Apadamtase alfa, sold under the brand name Adzynma, is an enzyme replacement therapy used for the treatment of thrombotic thrombocytopenic purpura.
[1] The most common side effects include headache, diarrhea, migraine, abdominal pain, nausea, upper respiratory tract infection, dizziness and vomiting.
[3] Apadamtase alfa is indicated for prophylactic (preventive) or on demand enzyme replacement therapy in people with congenital thrombotic thrombocytopenic purpura.
[5] The efficacy of on demand enzyme replacement therapy was evaluated based on the proportion of acute thrombotic thrombocytopenic purpura events responding to apadamtase alfa in both the prophylactic and the on-demand cohorts throughout the duration of the study.
[5] All acute and subacute thrombotic thrombocytopenic purpura events resolved after treatment with either apadamtase alfa or plasma based therapies.