Posted by Michael Wonder on 09 Nov 2023
FDA approves first treatment for patients with rare inherited blood clotting disorder
9 November 2023 - Today, the US FDA approved Adzynma, the first recombinant protein product indicated for prophylactic or on demand enzyme replacement therapy in adult and paediatric patients with congenital thrombotic thrombocytopenic purpura, a rare and life-threatening blood clotting disorder.
The very rare, inherited blood clotting disorder is caused by a disease-causing mutation in the ADAMTS13 gene, which is responsible for making an enzyme, also named ADAMTS13, that regulates blood clotting.
