The European Medicines Agency (EMA) has given a positive opinion towards Octapharma’s human cell line recombinant human FVIII, Nuwiq, recommending the granting of marketing authorisation for treatment and prophylaxis of bleeding in paediatric and adult patients with haemophilia A (congenital FVIII deficiency).
Nuwiq is the first new-generation FVIII replacement protein made from a human cell line, and is produced without animal-derived products, and devoid of non-human epitopes thought to underlie autoimmune reaction.
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Octapharma senior vice-president of international business units Olaf Walter said: "Nuwiq demonstrates outstanding efficacy in preventing and treating bleeding in haemophilia A patients previously treated with FVIII (PTPs)
"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."
The benefit with Nuwiq in terms of prevention and treatment of bleedings and haemostatic efficacy for surgical procedures was evaluated in three major trials.
In the GENA-01 study, Nuwiq was evaluated 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 one infusion.
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By GlobalDataAlso investigated was prophylaxis of bleeding events and the treatment of breakthrough 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.
A total of 59 paediatric subjects enrolled in the study were aged between two and 11 years of age, the mean rate of all BEs in prophylaxis was 0.338 BEs/month, while the monthly rate of all BEs was lower in patients aged two to five than in those aged six to 12 years.
In this trial, 68.6% of breakthrough bleeds were treated with one infusion and 81.3% with one or two infusions.
The company said that no side-effects were commonly reported in the safety database of 135 previously treated patients.
The immunogenicity of the drug was evaluated in clinical trials in 135 previously treated patients with severe haemophilia A and none of the patients developed inhibitors.
Image: Deficiency in coagulation factor VIII is the cause of haemophilia A. Photo: courtesy of ProteinBoxBot.
