Business Wire

Octapharma: Low inhibitor rate with octanate® in previously untreated patients (PUPs) with severe haemophilia A

Jaa

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.

Reference

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%.

About Octapharma
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

Contact information

Octapharma AG
International Business Unit - Haematology
Olaf Walter
Olaf.Walter@octapharma.com
or
Larisa Belyanskaya
Larisa.Belyanskaya@octapharma.com
Tel: +41 55 4512121

Tietoja julkaisijasta

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

Manchester United Shoots for Success with a “Digital Experience Platform” Powered by HCL16.8.2018 22:02Tiedote

Manchester United (NYSE:MANU) has today announced a major milestone on its digital transformation journey with HCL Technologies, with the successful launch of the Manchester United Official app powered by HCL’s digital experience platform. The launch is part of the ongoing digital transformation of the club to provide a real-time, engaging, personalized, and unified experience to Manchester United’s 659 million global followers, which supports the club’s strategy to become a Digital Sports Enterprise. At the heart of this transformation is the digital platform developed by HCL, providing the club with a single view of fans across web and app touchpoints, offering fans a one-stop shop for everything Manchester United. Manchester United’s Group Managing Director Richard Arnold comments: “This app will allow our 659 million Manchester United followers to easily connect to the club they love, wherever they are in the world. Fans will now have the opportunity to follow all Manchester United

4K 4Charity Fun Run Announces Open Registration for IBC201816.8.2018 20:41Tiedote

AWS Elemental, an Amazon Web Services (AWS) company, today announced that the fifth annual 4K 4Charity Fun Run at IBC Show will take place at 7:00 a.m. local time on Saturday, Sept. 15 at Amstelpark in Amsterdam. Registration is open at https://4k4charity.com/ibc. Among the non-profits supported by the 4K 4Charity Fun Run at IBC Show is Amsterdam-based StichtingNewTechKids, which provides computer science education for girls, minorities, and economically challenged youth in the Netherlands. The organization supports a computer science teacher training program for primary school teachers with proceeds from 4K 4Charity. Also supported is Iridescent, a global education non-profit that empowers underrepresented young people to become innovators and leaders through engineering and technology. Through their two programs, Curiosity Machine AI Family Challenge and Technovation, Iridescent introduces underserved communities to new technologies and empowers and equips them to apply those technol

2018 D.I.C.E. Cannes Announced Round 3 Speakers16.8.2018 17:01Tiedote

The Academy of Interactive Arts & Sciences’ D.I.C.E. Cannes event will take place 9-11 September 2018 at the Hotel Barrière Le Majestic Cannes in France. Leading video game executives will participate in the premier networking opportunities, listen to key speakers address the conference theme - Trailblazers - to tackle some of the industry’s biggest ideas and trends. Newly announced speakers include: Craig Duncan, Studio Head at Rare – Duncan will share his studio’s experience and learnings with “A Rare Voyage of Adventure.” Duncan will provide insight into Rare’s bold new IP Sea of Thieves, a game that has inspired friendships and players creating stories together. David Hubert, Animation and Cinematic Director at Eidos Montreal – In Hubert’s talk he will discuss how cinematics are redefining AAA story-driven games and how they will continue to shape the medium to push the envelope throughout the production process. Adam Orth – In a session titled “Digging for Fire: Virtual Reality 20

GN Hearing and Google Announce Partnership to Bring Direct Mobile Streaming from Android Devices to Hearing Aids16.8.2018 16:00Tiedote

GN Hearing and Google have today announced a new technology partnership that will make GN Hearing the first manufacturer to enable a full spectrum of direct audio streaming from Android devices to hearing aids. The expectations are that direct streaming will become available to hearing aid users of the recently launched hearing aids ReSound LiNX Quattro™ and Beltone Amaze™ in a future Android release. “According to the World Health Organization, around 466 million people worldwide have disabling hearing loss. This number is expected to increase to 900 million people by the year 2050. Google is working with GN Hearing to create a new open specification for hearing aid streaming support on future versions of Android devices,” states Seang Chau, Vice President of Engineering at Google. Users will be able to connect and monitor their hearing aids, so they can get the full advantages of their Android devices without using an intermediate device for streaming to their hearing aids. This will

Zurich Expands Global Use of Guidewire InsurancePlatform with Selection of Guidewire Cyence Risk Analytics16.8.2018 15:55Tiedote

Guidewire Software, Inc. (NYSE: GWRE), the provider of the industry platform Property and Casualty (P&C) insurers rely upon, today announced that Zurich Insurance Group (Zurich) has selected Guidewire Cyence™ Risk Analytics (Cyence) to leverage the cyber risk knowledge and insights the solution provides to help the company prospect, underwrite, and price risks. Zurich will roll out Cyence globally with an emphasis on enhanced analytics and intelligence for risk selection. Unprecedented technological advances of the 21st century have shifted the nature of risk across the P&C insurance industry. Ever-evolving cyber risks present unique challenges including limited availability of traditional actuarial data needed to model these risks. Cyence combines economic modeling, cybersecurity, machine learning and Internet scale data collection to help risk takers assess the financial impact of cyber risk. “After conducting a thorough search, we found that the capabilities of Guidewire Cyence Risk

Scientist.com Named One of America’s Ten Fastest-Growing Private Companies by Inc. Magazine16.8.2018 15:06Tiedote

Scientist.com, the world’s leading marketplace for outsourced scientific services, today announced that it ranked no. 9 on Inc. magazine’s 2018 list of the 5,000 fastest-growing, privately owned companies in America. Scientist.com ranked no. 2 nationally in the Health category and no. 2 in the state of California. The San Diego-based company builds private marketplaces that connect life science research organizations with a global network of over 2,600 contract research organizations (CROs) and providers of scientific services. “Our mission is to empower and connect scientists worldwide and make it possible to cure all human diseases by 2050,” stated Kevin Lustig, Scientist.com’s CEO and Founder. “On Scientist.com, a single, highly trained scientist can run an entire drug discovery program from concept to clinic from his or her laptop without ever physically entering a laboratory.” The pharmaceutical industry spends about $150B a year on medical research and produces about 25 new medic

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