European Commission approves Adzynma (recombinant ADAMTS13) for the treatment of ADAMTS13 deficiency in patients with congenital thrombotic thrombocytopenic purpura
Takeda announced that the European Commission (EC) approved Adzynma (recombinant ADAMTS13) for the treatment of ADAMTS13 deficiency in children and adult patients with congenital thrombotic thrombocytopenic purpura (cTTP)
Adzynma is now the first and only enzyme replacement therapy in the European Union (EU) specifically for the treatment of cTTP.
This approval includes confirmation of orphan medicinal product designation and follows a positive opinion from the Committee for Medicinal Products for Human Use, as announced by the company on May 31, 2024.
cTTP is an ultra-rare, chronic blood clotting disorder caused by a deficiency in the ADAMTS13 enzyme. It is associated with acute events and debilitating chronic symptoms or thrombotic thrombocytopenic purpura manifestations, which can include thrombocytopenia, microangiopathic hemolytic anemia, renal manifestations, stroke and abdominal pain. If left untreated, acute TTP events have a mortality rate of greater than 90%.
“A century after the scientific discovery of cTTP, significant unmet needs remain for patients who continue to face life-threatening acute events and debilitating chronic symptoms with limited treatment options,” said Ricardo Marek, President, Europe and Canada Business Unit at Takeda. “This approval marks the first treatment specifically indicated to address the root cause of the disease – ADAMTS13 deficiency. Building on our 70-year legacy of innovation in rare blood disorders, we’re proud to offer Adzynma to cTTP patients in the EU and remain committed to bringing innovative medicines to rare disease patients with high unmet need.”
The EC approval was supported by the totality of evidence provided by the interim analysis of efficacy, pharmacokinetic, safety and tolerability data from the first randomized, controlled open-label, crossover Phase III trial in cTTP, as well as safety and efficacy data from the continuation trial. Data from the Phase III trial (NCT03393975) were published in The New England Journal of Medicine in May 2024.
See- "Recombinant ADAMTS13 in Congenital Thrombotic Thrombocytopenic Purpura". Authors: Marie Scully, M.D., Ana Antun, M.D., Spero R. Cataland, M.D., Paul Coppo, M.D., Ph.D., Claire Dossier, M.D., Nathalie Biebuyck, M.D., Wolf-Achim Hassenpflug, M.D., +16, for the cTTP Phase III Study Investigators. Published May 1, 2024. N Engl J Med 2024;390:1584-1596 DOI: 10.1056/NEJMoa2314793,. VOL. 390 NO. 1.
Learning Zones
The Learning Zones are an educational resource for healthcare professionals that provide medical information on the epidemiology, pathophysiology and burden of disease, as well as diagnostic techniques and treatment regimens.
1 mins