Octapharma AG has received orphan drug exclusivity from the US Food and Drug Administration (FDA) for wilate, a new plasma formulation specifically for the treatment of spontaneous or trauma-induced bleeding episodes in patients with von Willebrand disease (VWD).
VWD is the most common bleeding disorder and is found in approximately 1–2% of the US population. Orphan drug status will provide clinical incentives to Octapaharm to develop the treatment, which caters for this relatively small patient market.
Octapharma group vice chairman Kim Bjornstrup said that FDA orphan drug exclusivity approval is an important aspect in Octapharma’s development of this drug.
“Wilate has a combination of two viral attenuation steps, high purity and a physiological 1:1 ratio of VWF and FVIII activity and is approved for treating all VWD patients including those with the most severe, type 3 disease,” Bjornstrup said.
Wilate is a high-purity, double virus inactivated von Willebrand Factor/Coagulation Factor VIII Concentrate (Human) and has demonstrated efficacy for all types of VWD including in paediatric patients during four sets of clinical trials.
Octapharma received FDA approval for the treatment of spontaneous and trauma-induced bleeding episodes in December 2009.