Paediatric use

Plasma-free C1-INH acute treatment from day 1

Onset of HAE attacks typically occurs during childhood or adolescence**.

In paediatric HAE patients, one dose*** of RUCONEST^®

Successfully treated 96% of HAE attacks in children aged 5 to 14 years^¹⁰

n=20 (70 of a total of 73 attacks)

Raised C1-INH levels back to normal**** in 90% of children aged 5 to 14 years^⁷

n=20 (18 of a total of 20 patients)

Raises C1-INH levels back to normal^**** in ≈91% of children aged 2 to <5 years^⁷

As shown on a population PK model.

Recombinant design^⁴

Safety
no risk of human
blood-borne viruses^⁴

No reported
C1-INH inhibitors^○^¹⁰

99% purity^{^‡^5,6}

* RUCONEST^® is indicated for treatment of acute angioedema attacks in adults, adolescents, and children (aged 2 years and above) with hereditary angioedema (HAE) due to C1 esterase inhibitor deficiency.
** Mean age of symptom onset in HAE patients: 4‐14 years.¹⁰
*** Dosing of RUCONEST^® is body weight dependent and one dose is, therefore, based on patient weight as follows; <42kg = up to 1 vial, 42-84 kg = 50 U/kg (up to 2 vials), >84kg = 2 vials. In children an additional dose may be administered if the patient has not responded adequately after 60 minutes.
**** Above the lower limit of the normal range as observed in healthy individuals(0.7-1.3 U/ml).⁴
○ No evidence of C1‐INH neutralizing antibodies (inhibitors) has been observed in children aged 5 to 14 years treated with rhC1‐INH.¹⁰
‡ Figure is rounded based upon 98.6% (Feussner⁶) and 98.7% (van Veen⁵); there are virtually no host related impurities.

Promotional webinar

Data for treatment of paediatric patients with hereditary angioedema with recombinant C1-INH therapy.

Speaker: Dr Scott Hackett, Consultant Paediatric Immunologist, University Hospital Birmingham, Birmingham, UK.

Content is based on the article by Reshef, A., et al. “Recombinant human C1 esterase inhibitor treatment for hereditary angioedema attacks in children.” Pediatr Allergy Immunol. 2019;30(5):562-568. https://pubmed.ncbi.nlm.nih.gov/30993784/