Takeda Gains EU Approval for ADZYNMA as First Recombinant ADAMTS13 Therapy for cTTP

Takeda, a global pharmaceutical company based in Osaka, Japan, and Cambridge, MA, USA, has announced that the European Commission (EC) has approved ADZYNMA (recombinant ADAMTS13) for the treatment of ADAMTS13 deficiency in patients suffering from congenital thrombotic thrombocytopenic purpura (cTTP). This approval makes ADZYNMA the first enzyme replacement therapy specifically designed for cTTP available in the European Union (EU).

cTTP is an extremely rare and chronic blood clotting disorder caused by a deficiency in the enzyme ADAMTS13. This condition can lead to severe acute events and chronic symptoms such as thrombocytopenia, microangiopathic hemolytic anemia, renal complications, strokes, and abdominal pain. Without treatment, acute TTP events have a mortality rate exceeding 90%.

Ricardo Marek, the President of Takeda's Europe and Canada Business Unit, highlighted the importance of this approval, mentioning that it addresses the fundamental cause of the disease—ADAMTS13 deficiency. Marek emphasized Takeda's long-standing commitment to innovation in treating rare blood disorders and expressed pride in offering a new treatment option for cTTP patients in the EU.

The EC's approval of ADZYNMA is based on comprehensive evidence from the first randomized, controlled, open-label, crossover Phase 3 trial in cTTP. The trial's interim analysis provided data on efficacy, pharmacokinetics, safety, and tolerability, which were published in The New England Journal of Medicine in May 2024. In this Phase 3 trial, patients received ADZYNMA every other week or weekly for six months, switching to alternate treatments for another six months. The trial concluded with all patients receiving ADZYNMA for the final six months.

No acute TTP events occurred in patients receiving ADZYNMA prophylactic treatment, while one acute event was reported in a patient receiving plasma-based therapies. During the trial's controlled comparison periods, one subacute TTP event occurred in a patient receiving ADZYNMA, compared to seven subacute events in patients receiving plasma-based therapies. The continuation phase showed consistent efficacy results, and ADZYNMA demonstrated a favorable safety profile, with common adverse reactions including headaches, diarrhea, dizziness, upper respiratory tract infections, nausea, and migraines.

Takeda is also exploring the potential of recombinant ADAMTS13 in treating immune-mediated thrombotic thrombocytopenic purpura (iTTP), the acquired form of TTP, in an ongoing Phase 2b trial.

ADZYNMA has received several designations to expedite its development and approval. It has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for treating and preventing TTP, including its acquired forms. The FDA has also given ADZYNMA Fast Track and Rare Pediatric Disease Designations, awarding Takeda a Rare Pediatric Disease Voucher. Additionally, the European Medicines Agency (EMA) and the Japanese Ministry of Health, Labour, and Welfare (MHLW) have granted ADZYNMA ODD status for treating TTP.

cTTP, despite its rarity, poses substantial health risks due to its acute and chronic manifestations. It results from a deficiency in ADAMTS13, leading to the accumulation of large von Willebrand factor (VWF) multimers in the blood. This accumulation causes uncontrolled platelet aggregation and adhesion, leading to abnormal clotting in small blood vessels, microangiopathic hemolytic anemia, and low platelet levels. The disorder can result in extensive organ damage and comorbidities due to the deficient state of ADAMTS13.

Takeda continues to focus on providing transformative treatments for various medical conditions, including rare diseases, through its research-driven approach. The company's dedication to patient care and innovative therapies aims to enhance health outcomes and offer new solutions for those with high unmet medical needs.

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