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
Om Business Wire
(c) 2018 Business Wire, Inc., All rights reserved.
Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.
Følg saker fra Business Wire
Registrer deg med din epostadresse under for å få de nyeste sakene fra Business Wire på epost fortløpende. Du kan melde deg av når som helst.
Siste saker fra Business Wire
SM-Optics Unveils First FLEXSET Chip, Paving the Way to Optical Micro Nodes23.2.2018 14:00 | Pressemelding
SM-Optics launches FLEXSET, a single chip embedding multiple processing functionalities raising the bar for performances, density and architectural flexibility. The FLEXSET is the heart of the Lightmode product family. FLEXSET OTN switching matrix scales from 300Gbps to 1Tbps and can be addressed by ports spanning from traditional low-speed services like 2Mbps over micro-OTN, to layer2 and layer3 over ODUFlex, 100G over ODU4 and 200G over FlexO. Thanks to its embedded capabilities including multi-technology OAM, packet processing, synchronization and fast protection functions, FLEXSET operates by design at the lowest latency performances and features an Intel Stratix 10 FPGA. Following the launch of micro-OTN, a revolutionary approach to efficiently extend OTN protocol to low speed services, the launch of FLEXSET advances the vision of a highly interconnected metro network and of micro nodes. Revolving around functional block elements, micro nodes collapse in few rack units OTN and pho
Bitcoin Miner HashGains’ Crowdsale to Build Green Cloud Mining Data Centers Receives Excellent Response23.2.2018 13:56 | Pressemelding
HashGains.com, a leading cryptocurrency mining platform, has launched its crowdsale program to build mega cryptocurrency cloud mining data centers in India and Canada which uses renewable energy like wind and solar as source of energy. “With the growing worries around bitcoin mining causing environmental damage, difficulty levels of mining increasing and returns heading southwards, there was no better opportunity but to build green energy data centers which run on free sources of energy like wind and solar, ensuring handsome mining rewards while taking care of environment,” said Mr Anuj Bairathi, CEO & Founder, HashGains, who is crypto enthusiast himself. HashGains is a growing cloud mining platform with more than 10,000 active customers enjoying mining returns and is expected to reach the mark of 1 million customers by 2020. To handle such a massive growth and ever growing demand of customers, it becomes even more important that mega mining centers are planned which can serve needs of
Stratecast Recognizes CARDINALITY as a “10 to Watch in 2018”23.2.2018 13:07 | Pressemelding
In their new report, “Digital Transformation Means New Tools for New Business” —part of the Global Operations, Orchestration, Data Analytics, and Monetisation (ODAM) stream—Stratecast recognises CARDINALITY for their new approach towards processing big data to deliver applications such as Customer Experience, Operational Intelligence, Network Analytics, Churn Management and Marketing Intelligence. Stratecast identified that the CARDINALITY Perception Platform brings large amounts of data from multiple CSP sources together in order to address a growing group of data analysis needs from different operational work teams and the wider business. It also does this at ingestion rates and storage levels previously thought not practical. "Using design principals tied to microservices and containerized solution components, the platform is a major advancement in meeting CSP data analysis needs at a price untouchable by previously-defined platforms," said Karl Whitelock, Stratecast director of glo
IBC2018 Welcomes Channel 4’s Keith Underwood as Guest Chair of Content Steering Group23.2.2018 11:03 | Pressemelding
IBC is thrilled to announce that Keith Underwood, Director of Strategy and Technology at Channel 4, has accepted the role of Guest Chair of the Conference for IBC2018. The position involves acting as the figurehead and brand ambassador for IBC, setting the vision for the programme and increasing awareness and engagement with IBC across both traditional and converging media markets. Keith is the Channel 4 Executive Committee member responsible for strategy and corporate development, broadcast operations, content management, corporate systems and the development of Channel 4’s digital products. His achievements at the public broadcaster include the launch of All 4, Channel 4’s award-winning video on demand service that replaced 4oD in 2015. “The media industry is experiencing profound shifts in consumer behaviour, competitive dynamics, and technological innovation.” Keith Underwood said. “Relentless disruption of established business models presents new opportunities and challenges for p
SES S.A.: Full Year and Fourth Quarter 2017 Results23.2.2018 07:00 | Pressemelding
SES S.A. announced financial results for the year and three months ended 31 December 2017. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20180222006522/en/ Full Year and Fourth Quarter 2017 Results (Photo: Business Wire) Key financial highlights Reported revenue EUR 2,035.0 million, down 1.6% (-5.2% like-for-like(1)); SES Video -3.6%(1) and SES Networks -1.9%(1) EBITDA margin 65.1% (2016: 70.2% as reported and 66.7% like-for-like(1)) Net profit of EUR 596.1 million (2016: EUR 962.7 million including EUR 495.2 million gain related to O3b consolidation) Board is proposing 2017 dividend per A share of EUR 0.80 (2016: EUR 1.34) Change (%) Change (%) EUR million FY 2017 FY 2016 Reported Like-for-like (1) Q4 2017 Q4 2016 Reported Like-for-like (1) Revenue 2,035.0 2,068.8 -1.6% -5.2% 507.8 578.7 -12.2% -8.7% EBITDA 1,324.2 1,451.5 -8.8% -7.6% 329.6 390.6 -15.6% -12.2% EBITDA margin 65.1% 66.7%(1) 64.9% 67.6%(1) Operating profit (2) 6
Lion/Gem Luxembourg 3 S.a.r.l. Announce First Quarter Results for FY 201823.2.2018 07:00 | Pressemelding
The First Quarter results for FY18 for Lion/Gem Luxembourg 3 S.a.r.l. (associated with Young’s Seafood Limited) will be made available on our Investor Relations website on February 23, 2018. The First Quarter results call for investors that accompanies this information is scheduled to take place at 13:00 GMT on February 23, 2018. The First Quarter covers the quarter to 30 December 2017; the financial year end for Lion/Gem Luxembourg 3 S.a.r.l. is September 30, 2018. For further information: If you are an investor or a potential investor in the 8¼%/ 9% Senior PIK Notes due 2019, of Lion/Gem Luxembourg 3 S.a.r.l., and would like access to this information, please register your interest on our Investor Relations website: https://youngsseafood.co.uk/investors/. If you have any questions about the registration process or need further information, please do not hesitate to contact Nicholas Donnelly, Communications Manager at Young’s Seafood: firstname.lastname@example.org This announc