PB1149 - Pooled analysis of long-term efficacy and safety of simoctocog alfa in previously treated patients with haemophilia A

Tuesday, July 12, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Co-Author(s)

MM

Mary Mathias

Haemophilia Centre Director
Great Ormond Street Hospital for Children, NHS Foundation Trust
London, England, United Kingdom
AB

Alessandra Borchiellini

Doctor, in charge of regional reference centre of haemorrhagic and thrombotic disorders.
A.O.U. Città della Salute e della Scienza di Torino
Torino, Piemonte, Italy
Flora Peyvandi, MD, PhD

Professor of Internal Medicine; Director of the Angelo Bianchi Bonomi Hemophilia & Thrombosis Center
Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy; Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy
Milan, Lombardia, Italy
AC

Antonio Chistolini

Medical Doctor, Haematology
Department of Translational and Precision Medicine, Sapienza University
Rome, Lazio, Italy
RM

Renato Marino

Medical Doctor
Haemophilia and Thrombosis Centre, University Hospital of Bari
Bari, Puglia, Italy
AR

Angiola Rocino

Responsabile UOC di Ematologia e Centro Emofilia e Trombosi
Ospedale del Mare Hospital
Napoli, Campania, Italy
MS

Maria Sol Cruz

Director of CEDINOR
Centro Diabetologico del Norte (CEDINOR)
Salta, Salta, Argentina
SC

Silvana Carolina Oliva

Attending physician
PEDIAS INC
Guatemala City, Alta Verapaz, Guatemala
AM

Alicia Molina Pantoja

Haematologist
Teodoro Maldonado Carbo Specialty Hospital
Guayaquil, Guayas, Ecuador
CG

Charlotte Grimley

Centre Director
Nottingham Haemophilia Comprehensive Care Centre
Nottingham, England, United Kingdom
SB

Sophie Bayart

Praticien hospitalier
Hôpital Pontchaillou
Rennes, Bretagne, France
AK

Anna Klukowska

Associate Professor
Department of Pediatrics, Hematology and Oncology, Warsaw Medical University
Warsaw, Mazowieckie, Poland
TL

Toshko Lissitchkov

Professor
"Specialized Hospital for Active Treatment of Haematological Diseases" EAD
Sofia, Sofiya, Bulgaria
Susan Halimeh, MD

Professor
Gerinnungszentrum Rhein Ruhr, Duisburg, Germany
Duisburg, Nordrhein-Westfalen, Germany
SW

Sylvia Werner

Senior Director Clinical R&D
Octapharma USA
Paramus, New Jersey, United States

Presenting Author(s)

Gita Pezeshki, PhD

Associate Director, Clinical Operations
Octapharma USA
Paramus, New Jersey, United States

Co-Author(s)

SK

Sigurd Franz Knaub, PhD

Senior Vice President Clinical R&D – Haematology
Octapharma AG
Lachen, Schwyz, Switzerland
Craig Kessler, MD

Professor of Medicine and Pathology and Section Chief of Hematology
Georgetown University Hospital
Washington DC, Washington, United States

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.