LACHEN, Switzerland. - Tuesday, May 27th 2014 [ME NewsWire]
(BUSINESS WIRE) Octapharma sponsored the symposium "Spotlight on the human factor: building a foundation for the future of haemophilia A management" during the World Federation of Hemophilia (WFH) World Congress in Melbourne, Australia on Monday May 12th 2014.
Major challenges in current haemophilia treatment include the development of inhibitors and the need for frequent venous access for FVIII injection. Octapharma’s human cell line recombinant FVIII (Human-cl rhFVIII) is a new- generation rFVIII derived from a human cell line without addition of animal derived proteins. Human-cl rhFVIII is a glycoprotein devoid of antigenic non-human epitopes. The development aim of Human-cl rhFVIII is to address the challenges of FVIII inhibitors and frequent infusions required during prophylaxis.
On May 22nd, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the granting of a marketing authorisation for the medicinal product Nuwiq®, intended for treatment and prophylaxis of bleeding in paediatric and adult patients with haemophilia A (congenital factor VIII deficiency).
Data for the product has also been submitted to authorities in Canada and Australia, with further worldwide submissions planned.
Presented during the symposium were the results of the recently completed multicenter clinical trials, which assessed the safety, pharmacokinetics, efficacy and immunogenicity of Human-cl rhFVIII in children and adults with severe haemophilia A. In addition, insights into the ongoing previously untreated patient (PUP) study, NuProtect, and the new personalised prophylaxis study, NuPreviq, were presented.
The symposium was co-chaired by Johannes Oldenburg, Chairman and Director of the Institute of Experimental Haematology and Transfusion Medicine and of the Haemophilia Center, University Clinic Bonn, and Craig M. Kessler, attending haematologist/oncologist and Professor of Medicine, Pathology, and Oncology at the Vincent Lombardi Comprehensive Cancer Center at Georgetown University Medical Center in Washington, D.C.
Craig Kessler opened the symposium by presenting unmet clinical needs in haemophilia care including the high risk of inhibitor development in PUPs and the development of new inhibitors throughout the lifetime of previously treated patients (PTPs). He mentioned that “one-size-fits-all” strategy for prophylaxis may pose an unnecessary bleeding risk to patients. A tailored dosing approach is desirable to ensure a high quality of life because of differences in FVIII half lives between patients. Dr. Kessler also highlighted that inhibitor rates are currently unknown for new- generation, artificially enlarged, rFVIII products, as are the potential considerations for product-specific assays of some novel rFVIIIs .
Current rFVIII products produced in hamster cell lines are associated with a cumulative incidence of inhibitors of up to 38% in PUPs. The recruitment rate for the ongoing NuProtect study has been high. Kate Khair, Nurse Consultant at Great Ormond Street Children’s Hospital London, presented “The NuProtect study: can inhibitor incidence in PUPs be reduced?” This study in unselected PUPs aims to differentiate Human-cl rhFVIII with a potentially reduced rate of inhibitors in all ethnic groups. So far, 33 patients have been screened and 24 have started treatment. Optional satellite studies aim to identify predictive markers of inhibitor development in order to identify individuals at increased risk of inhibitor formation.
Carmen Escuriola-Ettingshausen from the Hämophilie-Zentrum Rhein Main (HZRM), Mörfelden-Frankfurt, Germany, presented “Inhibitors in haemophilia: perspectives and challenges”. Octanate is a human plasma-derived FVIII naturally stabilised with von Willebrand factor (VWF) and is a fully sulfated protein. The incidence of clinically relevant inhibitors in an ongoing study in PUPs is 5.9% (3/51). Successful immune tolerance induction was demonstrated with >70% of octanate® patients (many poor prognosis) from the ongoing Observational Immune Tolerance Induction (ObsITI) study.
Dr. Escuriola- Ettinhausen further presented biochemical characteristics of Human-cl rh FVIII. Human-cl rh FVIII is also a fully sulfated human protein. It has significantly higher VWF binding affinity compared with existing full-length rFVIII produced in hamster cell lines. No product specific assay is required for Human-cl rhFVIII as has been shown by consistent results across assays.
Human-cl rhFVIII has completed its clinical development program in accordance with regulatory requirements in paediatric, adolescent and adult PTPs. Andreas Tiede, Head of the Haemophilia Care Centre at Hannover Medical School, Hannover, Germany, presented “The new Human-cl rhFVIII: overview of clinical trial experience in children and adults with haemophilia A”. Dr. Tiede presented that 96.7% of bleeds were controlled with one or two infusions (adults on-demand); mean ABR (annual bleeding rate; spontaneous bleeds) was 1.5 in paediatric and 1.2 in adult patients on prophylaxis and no inhibitors were reported in 135 PTPs treated so far (200 PTPs including NuPreviq). No drug-related serious or severe adverse events have been reported.
Individualisation of therapy is likely to play a major role in the near future of treatment. Claude Négrier, Head of the Haematology Department at Edouard Herriot University Hospital in Lyon, France and also Director of the Haemophilia Comprehensive Care Centre at the same institution, presented “Potential role of TGA in personalized prophylaxis”. Dr. Négrier highlighted that thrombin generation assay (TGA) reflects the bleeding phenotype and might represent a common marker for improvement/achievement of haemostasis. TGA may offer new possibilities to tailor the FVIII dose according to the patient’s haemostatic response.
Human-cl rhFVIII aims to provide patients with fewer infusions during personalized prophylactic treatment. Robert Klamroth, a specialist in internal medicine and coagulation disorders and Director of the Haemophilia Treatment Centre and the Department of Coagulation Disorders at the Vivantes Hospital in Berlin, presented “The NuPreviq study: Human-cl rhFVIII in a multicentre clinical study for personalized prophylaxis”. The ongoing NuPreviq study has enrolled 68 adult patients. Preliminary data in 24 patients indicate a median dosing interval of 3.5 days with 66.7% of patients on a twice-per-week or less infusion schedule with Human-cl rhFVIII. The potential prolonged dosing interval is without increased FVIII consumption when compared to standard prophylaxis. So far, no bleeding episodes have been reported in these 24 patients.
About Haemophilia A
Haemophilia A is an X-linked hereditary disorder caused by factor VIII (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 38%.
About Octapharma AG
Headquartered in Lachen, Switzerland, Octapharma AG is one of the largest human protein products manufacturers in the world and has been committed to patient care and medical innovation for over 30 years. Its core business is the development, production and sale of human proteins from human plasma and human cell-lines. Patients in over 100 countries are treated with products in the following therapeutic areas:
Haematology (coagulation disorders)
Immunotherapy (immune disorders)
Octapharma owns five state-of-the-art production facilities in Austria, France, Germany, Sweden and Mexico.
For more information visit www.octapharma.com