In the study, 25 asymptomatic HAE patients with a history of frequent attacks received once weekly administrations of 50 U/kg of Ruconest for 8 weeks.
The study results suggested that the patients reported a median of 60 HAE attacks (range 39 to 467) over the past two years, corresponding to an average of 0.6 attacks per week (range 0.4 to 4.5).
The breakthrough attack rate observed during the study was much lower with a median of 2 attacks over the 8 week period, corresponding to an average of 0.25 attacks per week (range 0 to 1.5).
However, the weekly administrations of 50 U/kg rhC1INH was generally safe and well tolerated.
Pharming chief medical officer Rienk Pijpstra said that the results of this exploratory study suggest that Ruconest, in addition to the approved indication in Europe for the acute treatment of angioedema attacks, could also offer significant value for long term prophylaxis in patients with frequent attacks.
"We plan further pharmacological investigations to optimize Ruconest dose and dosing interval for this indication," Pijpstra said.