Probability for rIX-FP Prophylaxis in Hemophilia B: A Repeated Time-to-Event Analysis. (PubMed, Clin Pharmacol Ther) - "Variability in bleeding hazard between individuals with similar FIX activity levels was substantial (182%). Simulations showed that targeting trough FIX activity levels to 20 IU/dL resulted in a median annual bleeding rate (ABR) of zero (range: 0-3)."

Journal • Hematological Disorders • Hemophilia • Hemophilia B • Mood Disorders • Rare Diseases

Performance of a Drug-Specific Calibration Curve for Monitoring Treatment With Albutrepenonacog Alfa: A Multicenter Study in Argentina. (PubMed, Int J Lab Hematol) - "Idelvion-specific calibration curve showed better performance and lower CV rates independently of the aPTT reagent or the platform used. Calibration using this specific standard might allow more laboratories to obtain acceptable FIX values when processing NC and LC levels and patients' plasmas."

Clinical • Journal • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Previously Treated With FIX Therapy (clinicaltrials.gov) - P3 | N=23 | Active, not recruiting | Sponsor: CSL Behring | Recruiting ➔ Active, not recruiting

Enrollment closed • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Surgical use of rIX-FP in patients with haemophilia B: French real-world data (EAHAD 2025) - No abstract available

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

Long-term, real-world use of rIX-FP in adult patients with haemophilia B in Italy: outcomes of more than 4 years of retrospective-prospective follow-up (IDEAL Part A+B) (EAHAD 2025) - No abstract available

Real-world • Real-world evidence • Retrospective data • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

An update on rIX-FP prophylaxis use in paediatric patients with haemophilia B: French real-world data (EAHAD 2025) - No abstract available

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

OrPHEe: Observatory of Patients With Haemophilia B Treated by IdElvion® (clinicaltrials.gov) - P=N/A | N=222 | Active, not recruiting | Sponsor: CSL Behring | Recruiting ➔ Active, not recruiting | N=100 ➔ 222

Enrollment change • Enrollment closed • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

ORPHEE: A Real-World Study on rIX-FP Prophylaxis Use in Adolescent/Adult Patients With Hemophilia B. (PubMed, Eur J Haematol) - "This analysis confirmed that switching to rIX-FP allows for reducing injection frequency and FIX consumption while maintaining good bleed protection."

Journal • Real-world • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Real-world experience of rIX-FP prophylaxis at dosing intervals of 14 days or more in adult patients with haemophilia B in Italy - Results from IDEAL Part B. (PubMed, Haemophilia) - No abstract available

Journal • Real-world • Real-world evidence • Hematological Disorders • Hemophilia • Rare Diseases

Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Previously Treated With FIX Therapy (clinicaltrials.gov) - P3 | N=23 | Recruiting | Sponsor: CSL Behring | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Disorders • Hemophilia • Rare Diseases

FIX Post infusion monitoring surveys: Results and analysis from the updated UK NEQAS BC Haemophilia programmes 2024 (ISTH 2024) - "Data was returned from 47 centres. Centres performed either one stage assays (OSA), chromogenic assays (CA) or both. OSA Alprolix Median value, 40 IU/dL with CV = 8.7%; Benefix Median value, 14.3 IU/dL with CV = 13.6%; Idelvion Median value, 20.4 IU/dL with CV = 14.5% and Refixia Median value, 15.4 IU/dL with CV = 18.2% CA Alprolix Median value, 35.7 IU/dL; Benefix Median value, 11.7 IU/dL; Idelvion Median value, 24.8 IU/dL and Refixia Median value, 13.4 IU/dL."

Hematological Disorders • Hemophilia • Rare Diseases

FIX is localised in the vessel wall and can generate thrombin when in complex with collagen IV in human synovial tissues (ISTH 2024) - "On surfaces with the FIX-collagen complex present, FIX-DP generated a peak thrombin concentration comparable to PNP. FIX binding was confirmed by a chromogenic activity assay and a dose-dependent effect of FIX coating concentration was observed. Collagen plates coated with extended half-life concentrates, Alprolix and Idelvion, generated a significant increase in the endogenous thrombin potential (ETP), when compared to short-acting concentrate, Replenine and Benefix (* p < 0.01)."

