The symposium, chaired by Craig Kessler, addressed the reality that no two haemophilia A patients are the same. An optimal treatment approach should therefore reflect each individual patient’s profile. Striving towards this goal, two approaches to using pharmacokinetic (PK) data to personalise prophylaxis with Nuwiq® were presented in the symposium. John Pasi (The Royal London Hospital, London, UK) shared a summary of the NuPreviq study, which uses an individual’s PK profile to optimise the treatment plan1. This individualised dosing approach enabled over half (57%) of patients in the NuPreviq study to reduce dosing with Nuwiq® to twice weekly or less, whilst maintaining effective bleed protection (median annualised bleeding rate [interquartile range]: 0 [0, 1.9]) for all bleeds). During personalised prophylaxis with Nuwiq®, 83% of patients were spontaneous bleed free. A second approach, using WAPPS (Web Accessible Population Pharmacokinetics Program), was presented by Stacy Croteau (Boston Children’s Hospital, Boston, USA). This approach uses the PK analysis of a group of people to predict the optimal treatment for an individual, and provides a flexible approach to dosing that requires fewer samples. A Nuwiq®-specific WAPPS model is available and is based on PK data from 114 patients, including 26 children, treated with Nuwiq®. These approaches represent promising steps towards achieving personalised haemophilia care.
For previously untreated patients (PUPs), the risk of inhibitor development remains the greatest concern with haemophilia treatment. Ellis Neufeld (St. Jude Children’s Research Hospital, Memphis, USA) presented an overview of interim data from the NuProtect study, which is investigating Nuwiq® in PUPs2. The interim data indicate a cumulative incidence of high-titre inhibitors of only 12.8% in patients treated with Nuwiq®.The presentation also included data from a recent sub-analysis of inhibitor development based on patients’ F8gene mutation status3. This analysis demonstrated a cumulative inhibitor incidence of 26.7% in patients with high-risk mutations, whilst no patients with low-risk mutations developed inhibitors. Dan Hart (The Royal London Hospital, London, UK) presented a novel genomic approach to further analyse data from the NuProtect study, with the aim of predicting inhibitor risk using transcriptional profiles.
Ri Liesner (Great Ormond Hospital for Children, London, UK) presented real-world data on the use of Nuwiq®for immune tolerance induction (ITI), which remains the only proven strategy to eradicate inhibitors. Three of the five patients in the cohort have achieved eradication of inhibitors, while the other two remain on ITI and show continued decline in inhibitor levels.
Larisa Belyanskaya, Head of Octapharma’s IBU Haematology stated “we at Octapharma are delighted with the excellent data presented at WFH 2018, which build on the growing wealth of positive experience with Nuwiq®. The broad range of data demonstrate the expanding value of Nuwiq® in clinical practice and highlight Octapharma’s commitment to ‘going further’ to address the developing demands of haemophilia A patients”. Olaf Walter, Board Member at Octapharma, added “The data presented at the symposium build on a broad portfolio of clinical experience with Nuwiq®. The WFH congress is a key platform for sharing information in the haemophilia field and Octapharma is proud to present new data on Nuwiq to the international haemophilia community at this meeting”.
About Nuwiq®
Nuwiq® is a 4th generation rFVIII protein4, produced in a human cell line without chemical modification or fusion with any other protein5. Nuwiq® is cultured without additives of human or animal origin5, is devoid of antigenic non-human protein epitopes6 and a high affinity for the von Willebrand coagulation factor7. Nuwiq®treatment has been assessed in seven completed clinical trials which included 201 PTPs8,9 (190 individuals) with severe haemophilia A, including 59 children10. 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.
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
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 2017, the Group achieved €1.72 billion in revenue, an operating income of €349 million and invested €287 million to ensure future prosperity. Octapharma employs around 7,700 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 has seven R&D sites and six state-of-the-art manufacturing facilities in Austria, France, Germany, Mexico and Sweden.
For more information visit www.octapharma.com
References:
1. Lissitchkov T, et al. Haemophilia 2017;23:697-704.
2. Liesner R, et al. Haemophilia 2018;24:211-220.
3. Liesner R, et al. Blood eLetter 2018; available at http://www.bloodjournal.org/content/early/2017/08/02/blood-2017-06-791756
4. Lieuw K. J Blood Med 2017; 8: 67–73.
5. Casademunt E, et al. Eur J Haematol 2012; 89: 165-76.
6. Kannicht C, et al. Thromb Res. 2013; 131: 78-88.
7. Sandberg H, et al. Thromb Res 2012; 130: 808-17.
8. Valentino LA, et al. Haemophilia 2014; 20(Suppl. 1): 1-9.
9. Lissitchkov T, et al. Haemophilia 2017; 23: 697–704.
10. Klukowska A, et al. Haemophilia 2016; 22, 232-39.