Regulatory Decision Summary for Haegarda

Hereditary angioedema (HAE) is a rare and potentially life-threatening genetic condition that occurs in about 1 in 10,000 to 1 in 50,000 population. Haegarda is the first subcutaneous therapy indicated for routine prophylaxis to prevent hereditary angioedema (HAE) attacks in adolescent and adult patients. The efficacy and safety of Haegarda, as replacement therapy, in the prevention of hereditary angioedema (HAE) attacks were demonstrated in clinical studies in adolescent and adult patients with HAE, administered by subcutaneous (SC) injection. Haegarda is expected to provide an advantage in the prevention of HAE attacks with the ease and flexibility of SC administration.

The most frequent adverse events observed in clinical studies were injection site reactions and they were mostly mild to moderate in intensity. To ensure the patients safety, the maximum SC injection dose was addressed in the Product Monograph. Other potential risk factors related to products derived from human plasma were also addressed in the Product Monograph.

Based on the clinical review of the efficacy and safety data, the overall benefit/risk profile of Haegarda is considered to be favourable for routine prophylaxis to prevent hereditary angioedema (HAE) attacks in adolescent and adult patients.