Forschung & Entwicklung
Octapharma announces positive new clinical data in 66 previously treated patients (PTPs) with severe haemophilia A (HA) undergoing PK-guided personalized prophylaxis with Nuwiq® according to the NuPreviq Approach. Data from the NuPreviq study (GENA-21, NCT01863758) were published online in April 2017 as an Early View article in the internationally renowned medical journal, Haemophilia.
The NuPreviq study was initiated in August 2013 with the aim of developing a fully personalized service for HA patients undergoing prophylactic therapy. Octapharma’s aim is to improve patient treatment experiences by recognizing that no two HA patients are the same, therefore a “one size fits all” approach to prophylaxis is not ideal. The NuPreviq study investigated a novel approach, allowing the Nuwiq® treatment regimen to be adapted to the specific needs of each individual patient. The NuPreviq Approach measures and profiles each individual patient’s pharmacokinetic response to treatment, which then allows the dosing of Nuwiq® to be personalized according to the specific circumstances of that patient.
The results of the study showed that this personalized prophylaxis approach was very effective at preventing bleeds, while reducing the frequency of dosing and the amount of Nuwiq® needed. Over 80% of patients did not experience a spontaneous bleed during the 6 months of personalized prophylaxis with Nuwiq®. More than half of the patients received infusions twice a week or less, and dose requirements were reduced by 7%. An ongoing extension study (GENA-21b, NCT02256917) will further evaluate the long-term efficacy and safety of personalized prophylaxis with Nuwiq®.
Larisa Belyanskaya, Head of IBU Haematology at Octapharma, said: “We are very excited by the results of the NuPreviq study and pleased that the GENA-21 study data are now available to the wider public in the specialist peer-reviewed publication, Haemophilia. The study shows that personalized prophylaxis with Nuwiq® provides excellent bleeding protection, extension of the dosing interval for most patients, and a reduction in dose. These data further differentiate Nuwiq® from other products on the market.”
Olaf Walter, Board Member at Octapharma, commented that “Octapharma’s principal motivation lies with providing dedicated service to our patients. Developing a custom care package which offers flexible FVIII therapy, truly personalized to suit their needs and lifestyle, has long been a goal of the company and we celebrate today this landmark publication of the extremely positive NuPreviq clinical data.”
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.
The GENA-21 and GENA-21b studies, initiated in August 2013 and March 2015, respectively, are prospective, open-label, multicentre, Phase 3b studies assessing the safety and efficacy of PK-guided personalized prophylaxis with Nuwiq® in previously treated adult patients with severe HA. The studies are each conducted across 29 study locations worldwide.
Nuwiq® is a naturally long-acting, 4th generation rFVIII protein, produced in a human cell line without chemical modification or fusion with any other protein. Nuwiq® is cultured without additives of human or animal origin, is devoid of antigenic non-human protein epitopes and has a high affinity for the von Willebrand coagulation factor. Nuwiq® treatment has been assessed in seven completed clinical trials which included 201 PTPs (190 individuals) with severe HA, including 59 children. Nuwiq® is approved for use in the treatment and prophylaxis of bleeding across all age groups of PTPs with haemophilia A in the EU, US, Canada, Australia, Latin America and Russia. Further worldwide submissions for Nuwiq® are planned.
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 neutralizing 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
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Forschung & Entwicklung