Esperoct (antihemophilic factor (recombinant), glycopegylated-exei) / Novo Nordisk - LARVOL DELTA

FVIII half-life products: A real-world experience. (PubMed, Thromb Res) - "EHL FVIII products offer significant clinical benefits, reducing the burden of frequent infusions and improving adherence while maintaining effective bleeding control. Despite these advancements, comprehensive evaluations of cost, safety, and long-term outcomes are essential to optimize their integration into haemophilia care."

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

[Translated article] Pharmacokinetic-guided switching from standard half-life factor VIII to extended half-life pegylated factor VIII in haemophilia A therapy in clinical practice. (PubMed, Farm Hosp) - "The pharmacokinetically guided switch from standard half-life FVIII to pegylated FVIII demonstrated significant clinical benefits with reduced bleeding rates and improvements in joint health. Additionally, improvements in pharmacokinetic parameters were observed, allowing for reduced treatment burden by decreasing administration frequency, as well as lower consumption and costs."

Journal • PK/PD data • 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 | Recruiting | Sponsor: Swedish Orphan Biovitrum | Trial completion date: Apr 2025 ➔ Sep 2025 | Trial primary completion date: Mar 2025 ➔ Aug 2025 | Not yet recruiting ➔ Recruiting

Enrollment open • Trial completion date • Trial primary completion date • Hematological Disorders • Hemophilia • Hemophilia A • 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

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

Pharmacokinetic-guided switching from standard half-life factor VIII to extended half-life pegylated factor VIII in the therapy of hemophilia A in clinical practice. (PubMed, Farm Hosp) - "The pharmacokinetically guided switch from standard half-life FVIII to pegylated FVIII demonstrated significant clinical benefits with reduced bleeding rates and improvements in joint health. Additionally, improvements in pharmacokinetic parameters were observed, allowing for reduced treatment burden by decreasing administration frequency, as well as lower consumption and costs."

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

Real-world insights into the management of hemophilia A in Italy: treatment patterns and healthcare resource utilization. (PubMed, Blood Res) - "These findings revealed an extensive use of EHL FVIII products, suggesting growing efforts from clinicians to optimize prophylactic strategies and achieve better bleeding protection."

HEOR • Journal • Real-world • Real-world evidence • 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

Evaluation of plasmin generation in severe haemophilia A patients treated with novel therapies (ISTH 2024) - "Aims: To evaluate PG kinetics pre and post treatment with an extended half-life recombinant factor VIII concentrate (rFVIII-EHL) and factor VIII (FVIII) bispecific antibody (FVIII-bsAb) in SHA A calibrated plasmin generation assay (PGA) was performed on samples from healthy volunteers (n=35), SHA patients treated with rFVIII-EHL, turoctocog alfa pegol (n=19) and SHA patients treated with FVIII-bsAb, emicizumab (n=19), after informed consent and ethics approval. There were no statistical differences in core PG parameters (EPP, peak and velocity) between HV and pre-treatment SHA (FVIII ≤5 IU/dL) samples (Table 1). A significant weak correlation (r=0.348, p=0.040) was seen in HV between EPP and FVIII levels (FVIII:62-220 IU/dL). Interestingly, no correlation (r= 0.052, p=0.831) was observed between EPP and FVIII levels (FVIII:97-171 IU/dL) in post-EHL rFVIII treatment samples."

Clinical • Hematological Disorders • Hemophilia • Rare Diseases

Real world evidence after switching to turoctocog alfa pegol in patients from Iberian countries (ISTH 2024) - "Since February 2020, 95 patients with hemophilia A started prophylaxis with turoctocog alfa pegol. The median follow-up was 32.83 months (range 5.37 – 48.03). The mean age was 34.05 years (range 15-56)."

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

ACCURATE MEASUREMENT OF FACTOR VIII ACTIVITY AND INHIBITORS IN THE PRESENCE OF MIM8 (THSNA 2024) - " To assess FVIII activity of SHL and EHL products, severe HA plasma was spiked with ADVATE®, Novoeight®, Esperoct®, or ELOCTATE® (5, 10, 15, 20, and 100 IU/dL final concentrations) and Mim8 (0, 3, 6, and 12 µg/mL final concentrations). FVIII activity of SHL and EHL products was accurately measured in the presence of Mim8 using bovine CSAs. Using FVIII CSAs with bovine reagents, FVIII inhibitor levels up to approximately 5.0 BU were accurately measured in HA plasma in the presence of up to 40 µg/mL Mim8."

Hematological Disorders • Hemophilia • Rare Diseases

Cost-Effectiveness Analysis of Turoctocog Alfa Pegol in Patients With Hemophilia A Without Inhibitors in Colombia (ISPOR 2024) - "Given the assumptions of the study, the use of turoctocog alfa pegol for hemophilia A patients without inhibitors in Colombia results in lower treatment costs of spontaneous bleeds, fewer total bleeds and more QALYs."

Clinical • Cost effectiveness • HEOR • Hematological Disorders • Hemophilia • Rare Diseases

Noninterventional study assessing joint health in persons with hemophilia A after switching to turoctocog alfa pegol: design of pathfinderReal. (PubMed, Res Pract Thromb Haemost) - P | "An exploratory endpoint is included to measure the number of patients achieving improved Hemophilia Joint Health Score from the initiation of N8-GP until the end of the study. The pathfinderReal study will provide insights regarding the impact of N8-GP on joint health in persons with hemophilia A in a real-world setting."

