Research & Development
Octapharma is playing a prominent role this week at the International Society on Thrombosis and Haemostasis (ISTH) Congress in Berlin, Germany. As a platinum sponsor of the congress, Octapharma’s active involvement includes two sponsored scientific symposia, one of which is dedicated to exploring recent data and real-world experiences with Nuwiq®, the most recent addition to the company’s expanding haematology portfolio. The well-attended symposium, “Towards Life Without Limitation, Growing Experience with Nuwiq® in Patients with Haemophilia A”, was held on Sunday 9th July and presented key data from clinical trials alongside real-life case studies and experiences with Nuwiq® in the treatment of haemophilia A.
Nuwiq® is a naturally long-acting, 4th generation recombinant factor VIII protein produced in a human cell line. The absence of non-human epitopes makes Nuwiq® an exciting prospect for reducing rates of inhibitor development, a major complication of haemophilia A treatment. The safety and efficacy of Nuwiq® has already been demonstrated in seven clinical trials. This most recent data, presented during the ISTH symposium, focused on using Nuwiq® to treat previously untreated patients (PUPs) as well as on a pharmacokinetic (PK)-based personalised approach to optimising dosage during haemophilia treatment (NuPreviq).
The session, chaired by Pier M. Mannucci, began with a presentation of interim data from NuProtect, a clinical study using Nuwiq® in a cohort of true PUPs, who are at the greatest risk of inhibitor development (manuscript accepted by Haemophilia on July 5th). Ellis J. Neufeld’s presentation, “Focus on PUPs; latest Nuwiq® efficacy, safety and immunogenicity data in the NuProtect study” described promising inhibitor rate during Nuwiq® treatment based on an interim analysis, particularly for high-titre (HT) inhibitors (12.8% HT; 20.8% overall inhibitors). Convincing evidence of the efficacy of Nuwiq® in bleeding prevention (median annual bleeding rate of zero for spontaneous bleeds; 2.4 for all bleeds) and control of breakthrough bleeds (1 or 2 infusions sufficient to control bleeds in 92.4% of cases) was also presented in this population of PUPs with severe haemophilia A.
Natascha Marquardt went on to discuss her experiences using Nuwiq® to treat a broad range of patients in a single centre in Germany in her talk, “Nuwiq® in practice: experience from a German treatment centre, the Universitätsklinikum Bonn”. She described excellent efficacy with Nuwiq® in all patient categories (including PUPs, previously treated patients (PTPs) and female carriers) and for all forms of treatment (prophylaxis, on-demand treatment and surgical prophylaxis).
The second half of the session discussed the impressive results from real-life clinical experience with the NuPreviq approach, which uses a PK-based personalised prophylaxis to reduce infusion frequency whilst maintaining a low bleeding rate for haemophilia A patients on an individual patient by patient basis. By providing both a high level of bleed protection and the flexibility for adjustments to precisely meet each person’s unique needs, the NuPreviq Approach helps to optimise FVIII dose quantity and frequency by combining individual PK profiling with robust scientific analysis. John Pasi presented data from his experience with NuPreviq in “The NuPreviq Approach: Optimising PK-guided Personalised Prophylaxis with Nuwiq®”, which described the efficacy & safety of personalised PK-based prophylaxis with Nuwiq® in adult PTPs with severe haemophilia A. He described how NuPreviq offers an effective approach to individualising therapy, presenting data that demonstrated excellent protection from bleeds (83% of people were spontaneous bleed-free throughout the 6 month study period) and an overall dose reduction of 7.2%, with 57% of people able to decrease infusions to twice-weekly or less. These convincing results were further supported by the next presentation, “Nuwiq® in action: an Italian multicentre experience using the NuPreviq approach in practice”, presented by Massimo Morfini. Dr. Morfini described data from an Italian multicentre experience using NuPreviq, again demonstrating excellent bleeding protection and less frequent and/ or reduced overall dosage compared to previous treatment. These exciting data support the fundamental principles of the NuPreviq Approach: that every haemophilia A patient is different, with individual treatment needs and a unique PK profile, which can only be fully addressed with personalised prophylaxis. Such an approach can reduce both the burden of haemophilia treatment on the patient, and the demands placed on hospital resources.Combined with four poster presentations focusing on Nuwiq® in the scientific sessions, the overwhelmingly positive data presented at ISTH this week promise an exciting and significant future role for Nuwiq® in haemophilia A treatment. Larisa Belyanskaya, Head of IBU Haematology at Octapharma, expressed her enthusiasm regarding the successful symposium,
“We at Octapharma are delighted with the very promising results and experiences with Nuwiq® to date and are excited to share them with the wider haematology community at ISTH. We look forward to the completion of the PUP study and to seeing the full potential of Nuwiq® and the NuPreviq approach in helping our patients go further in life”.
Olaf Walter, Board Member at Octapharma, commented that
“Octapharma is committed to broadening knowledge in haemophilia A and pushing treatment boundaries. International congresses offer a vital platform to facilitate information exchange and Octapharma is very proud to be able to share these positive results at the ISTH 2017 Congress.”
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%.
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.
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:
Octapharma owns six state-of-the-art production facilities in Austria, France, Germany, Mexico and Sweden.
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Research & Development