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

Data Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

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.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.
“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.