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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.

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.

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“Combined with the excellent safety profile, this data supports further development of recombinant C1INH as a useful preventive therapy for HAE.”

UCSD’s US HAEA Angioedema Centre medicine and clinical director professor and co-prinicipal investigator for the study Marc Riedl said: “The results of this well-controlled prophylactic study demonstrate a clinically relevant reduction of HAE attack frequency and a high responder rate with the recombinant C1INH treatment.

“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.