Safety of Ruconest in 2-13 Year Old Hereditary Angioedema (HAE) Patients

This study was an open-label, Phase 2, non-comparative, multinational, multicenter clinical
study in pediatric patients from 2 up to and including 13 years of age, with a confirmed
diagnosis of HAE. Patients were eligible for treatment with rhC1INH if they presented to the
clinic within 5 hours of onset with an acute attack of at least moderate severity without
signs of spontaneous regression. Patients received rhC1INH at a dose of 50 U/kg body weight
up to a maximum of 4200 U. The reconstituted solution was administered as a slow intravenous
(iv) injection over approximately 5 minutes. The patients remained in hospital and were
closely monitored in the study center for at least 4 hours after study medication
administration.

Inclusion Criteria:

- From 2 up to and including 13 years of age

- Clinical and laboratory confirmed diagnosis of HAE (baseline C1INH activity <50% of
normal)

- Signed written informed consent (parental permission) signed by the legal guardian(s)

- Clinical symptoms of an acute HAE attack

- Onset of eligible symptoms within 5 hours from the moment at which medical evaluation
to determine eligibility has occurred

- Attack severity moderate or greater, as rated by the investigator

Exclusion Criteria:

- A diagnosis of acquired C1INH deficiency (AAE)

- A medical history of allergy to rabbits or rabbit-derived products or positive
anti-rabbit epithelium (dander) IgE test