The European Commission (EC) has granted approval to Takeda for ADZYNMA (recombinant ADAMTS13) - the first and only enzyme replacement therapy in the European Union (EU) for congenital thrombotic thrombocytopenic purpura (cTTP).
cTTP is an ultra-rare, life-threatening blood clotting disorder caused by the ADAMTS13 enzyme deficiency.
ADZYNMA is approved to treat ADAM metallopeptidase with thrombospondin type 1 motif 13 (ADAMTS13) deficiency in children and cTTP in adults.
The approval follows a positive opinion from the Committee for Medicinal Products for Human Use, announced on 31 May 2024.
The EC's decision was informed by comprehensive data from a Phase III trial and subsequent continuation study, demonstrating the efficacy and safety of ADZYNMA.
In the pivotal Phase III trial, 45 patients treated with ADZYNMA did not experience any acute TTP events. One acute event was reported in 46 patients receiving plasma-based therapies.
Additionally, one patient experienced a subacute TTP event when treated with ADZYNMA, compared to seven events in six subjects on plasma-based treatments. These findings were consistent in the continuation phase of the trial.
ADZYNMA's safety profile was also favourable when compared to plasma-based therapies, with the most common adverse reactions being diarrhoea, headache, dizziness, nausea, upper respiratory tract infection and migraine.
Takeda is further evaluating recombinant ADAMTS13's potential in treating immune-mediated thrombotic thrombocytopenic purpura (iTTP) in an ongoing Phase IIb trial.
ADZYNMA also received approval from the US Food and Drug Administration (FDA) and the Japanese Ministry of Health, Labour and Welfare (MHLW) for the prophylactic and on-demand treatment of cTTP patients.
Takeda Europe and Canada Business Unit president Ricardo Marek stated: “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.”
In March 2024, Takeda secured approval from the MHLW for ADZYNMA Intravenous Injection 1500 (apadamtase alfa/cinaxadamtase alfa) to treat cTTP.
