Table 1: Approval status of products for the treatment of HAE-C1-INH (Europe and USA) < 18 years old.

Active ingredient/trade name

Approval in

Approval for pediatric patients

Indication in HAE-C1-INH

Route of administration

Human pdCq-INH concentrates

 

 

 

 

Berinert

 

Europe USA

 

Children, adolescent

 

Acute attack, home therapy

 

Intravenous

 

Cinryze

Europe

USA

Adolescent

 

Acuteattack, short-term and long-termprophylaxis, home therapy

Long-termprophylaxis

Intravenous

 

Recombinat human C1-INH concentrate

 

 

 

 

Ruconest

Europe

No

Acute attack

Intravenous

Kallikrein inhibitor and bradykynin receptor antagonist

 

 

 

 

Icatibant/Firazyr

Europe, USA

No

Acute attack, home therapy

Subcutaneous

Ecallantide/Kalbitor

USA

Adolescent > 16 yearsold

Acute attack

Subcutaneous

Attenuated androgens

 

Ԑ-Aminocaproicacid (Amicar) isnotapprovedfor HAE therapy

 

 

Danazol/Danatrol

Switzerland

Adolescents

Long-termprophylaxis

Oral

Danazol/Danokrin

Austria

No

Long-termpropylaxis

Oral

Danazol/Danocrine

USA

No specific approval for children and adolescent

Long-termpropylaxis

Oral

Stanozolol/Winstrol

USA

Children and adolescent

Long-term propylaxis

Oral

Antifibrinolytics

 

 

 

 

Tranexamicacid/Cyklokapron

Austria

No specific approval for children and adolescent

HAE

Oral

 

Switzerland

Children and adolescent

Long-term prophylaxis, acute attacks

(for prodromal symptoms)

Oral

 

Germany

Children and adolescent

Long-term prophylaxis, short-term propylaxis posible

Oral