Octapharma: Low inhibitor rate with octanate® in previously untreated patients (PUPs) with severe haemophilia A
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; https://doi.org/10.1111/hae.13385
About octanate ®
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)
- Critical care
Octapharma owns six state-of-the-art production facilities in Austria, France, Germany, Mexico and Sweden.
For more information visit www.octapharma.com
For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.
Tilaa tiedotteet sähköpostiisi
Haluatko tietää asioista jo ennen kuin ne uutisoidaan? Kun tilaat tiedotteemme, saat ne sähköpostiisi yhtä aikaa suomalaisen median kanssa. Tilauksen voit halutessasi perua milloin tahansa.
Lue lisää julkaisijalta Business Wire
Octapharma Share Promising Preclinical Data for SubQ-8, a Novel Subcutaneous Recombinant FVIII, at WFH 201828.5.2018 10:58 | Tiedote
Octapharma announced today that pre-clinical data for SubQ-8 were presented during a symposium at the recent World Federation of Hemophilia (WFH) 2018 World Congress in Glasgow, UK. SubQ-8, currently under development, is a recombinant FVIII from a human cell line for subcutaneous administration. SubQ-8 is based on Octapharma’s human cell line-derived rFVIII product simoctocog alpha combined with a fragment of the von Willebrand Factor (VWF) protein, and harnesses the protective power of VWF in an innovative approach to facilitate transportation of FVIII into the circulation. Regular intravenous administration of FVIII poses a considerable burden to people with haemophilia A and their families. Alternative routes of administration may help to alleviate some of this burden and improve adherence to prophylaxis therapy. This symposium, entitled ‘Taking FVIII into the future: The development of subcutaneous recombinant human FVIII for the treatment of haemophilia A’, reviewed the challenge
Octapharma present new data on the benefits of Nuwiq® in patients with haemophilia A at the World Federation of Hemophilia 2018 World Congress28.5.2018 10:57 | Tiedote
Octapharma announced today that new data on the benefits of Nuwiq® in patients with haemophilia A were presented during a symposium at the recent World Federation of Hemophilia (WFH) 2018 World Congress in Glasgow, UK. Nuwiq® is a 4th generation human cell line-derived recombinant FVIII (rFVIII) approved for the prevention and treatment of bleeding episodes in patients of all ages with haemophilia A. The symposium, entitled ‘Going further to meet clinical needs: New data with Nuwiq ® ▼ (simoctocog alfa; human-cl rFVIII) from clinical trials and real-world experience’, focused on the challenges within the current haemophilia treatment landscape and opportunities to improve patient care. The symposium, chaired by Craig Kessler, addressed the reality that no two haemophilia A patients are the same. An optimal treatment approach should therefore reflect each individual patient’s profile. Striving towards this goal, two approaches to using pharmacokinetic (PK) data to personalise prophylaxi
Gemalto Announces Collaboration with Qualcomm Technologies to Integrate eSIM Innovation into the Snapdragon Mobile PC Platform28.5.2018 08:00 | Tiedote
Gemalto announces a new collaboration that will see its advanced mobile connectivity solutions integrated with Qualcomm® Snapdragon™ mobile PC platform, a product by Qualcomm Technologies Inc., a subsidiary of Qualcomm Incorporated. This paves the way for iSIM commercialization on a growing range of Always Connected PCs, laptops and tablets. The effort will support the integration of Gemalto’s eSIM technology and remote subscription management solutions, with the new Secure Processing Unit (SPU) on the Snapdragon mobile PC platform. As a result, this innovation will provide seamless LTE and forthcoming 5G connection, extended battery life, and a foundation for consumer applications such as online payments, transport ticketing and authentication to cloud services. This initiative represents the first time an eSIM will be integrated with processing platforms designed to power Always Connected PCs and similar consumer devices. Providing benefits for industry players: OEMs optimize their b
CES Asia 2018 Announces Innovation Award Honorees27.5.2018 23:00 | Tiedote
The Consumer Technology Association (CTA) today announced its Innovation Awards Honorees for CES Asia 2018. Now in its second year, the CES Asia Innovation Awards Program celebrates outstanding product design and engineering in brand-new consumer technology products from CES Asia exhibitors. The program recognizes groundbreaking technologies across 20 product categories, including first-time category artificial intelligence (AI) located primarily in SNIEC, Hall N3 at CES Asia 2018. Additional areas of recognition include augmented reality (AR), virtual reality (VR), content, mobile, vehicle tech, smart home, digital health, wearables and more. CES Asia is a global show with exhibiting companies originating from 23 countries, regions and territories. An elite panel of judges with industry expertise in product design and engineering deliberated on the honorees for this prestigious awards program. The honored products excelled in five criteria: engineering, aesthetics, function and user v
FCF: Greenpeace Allegations Unfair and Deceptive26.5.2018 21:02 | Tiedote
FCF Fishery, Co. President Max Chou today released the following statement regarding recent misleading Greenpeace human rights abuse allegations made against the company: “We realize that as one of the world’s largest marine products integrated supply chain service providers with more than 30 subsidiaries, fishing bases, and shipping agents around the world, we are a prime target for organizations seeking to garner publicity and those not fully aware of the latest development and improvements that are currently taking place in the fishery industry. However, in their efforts to curb egregious human rights abuses, we believe it is unfair and deceptive to lump our company in with those who condone cruelty and neglect of their laborers. “Although we recognize the meaningful work of Greenpeace in exposing and eliminating human trafficking and sustainability abuses, we are equally disappointed that they are implicating FCF in old incidents and cases that have since been in all instances addr
NBC Universal, Havas Group and Endemol Shine Discuss a Future with AI, Blockchain and VR at IBC201825.5.2018 21:20 | Tiedote
As is fitting for the home of technical invention and innovation, IBC2018 will present a broad range of ambitious and timely elements to engage, inform and inspire content creators and owners from around the world that will visit the RAI, Amsterdam from 13-18 September 2018. The must-attend event includes fresh inspiring conference themes that will focus on innovative new platforms, audience engagement and interaction/immersion, as well as a wide-ranging exhibition that will encompass all the latest technologies from AI and VR to advanced cloud-based workflows. Registration for IBC2018 is now open at: https://ibc.itnint.com/IBC18/Online/RegLogin.aspx “The media landscape is evolving at an unprecedented rate, with new platforms, technologies and even new realities at our disposal that offer greater opportunities for content innovation and audience engagement,” said Michael Crimp, CEO, IBC. “IBC is preparing an outstanding programme of conference sessions, forums and exhibition features
Uutishuoneessa voit lukea tiedotteitamme ja muuta julkaisemaamme materiaalia. Löydät sieltä niin yhteyshenkilöidemme tiedot kuin vapaasti julkaistavissa olevia kuvia ja videoita. Uutishuoneessa voit nähdä myös sosiaalisen median sisältöjä. Kaikki STT Infossa julkaistu materiaali on vapaasti median käytettävissä.Tutustu uutishuoneeseemme