Janssen&Janssen schreef op 17 oktober 2022 20:06:
A new study revealed high levels of satisfaction among patients with hereditary angioedema (HAE) who switched from injectable prophylactic treatments to berotralstat (Orladeyo®) monotherapy. The results of the study are being presented as a poster at the National Organization for Rare Disorders’ Rare Disease and Orphan Products Breakthrough Summit.
www.rarediseaseadvisor.com/conference...This study included 120 patients (114 adults and 6 children 12 years and over) with type I or II HAE who experienced at least two investigator-confirmed attacks within the first 8 weeks of the run-in period and took at least one dose of study treatment. Nine patients were aged = 65 years. Patients were randomised into 1 of 3 parallel treatment arms, stratified by baseline attack rate, in a 1:1:1 ratio (berotralstat 110 mg, berotralstat 150 mg or placebo by oral administration once daily, with food) for the 24-week treatment period.
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A total of 81 patients received at least one dose of berotralstat in the 24-week treatment period. Overall, 66% of patients were female and 93% of patients were Caucasian with a mean age of 41.6 years. A history of laryngeal angioedema attacks was reported in 74% of patients and 75% reported prior use of long-term prophylaxis. The median attack rate during the prospective run-in period (baseline attack rate) was 2.9 per month. Of patients enrolled, 70% had a baseline attack rate of = 2 attacks per month.
Patients discontinued other prophylactic HAE medicinal products prior to entering the study; however, all patients were allowed to use rescue medicinal products for treatment of breakthrough HAE attacks. In berotralstat-treated patients, 51.4% of breakthrough attacks were treated with C1-INH (see section "Warnings and precautions"). Concomitant use of C1-INH and berotralstat did not result in any identifiable adverse reactions.
www.swissmedic.ch/dam/swissmedic/it/d...Orladeyo —> Ruconest goeije combi