Takeda’s recombinant ADAMTS13 (rADAMTS13) has been recommended by the European Medicines Agency’s human medicines committee to treat ADAMTS13 deficiency in children and adults with congenital thrombotic thrombocytopenic purpura (cTTP).
If approved by the European Commission, rADAMTS13 will be the first and only enzyme replacement therapy in the EU indicated for the ultra-rare inherited blood clotting disorder.
Caused by a deficiency in the ADAMTS13 enzyme that regulates blood clotting, cTTP can result in severe bleeding episodes, stroke and damage to vital organs and, if left untreated, mortality rates are high.
Takeda’s rADAMTS13 is a purified recombinant form of the ADAMTS13 enzyme that works by providing a replacement for the low levels of the deficient enzyme.
The Committee for Medicinal Products for Human Use’s decision on the therapy was supported by a totality of evidence, including results from a late-stage trial of cTTP patients who were randomised to receive six months of rADAMTS13 or plasma-based therapies and then crossed over to the other treatment for another six. All patients then received rADAMTS13 for months 13 to 18.
No patient experienced an acute thrombotic thrombocytopenic purpura (TTP) event while receiving rADAMTS13 as a preventative treatment, while there was one acute TTP event in a patient receiving plasma-based therapies.
Additionally, no subacute TTP events were reported in patients receiving rADAMTS13 during periods one and two of the study, compared to five subacute TTP events in four patients receiving plasma-based therapies. In the continuation period, two patients receiving rADAMTS13 prophylaxis had two subacute events.
Obi Umeh, vice president, franchise global programme leader at Takeda, said: “People living with cTTP experience serious, potentially fatal health challenges and have limited treatment options in the EU.
“With this positive opinion for recombinant ADAMTS13, we are one step closer to offering patients in the EU the first treatment specifically indicated for cTTP.”
The recommendation comes just over six months after rADAMTS13 was approved by the US Food and Drug Administration, under the brand name Adzynma, as a preventative or on-demand treatment for adult and paediatric patients with cTTP.
The therapy has been approved in Japan for the same indication and is also being evaluating in adults with immune-mediated thrombotic thrombocytopenic purpura, the acquired form of TTP.