Octapharma gets EMA positive opinion for Nuwiq to treat haemophilia A

Nuwiq, the first new generation FVIII replacement protein derived from a human cell line, is produced without animal derived products, and devoid of non human epitopes thought to underlie autoimmune reaction.

Octapharma senior vice president of International Business Units Olaf Walter said Nuwiq shows 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," Walter said.

Benefit with Nuwiq in terms of prevention and treatment of bleedings and haemostatic efficacy for surgical procedures was assessed in three major clinical trials.

In GENA-01 trial, the drug 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 1 infusion.

The trial GENA-08 evaluated 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 GENA-03 trial conducted in 59 paediatric subjects between 2 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 2 to 5 than in those aged 6 to 12 years.