Low inhibitor rate with octanate® in previously untreated patients (PUPs) with severe haemophilia A22.01.2018
Lachen, Switzerland, January 22nd, 2018: Octapharma is delighted to announce the publication of final data from a Good Clinical Practice (GCP) trial in PUPs in the internationally renowned medical journal Haemophilia. The study assessed the immunogenicity, efficacy, safety and tolerability of octanate® in PUPs with severe haemophilia A.
PUPs with haemophilia A are at greatest risk of inhibitor development. Approximately 35% develop inhibitors, which are associated with detrimental health and economic consequences.
In this prospective, multinational study, 51 PUPs with severe haemophilia A received octanate® exclusively as replacement therapy, either prophylactically or for on-demand treatment of bleeds, for a total of 100 exposure days (EDs) or 5 years.
Five (9.8%) of the 51 patients developed inhibitors, 4 (7.8%) of which were high titre (≥5 BU/mL) and 1 low titre (<5 BU/mL). All inhibitors developed during on-demand treatment and all 4 high-titre inhibitors developed within the first 20 EDs. All patients who developed inhibitors had major F8 gene defects (intron 22 inversions or large deletions of exons 7-12) that are associated with a high risk of inhibitor development. Haemostatic efficacy was rated as “excellent” for 99.6% of all infusions. Tolerability was rated “very good” for 99.98% of infusions. No complications were reported during 23 surgical procedures.
Early and efficient prophylaxis is key to successful long-term management of patients with haemophilia A. These new data demonstrate that octanate® is efficacious and associated with a low rate of inhibitor development in PUPs, including those undergoing surgical procedures.
Larisa Belyanskaya, Head of IBU Haematology, said: “We are very excited by the low inhibitor rates and the excellent efficacy and tolerability achieved with octanate® in this particularly challenging patient population. These data confirm the clinical benefits of octanate® in PUPs with haemophilia A and add to the already extensive clinical experience with octanate® gained over the past 20 years.”
Olaf Walter, Board Member at Octapharma, added that “This publication is a further important step towards Octapharma’s goal of enabling patients with coagulation disorders to live a normal life.”
Octapharma would like to thank everyone involved in the study, in particular the patients and their families, without whom this research would not be possible.
Klukowska A, Komrska V, Vdovin V, et al. Low incidence of factor VIII inhibitors in previously untreated patients with severe haemophilia A treated with octanate®: Final report from a prospective study. Haemophilia 2018; doi.org/10.1111/hae.13385
octanate® is a human, plasma‑derived, high‑purity, freeze‑dried, double virus‑inactivated coagulation factor VIII (FVIII) concentrate for intravenous administration. Coagulation FVIII present in octanate® is bound to its natural stabiliser, von Willebrand factor (VWF), in a VWF/FVIII ratio of approximately 0.4. Therefore, no additional stabilisers are required during manufacturing. octanate® is available in 250 IU, 500 IU and 1000 IU presentations. octanate® has been marketed since 1998 and is approved in more than 85 countries for the treatment and prophylaxis of bleeding in patients with all types of haemophilia A, including for surgical procedures, and in 40 countries for immune tolerance induction. Considerable clinical experience exists with octanate®, with ~10 billion international units (IU) infused worldwide as of April 2017.
About Haemophilia A
Haemophilia A is an X-linked hereditary disorder caused by FVIII deficiency which, if left untreated, leads to haemorrhages in muscles and joints and consequently to arthropathy and severe morbidity. FVIII replacement prophylactic treatment reduces the number of bleeding episodes and the risk of permanent joint damage. This disorder affects one in every 5,000 to 10,000 men worldwide. Globally, 75% of haemophilia cases are left undiagnosed or untreated. The development of neutralising FVIII antibodies (FVIII inhibitors) against infused FVIII represents the most serious treatment complication. The cumulative risk of FVIII inhibitor development is reported to be currently up to 39%.
Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines. As a family-owned company, Octapharma believes in investing to make a difference in people’s lives and has been doing so since 1983; because it’s in our blood.
In 2016, the Group achieved €1.6 billion in revenue, an operating income of €383 million and invested €249 million to ensure future prosperity. Octapharma employs more than 7,100 people worldwide to support the treatment of patients in 113 countries with products across three therapeutic areas:
Haematology (coagulation disorders)
Immunotherapy (immune disorders)
Octapharma owns six state-of-the-art production facilities in Austria, France, Germany, Mexico and Sweden.
For more information visit www.octapharma.com