Journal • Observational data • Hematological Disorders • Hemophilia • Osteoarthritis • Pain • Rare Diseases

Reduced recovery of turoctocog alfa pegol is not always associated with antidrug antibodies against polyethylene glycol: data from three patients (WFH 2024) - No abstract available

Clinical

Reduced recovery of turoctocog alfa pegol is not always associated with antidrug antibodies against polyethylene glycol: data from three patients (WFH 2024) - No abstract available

Clinical

POSITIVE CLINICAL IMPACT AFTER SWITCHING TO TUROCTOCOG ALFA PEGOL: IBERIAN EXPERIENCE (EAHAD 2024) - No abstract available

Clinical

PHARMACOKINETIC ANALYSIS IN PATIENTS WITH HEMOPHILIA A IN PROPHYLAXIS WITH TUROCTOCOG ALFA PEGOL IN ONE CENTRE IN SPAIN (EAHAD 2024) - No abstract available

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

The efficacy, safety, and pharmacokinetics of N8-GP in previously treated Chinese patients with haemophilia A: results from the phase 3b pathfinder10 study (EAHAD 2024) - No abstract available

Clinical • P3 data • PK/PD data • Hematological Disorders • Hemophilia • Rare Diseases

Real-World Experience of People with Hemophilia A Receiving Turoctocog Alfa Pegol (N8-GP): Results from a Patient Experience Survey. (PubMed, Patient Prefer Adherence) - "Patients believed that N8-GP can offer a long-term impact in areas such as ability to perform day-to-day activities (68%), independence to live like a person without hemophilia (63%), ability to travel (60%) with a feeling of optimism and hopefulness (82%). Lower frequency of injections, storage flexibility and improved QoL drove satisfaction and preference for N8-GP over previous treatments among patients with HA."

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

pathfinder6: Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A (clinicaltrials.gov) - P3 | N=125 | Completed | Sponsor: Novo Nordisk A/S | Phase classification: P3a ➔ P3

Phase classification • Hematological Disorders • Hemophilia • Pediatrics • Rare Diseases

Cases of less-than-expected FVIII activity in previously treated patients during post-marketing surveillance of N8-GP. (PubMed, Haemophilia) - "In conclusion, the 14 reported cases of less-than-expected FVIII activity, without presence of detectable FVIII inhibitors, presented with heterogenous characteristics, and wide variations in FVIII activity and anti-PEG antibody titre. FVIII activity returned to the expected range after switching to alternative FVIII products. In line with WFH guidelines, monitoring of FVIII activity can ensure FVIII activity in the expected range. The safety surveillance of N8-GP continues."

Journal • P4 data • Hematological Disorders • Hemophilia • Rare Diseases

pathfinder6: Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A (clinicaltrials.gov) - P3a | N=125 | Completed | Sponsor: Novo Nordisk A/S | Active, not recruiting ➔ Completed

Trial completion • Hematological Disorders • Hemophilia • Pediatrics • Rare Diseases

The immunogenicity, safety, and efficacy of N8-GP in previously untreated patients (PUPs) with severe hemophilia A: pathfinder6 end-of-trial results. (PubMed, J Thromb Haemost) - P3a | "Long-term end-of-trial PUP N8-GP prophylaxis data were reported. The inhibitor incidence were consistent with previous results. Median ABR during prophylaxis was 1.4. There were no lasting clinical impacts or safety concerns for patients with an observation of temporarily decreased IR. These data indicate that PUPs respond well to long-term N8-GP treatment."

Journal • Hematological Disorders • Hemophilia • Pediatrics • Rare Diseases

Mim8 interference on the measurement of FVIII Standard and Extended half-life products using selected FVIII Chromogenic Assays (ISTH 2023) - " Severe Hemophilia A plasma (HRF, Inc.) spiked with ADVATE®, Novoeight®, Esperoct® or ELOCTATE® to concentrations of 0.050, 0.100, 0.150, 0.200, and 1.000 IU/mL and Mim8 to 0, 3, 6 and 12 µg/mL, were used to investigate Mim8 interference. The bovine CSA showed no notable interference at all FVIII/Mim8 concentrations for all products (Fig 1). For the bovine/human CSA (Fig 2), FVIII levels ≤0.20 IU/mL, showed a Mim8 dose dependent interference increase with the largest fold increase (2.4 to 4.5, ADVATE®) at low FVIII levels (0.05 IU/mL). More interference by Mim8 was observed in SHL products."

Hematological Disorders • Hemophilia • Rare Diseases

A UK Single Centre Experience of Switching Adolescents with Severe Haemophilia A to Turoctocog Alfa Pegol (ISTH 2023) - "Four patients switched to TAP and details are summarised in Table 1.Patient 1 has no spontaneous bleeds and continues with their new prophylactic regimen.Patient 2 had a traumatic left elbow soft tissue bleed after ED 3 (of TAP). A 40-hour FVIII level was < 0.01IU/mL and negative FVIII inhibitor. He treated with daily TAP before returning to EOD TAP prophylaxis which continues.Patient 3 had a spontaneous left thigh bleed after ED 6 (of TAP)."

Clinical • Hematological Disorders • Hemophilia • Rare Diseases