Pooled analysis of long-term efficacy and safety of simoctocog alfa in previously treated patients with haemophilia A

M. Mathias¹, A. Borchiellini², F. Peyvandi³, A. Chistolini⁴, R. Marino⁵, A. Rocino⁶, M. Sol Cruz⁷, S. Carolina Oliva⁸, A. Molina Pantoja⁹, C. Grimley¹⁰, S. Bayart¹¹, A. Klukowska¹², T. Lissitchkov¹³, S. Halimeh¹⁴, S. Werner¹⁵, G. Pezeshki¹⁵, S. Knaub¹⁶, C. Kessler¹⁷

¹Great Ormond Street Hospital for Children, NHS Foundation Trust, London, England, United Kingdom, ²A.O.U. Città della Salute e della Scienza di Torino, Torino, Piemonte, Italy, ³Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Università degli Studi di Milano, Department of Pathophysiology and Transplantation, and Fondazione Luigi Villa, Milan, Italy., Milan, Lombardia, Italy, ⁴Department of Translational and Precision Medicine, Sapienza University, Rome, Lazio, Italy, ⁵Haemophilia and Thrombosis Centre, University Hospital of Bari, Bari, Puglia, Italy, ⁶Ospedale del Mare Hospital, Napoli, Campania, Italy, ⁷Centro Diabetologico del Norte (CEDINOR), Salta, Salta, Argentina, ⁸PEDIAS INC, Guatemala City, Alta Verapaz, Guatemala, ⁹Teodoro Maldonado Carbo Specialty Hospital, Guayaquil, Guayas, Ecuador, ¹⁰Nottingham Haemophilia Comprehensive Care Centre, Nottingham, England, United Kingdom, ¹¹Hôpital Pontchaillou, Rennes, Bretagne, France, ¹²Department of Pediatrics, Hematology and Oncology, Warsaw Medical University, Warsaw, Mazowieckie, Poland, ¹³Blood Diseases at Clinical Hematology Clinic, Sofia, Sofiya, Bulgaria, ¹⁴Gerinnungszentrum Rhein-Ruhr, Duisburg, Nordrhein-Westfalen, Germany, ¹⁵Octapharma USA, Paramus, New Jersey, United States, ¹⁶Octapharma AG, Lachen, Schwyz, Switzerland, ¹⁷Georgetown University Medical Center, Washington, District of Columbia, United States

Abstract Number: PB1149

Meeting: ISTH 2022 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical

Background: Simoctocog alfa (Nuwiq®) is a human cell line-derived recombinant human FVIII that was developed with the aim to provide a low immunogenicity product with excellent haemostatic efficacy for people with haemophilia A. The efficacy and safety of Nuwiq® have been demonstrated in clinical trials of previously treated patients (PTPs) and previously untreated patients with severe haemophilia A.

Aims: To assess the long-term immunogenicity, safety and efficacy of Nuwiq® in a pooled analysis of PTPs with haemophilia A.

Methods: Data from patients who received Nuwiq® prophylaxis for ≥100 exposure days in two observational and four interventional post-authorisation studies were pooled. These studies included male PTPs with severe haemophilia A (FVIII:C < 1%); one study included patients with FVIII:C ≤2%. Cut-off dates were used for studies ongoing at the time of database lock.

Results: Demographics of the 216 patients included in the analysis are shown in Table 1. There were no reports of inhibitor development in any patient. Twelve adverse drug reactions occurred in 8 patients; one was serious (mild pyrexia requiring hospitalisation) but resolved and treatment was continued. No thromboembolic events occurred. The median (interquartile range [IQR]) duration of prophylaxis was 16.8 months (9.4–24.4); 108 (50.0%) patients experienced no spontaneous bleeding episodes (BEs) during prophylaxis. Median (IQR) annualised bleeding rates were 0.11 (0–1.45), 0.50 (0–1.95) and 1.78 (0–4.24) for spontaneous, traumatic and all BEs, respectively. A total of 1011 BEs in 152 patients were treated with Nuwiq®. Most BEs (860 [85.1%]) resolved with ≤2 infusions of Nuwiq®; treatment efficacy was rated excellent or good for the majority (84.3%) of BEs. Of 36 surgeries with a postoperative rating by a haematologist, 31 (86.1%) were rated successful.

Conclusion(s): These pooled long-term data from PTPs confirm the safety and efficacy of Nuwiq® demonstrated previously in clinical trials.

Table

Table 1. Demographics and baseline characteristics of patients in pooled analysis

To cite this abstract in AMA style:

Mathias M, Borchiellini A, Peyvandi F, Chistolini A, Marino R, Rocino A, Sol Cruz M, Carolina Oliva S, Molina Pantoja A, Grimley C, Bayart S, Klukowska A, Lissitchkov T, Halimeh S, Werner S, Pezeshki G, Knaub S, Kessler C. Pooled analysis of long-term efficacy and safety of simoctocog alfa in previously treated patients with haemophilia A [abstract]. https://abstracts.isth.org/abstract/pooled-analysis-of-long-term-efficacy-and-safety-of-simoctocog-alfa-in-previously-treated-patients-with-haemophilia-a/. Accessed October 1, 2023.

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