Research & Development
Octapharma announced today that new data on the benefits of Nuwiq® in patients with haemophilia A were presented during a scientific symposium at the 27th International Society on Thrombosis and Haemostasis (ISTH) Congress in Melbourne, Australia. The symposium, entitled ‘Conveniently Protecting What Matters: Nuwiq® (Simoctocog Alfa) in PUPs and Personalised Prophylaxis’, focussed on addressing some of the most important clinical needs of people with haemophilia A, and included final data from the NuProtect Study in previously untreated patients (PUPs), which were presented for the first time. The symposium was chaired by Ellis J. Neufeld (St. Jude Children’s Research Hospital, Memphis, USA).
Larisa Belyanskaya, Head of the Octapharma’s Haematology International Business Unit stated “We are very pleased to present the final data from the NuProtect study in PUPs, confirming the favourable rate of inhibitor development with Nuwiq® reported in the earlier interim analysis.”.
Olaf Walter, Board Member at Octapharma, said “The data presented at the symposium build on a broad clinical experience with Nuwiq®. The ISTH congress is a key platform for exchanging information in the international haemophilia field and we are proud to present these new and robust data in PUPs, which we believe will help to improve haemophilia A treatment”.
No two people with haemophilia A are the same, and therapy should therefore reflect the characteristics and needs of each individual. Robert Klamroth (Vivantes Klinikum, Berlin, Germany) presented data on the use of pharmacokinetic (PK) information to personalise prophylaxis with Nuwiq®. In the NuPreviq study, an individualised dosing approach enabled over half (57%) of patients to reduce dosing with Nuwiq® to twice-weekly or less, whilst providing effective bleed protection, with 83% of people free from spontaneous bleeds during the 6 months of personalised prophylaxis. There were no FVIII inhibitors or treatment-related serious or severe adverse events.
Inhibitor development remains a serious treatment complication of haemophilia A, particularly in PUPs, with up to 35% of PUPs developing inhibitors to FVIII. Ri Liesner (Great Ormond Street Hospital, London, UK) presented for the first time the final data from the NuProtect study, which investigated the development of inhibitors in 108 PUPs treated with Nuwiq®. The cumulative incidence of high-titre inhibitors was only 17.6%, and no patients with non-null F8 gene mutations developed inhibitors. Nuwiq® was well tolerated.
In people with haemophilia A who develop inhibitors, immune tolerance induction (ITI) is the only approach clinically proven to remove inhibitors and allow people to resume effective FVIII prophylaxis therapy. Georgina Hall (University of Oxford and John Radcliffe Hospital, Oxford, UK) shared real-world experience of using Nuwiq® for ITI, reporting inhibitor elimination in 8 of 10 (80%) patients treated so far. As new treatments become available for people with haemophilia A and inhibitors there is growing interest in the combination of ITI with other products. Carmen Escuriola Ettingshausen (Haemophilia Centre Rhein Main, Mörfelden-Frankfurt, Germany) introduced the investigator-initiated MOTIVATE study, which will investigate the efficacy and safety of “standard of care” ITI vs novel approaches combining FVIII with emicizumab, or emicizumab prophylaxis alone, in people with inhibitors.
Nuwiq® is a 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 previously treated patients (PTPs; 190 individuals) with severe haemophilia A, 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 Nuwiqare 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 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 up to 39%.
The vision of Octapharma is: “Our passion drives us to provide new health solutions advancing human life”. 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. Our company values are Ownership, Integrity, Leadership, Sustainability and Entrepreneurship.
In 2018, the Group achieved €1.8 billion in revenue, an operating income of €346 million and invested €240 million into R&D and in capital expenditures in order to ensure future prosperity. Octapharma employs 8,314 people worldwide to support the treatment of patients in 115 countries with products across three therapeutic areas:
Octapharma has seven R&D sites and six state-of-the-art manufacturing facilities in Austria, France, Germany, Mexico and Sweden.
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Research & Development