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Publication of Pivotal Data Demonstrating Successful Standard Prophylaxis in Adults with Nuwiq®

18.09.2015

Lachen, Switzerland, September 18th, 2015: Today Octapharma announced the recent publication in Haemophilia of data from the pivotal GENA-08 study, demonstrating outstanding success of their newest-generation human-cell-line recombinant human Factor VIII product, Nuwiq®, in prevention and treatment of bleeding episodes in adults with severe Haemophilia A.

 

These positive data report the results of the prospective GENA-08 study, assessing the efficacy and safety of Nuwiq® in patients with ≥150 previous exposure days to different FVIII concentrates. Prophylactic treatment of Haemophilia A is recommended by the World Federation of Hemophilia and numerous other expert groups, and publication of this highly encouraging data represents further important validation for Nuwiq®.

 

Thirty-two adults with severe haemophilia A received Nuwiq® for standard prophylaxis for at least six months. Prophylactic efficacy of Nuwiq® was rated as “excellent” or “good” in 97% and 100% of patients respectively for the prevention of all bleeds, and spontaneous bleeds. There were no major, life-threatening bleeds and 50% of patients experienced zero bleeds. Consistent with these findings, Nuwiq® demonstrated “excellent” haemostatic efficacy in 4 surgical procedures (3 major, 1 minor) and “moderate” efficacy in 1 major surgical procedure. Bleeding rates during standard prophylaxis with Nuwiq® were low overall and most importantly decreased compared with historical controls, demonstrating a dramatic 99% reduction for patients previously treated on-demand only, indicating that prophylaxis with Nuwiq® is effective, even in adults who have formerly experienced recurrent bleeds.

 

No inhibitors were seen in any patient and there were no treatment-related serious or severe adverse events, further supporting the benefits of Nuwiq® in standard prophylaxis. This excellent safety profile with Nuwiq® may result from the complete absence of non-human, potentially immunogenic epitopes, alongside a high VWF-binding capacity.

Larisa Belyanskaya, Head of Octapharma’s International Business Unit for Haematology, said: “Clinical studies with Nuwiq® continue to provide highly positive efficacy and safety data and further attest to the strong clinical development rationale behind Octapharma’s goal to produce a novel, truly human recombinant FVIII product”.

 

Forthcoming studies with Nuwiq® include trials in previously untreated patients (PUPs), a sub-population known to be at higher risk of developing inhibitors, with the aim of enrolling 100 patients in the NuProtect study. Octapharma is also committed to the ongoing NuPreviq study in PTPs, testing a robust programme of individually personalised prophylaxis, designed to increase flexibility and convenience by permitting reductions in the number of FVIII infusions required for prophylactic coverage.

 

About GENA-08 study: The GENA-08 study, initiated in 2010, was a prospective, open-label, multinational phase III study in patients enrolled at 11 sites in 4 countries (Austria, Bulgaria, Germany and the United Kingdom). The GENA clinical programme aims to assess the benefits of Nuwiq® across a number of patient populations, including both previously treated patients (PTPs) and previously untreated patients (PUPs), with the aim of reducing inhibitor incidence and minimising the frequent infusions needed for bleeding prophylaxis, allowing patients proven bleeding protection, greater convenience and tailored dosing.

 

About Nuwiq®: Nuwiq® is a new generation B-domain-deleted rFVIII protein, without chemical modification or fusion with any other protein, produced in a human cell line. 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® is approved for use in the treatment and prophylaxis of bleeding across all age groups of PTPs with haemophilia A in the EU, U.S., Canada and Australia. Further worldwide submissions 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 AG: Headquartered in Lachen, Switzerland, Octapharma AG is one of the largest human protein product manufacturers in the world and has been committed to patient care and medical innovation since 1983. 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)
  • Critical Care

 

Octapharma owns five state-of-the-art production facilities in Austria, France, Germany, Sweden and Mexico.