In February, HAE France (AMSAO) presented a video conference meeting with the adult and pediatric team from the angioedema reference center CREAK in Grenoble. The event was intended for children or adolescents and their parents as it enabled the sharing of information and experiences between caregivers and patients.
Dr. Boccon-Gibod talked about the 130 children currently followed by CREAK, while the pediatric psychologists Karine Guichardet and Gaëlle Buisson-Papet discussed with participants the feelings experienced by children or their parents during HAE attacks. The program also included a talk by Dr. Kevorkian-Verguet on clinical trials in children, while President Michel Raguet of HAE France briefly recounted the history of the patient association founded 24 years ago. He highlighted the move towards better diagnostics, the arrival of emergency treatments (Berinert and Firazyr) and more recently the arrival of two new prophylactic treatments with monoclonal antibodies which restrict the production of kallikrein (lanadelumab and berotralstat).
The French National Agency for Medicines and Health Products Safety has granted a temporary authorization for use for Orladeyo (berotralstat) to prevent HAE attacks in patients aged 12 and older. This allows people with HAE in France to receive treatment with berotralstat before the drug is granted marketing authorization by the European Commission. The final approval decision from the Commission on the marketing authorization application for Orladeyo – a brand of plasma kallikrein inhibitor delivered through oral administration and is approved for self-administration – is expected in the second quarter of 2021.
