Pharming Group has reported positive results from a Phase II clinical study of RUCONEST (recombinant C1 esterase inhibitor, 50 international units (IU) / kg) for prophylaxis in patients suffering with hereditary angioedema (HAE).
The study found that RUCONEST significantly reduced attack frequency for both the twice-weekly and once-weekly treatment regimens when compared with placebo.
Go deeper with GlobalData
Discover B2B Marketing That Performs
Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.
The randomised, double-blind, placebo-controlled study enrolled 32 HAE patients deficient in C1 esterase inhibitor and a history of at least four attacks per month.
These patients received RUCONEST once and twice weekly and placebo in each of three four-week treatment periods in a cross-over design.
During the intent-to-treat analysis (ITT), the trial found a significant difference in the mean number of HAE attacks that patients experienced during treatment with both the twice-weekly (p-value <0.0001) and once-weekly (p-value =0.0004) RUCONEST regimen as compared with placebo.
Patients on placebo had a mean of 7.2 attacks per four week treatment period, which was reduced to a mean of 2.7 attacks on RUCONEST twice weekly and a mean of 4.4 attacks on RUCONEST once-weekly.
US Tariffs are shifting - will you react or anticipate?
Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis.
By GlobalData“Combined with the excellent safety profile, this data supports further development of recombinant C1INH as a useful preventive therapy for HAE.”
A rarely occurring genetic disorder, HAE shows symptoms such as spontaneous and recurrent swellings of the skin in different part of the anatomy.
Ruconest is currently also being tested in a Phase II clinical trial to treat HAE in young children aged between two and 13 years.
Image: Swollen hand of patient suffering from HAE. Photo: courtesy of LucyHAE via Wikipedia.
