Octapharma confirmed today the European Medicines Agency (EMA) has adopted a positive opinion towards human cell line recombinant human FVIII, Nuwiq®, recommending the granting of a marketing authorisation for the medicinal product for treatment and prophylaxis of bleeding (also during and after surgery) in paediatric and adult patients with haemophilia A (congenital FVIII deficiency).
Nuwiq® is the first new generation FVIII replacement protein, derived from a human cell line awaiting marketing authorisation in Europe. Nuwiq® is produced without animal derived products, and devoid of non human epitopes thought to underlie autoimmune reaction. Inhibitor antibodies to replacement FVIII typically neutralise the beneficial effect of the protein, and represent the major challenge in today's management of haemophilia A.
"Nuwiq® demonstrates outstanding efficacy in preventing and treating bleeding in haemophilia A patients previously treated with FVIII (PTPs), and its human characteristics may underlie the absence of neutralising antibodies in this population to date. Nuwiq® may represent a leap forward in combating the single greatest problem facing newly treated haemophiliacs today", says Olaf Walter, MD, PhD, MBA, Senior Vice President of International Business Units at Octapharma.
The benefit with Nuwiq® in terms of prevention and treatment of bleedings and haemostatic efficacy for surgical procedures was studied in 3 pivotal trials; study GENA-01 in 22 adolescent and adult subjects where overall, 94.4% of the bleeding episodes (BEs) were treated on-demand with excellent or good efficacy and a large majority of BEs required only 1 infusion; study GENA-08 investigated prophylaxis of bleeding events and the treatment of break-through bleeds in 32 adults, where the mean bleeding rates per patient during the prophylactic treatment period were 0.188/month for all types of bleeds. In study GENA-03 in 59 paediatric subjects between 2 and 11 years of age, the mean rate of all BEs in prophylaxis was 0.338 BEs/month; the monthly rate of all BEs was lower in patients aged 2 to 5 than in those aged 6 to 12 years (0.213 BEs/month and 0.459 BEs/month, respectively). In this study 68.6% of break-through bleeds were treated with one infusion and 81.3% with one or 2 infusions.
No side effects were commonly reported in the safety database of 135 previously treated patients. The immunogenicity of Nuwiq® was evaluated in clinical trials in 135 previously treated patients with severe haemophilia A (74 adult and 61 paediatric patients). None of the patients developed inhibitors.
Currently, an ongoing study (NuProtect) is investigating the efficacy and safety of Nuwiq® in previously untreated patients (PUPs, target enrolment 100 patients) who are typically at high risk for the development of neutralising inhibitors. Additional studies in PTPs are also taking place, including the NuPreviq study, investigating personalised prophylaxis in 65 adult patients.
Data for Nuwiq® have also been submitted to authorities in Canada and Australia, with further worldwide submissions planned.