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The enzyme replacement therapy is now the first treatment approved in the UK for this patient population
Takeda’s Adzynma (rADAMTS13) has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) to treat congenital thrombotic thrombocytopenic purpura (cTTP).
The enzyme replacement therapy, which has been authorised to treat cTTP in patients of all ages, is now the first treatment approved in the UK for the ultra-rare blood clotting disorder.
cTTP is an inherited condition caused by a deficiency in ADAMTS13, an enzyme critical to blood clotting. Patients can experience a variety of symptoms and severe complications, including thrombocytopenia, stroke, and renal and cardiovascular disease.
Administered intravenously, Takeda’s Adzynma is a purified recombinant form of ADAMTS13 that is designed to provide a replacement for the low levels of the deficient enzyme.
The MHRA’s decision on Adzynma occurred through the International Recognition Procedure, a fast-track approval process that takes into account prior authorisations from other regulatory partners, and follows the European Commission’s approval of the therapy in August.
Among the evidence supporting the EU authorisation were results from a phase 3 trial of cTTP patients who were randomised to receive six months of Adzynma or plasma-based therapies and then crossed over to the other treatment for another six. All patients then received Takeda’s therapy for months 13 to 18.
No patient experienced an acute thrombotic thrombocytopenic purpura (TTP) event while receiving Adzynma as a preventative treatment, while there was one acute TTP event in a patient receiving plasma-based therapies.
Additionally, one subacute TTP event was seen in the Adzynma cohort during periods one and two of the study, versus seven subacute TTP events in six patients receiving plasma-based therapies. Efficacy results were consistent in the continuation period, the company said.
“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,” Ricardo Marek, president, Europe and Canada business unit, Takeda, said in August.
Adzynma was also approved by the US Food and Drug Administration for CTTP in 2023.
