• 09 Aug 2024
• Admin
• News Article

Takeda Announces Approval for ADZYNMA® as First cTTP Therapy

Overview

Takeda has received approval from the European Commission (EC) for ADZYNMA® (recombinant ADAMTS13) to treat ADAMTS13 deficiency in children and adults with congenital thrombotic thrombocytopenic purpura (cTTP).

About ADZYNMA

• ADZYNMA (recombinant ADAMTS13) is the first enzyme replacement therapy approved in the EU for treating ADAMTS13 deficiency in cTTP patients. 
• It is also approved by the US FDA and the Japanese Ministry of Health, Labour, and Welfare for prophylactic and on-demand treatment of cTTP. 
• ADZYNMA has been granted Orphan Drug Designation by the US FDA and the European Medicines Agency, and Fast Track and Rare Pediatric Disease Designation by the US FDA.

About cTTP

• cTTP is a very rare, chronic blood clotting disorder caused by a deficiency of the ADAMTS13 enzyme. 
• It leads to serious symptoms like thrombocytopenia, microangiopathic hemolytic anemia, kidney problems, strokes, and abdominal pain. 
• Without treatment, acute TTP events have a mortality rate of over 90%.

First Enzyme Replacement Therapy in EU

This makes ADZYNMA the first enzyme replacement therapy in the European Union (EU) specifically for cTTP. The approval, which follows a positive opinion from the Committee for Medicinal Products for Human Use announced on May 31, 2024, also confirms its orphan medicinal product designation.

Behind cTTP—ADAMTS13 Deficiency

• Despite a century of research, cTTP patients still face life-threatening events and chronic symptoms with few treatment options. 
• This approval addresses the root cause of cTTP—ADAMTS13 deficiency. Building on our long history of innovation in rare blood disorders, we are proud to offer ADZYNMA to EU patients and remain dedicated to providing innovative treatments for rare diseases.

Approval by EC

The EC's approval was based on data from a Phase 3 trial, including interim analysis of efficacy, pharmacokinetics, safety, and tolerability. Results were published in The New England Journal of Medicine in May 2024.

Phase 3 Trial Process

• In the Phase 3 trial, patients were given 40 IU/kg of ADZYNMA IV or plasma-based therapy every other week or weekly for the first six months, then switched treatments for the next six months. 
• All patients received ADZYNMA for months 13-18. 
• No acute TTP events occurred in patients receiving ADZYNMA, while one event occurred in a patient on plasma-based therapy. 
• One subacute TTP event was reported in a patient on ADZYNMA compared to seven subacute events in six patients on plasma-based therapies.

ADZYNMA Safety Profile

ADZYNMA showed a favourable safety profile compared to plasma-based therapies. Common side effects included headache, diarrhoea, dizziness, upper respiratory tract infections, nausea, and migraines.

Takeda 

Takeda is also investigating recombinant ADAMTS13 for immune-mediated thrombotic thrombocytopenic purpura (iTTP) in an ongoing Phase 2b trial.