Hematological Disorders • Hemophilia • Rare Diseases • Thrombosis

Evaluation of recovery of factor IX replacement therapy with HemosIL Chromogenic Factor IX (ISLH 2024) - "HemosIL Chromogenic Factor IX demonstrated the ability to correctly measure FIX activity in plasma pools with 3 FIX replacement therapies: Alpha Nine SD, BeneFIX, and REBINYN. Results with albutrepenonacog alpha (recombinant FIX fused with recombinant human albumin; Idelvion) demonstrated over recovery of 59% when compared to the theoretical value based on the potency assignment by the manufacturer. In 2019 Rosén et al."

Hematological Disorders • Hemophilia • Rare Diseases

SPANISH REAL-LIFE EXPERIENCE WITH rIX-FP IN SURGERIES AND PROPHYLAXIS (ISTH 2024) - "All patients were on treatment with rFIX-SHL (short half-life) or plasma derivated factor and the average follow-up was 33 months. There were 9 patients with severe HB and 4 with moderate HB, of which nine were previously treated on a prophylactic regimen and four on-demand regimen. A description of the patients is shown in Table 1."

Clinical • Surgery • Hematological Disorders • Hemophilia • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Pain • Rare Diseases • Rheumatology

Audit of comprehensive care of haemophilia B against World Federation of Haemophilia guidelines (BSH 2024) - "5/5 switched prophylaxis from standard to extended half-life products (Alprolix, Refixia or Idelvion) due to breakthrough bleeds (4/5) or compliance (1/5). Fifty-six patients with haemophilia B were identified. Data collection included 19 patients: 7 mild, 5 moderate, 5 severe and 2 carriers from 2018 to 2023. Prophylaxis: Hundred percent of patients with severe haemophilia B were on prophylaxis."

Hematological Disorders • Hemophilia • Musculoskeletal Diseases • Musculoskeletal Pain • Pain • Rare Diseases

Recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in Chinese subjects with hemophilia B previously treated with FIX therapy (clinicaltrialsregister.eu) - P3 | N=23 | Sponsor: CSL Behring GmbH

New P3 trial • Hematological Disorders • Hemophilia • Rare Diseases

Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Previously Treated With FIX Therapy (clinicaltrials.gov) - P3 | N=23 | Not yet recruiting | Sponsor: CSL Behring

New P3 trial • Hematological Disorders • Hemophilia • Rare Diseases

OrPHEe: Observatory of Patients With Haemophilia B Treated by IdElvion® (clinicaltrials.gov) - P=N/A | N=100 | Recruiting | Sponsor: CSL Behring | Trial completion date: Sep 2026 ➔ Dec 2027 | Trial primary completion date: Sep 2026 ➔ Dec 2027

Trial completion date • Trial primary completion date • Hematological Disorders • Hemophilia • Rare Diseases

Six-Year, Real-World Use of Prophylaxis with Recombinant Factor IX-Albumin Fusion Protein (rIX-FP) in Persons with Hemophilia B: A Single-Center Retrospective-Prospective Study. (PubMed, J Clin Med) - "Very low bleeding rates (most traumatic bleeds and the last quartile of the infusion interval), improved orthopedic and pain scores, unchanged HEAD-US scores and problem joints, and high treatment adherence (>90%) and satisfaction were registered. Personalized, carefully adjusted rIX-FP regimens contribute to the diffusion and optimization of prophylaxis in persons with severe and moderate hemophilia B, with long-term favorable bleeding, joint, and patient-reported outcomes."

Journal • Real-world • Real-world evidence • Retrospective data • Hematological Disorders • Hemophilia • Orthopedics • Pain • Rare Diseases

Safety and Efficacy of Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP) in Previously Untreated Patients with Hemophilia B. (PubMed, TH Open) - "Conclusion  This study provides data to support the safety and efficacy of rIX-FP in PUPs requiring on-demand or prophylactic treatment for moderately severe/severe hemophilia B, consistent with results in previously treated patients. Overall, 1/12 patients developed an inhibitor against FIX."

Journal • Cardiovascular • Hematological Disorders • Hemophilia • Pediatrics • Rare Diseases