Takeda has received the US Food and Drug Administration (FDA) approval for Adzynma (ADAMTS13, recombinant-krhn) for the treatment of patients with congenital thrombotic thrombocytopenic purpura (cTTP).

Adzynma is a human recombinant ADAMTS13 (rADAMTS13) protein. It is designed to replace the deficient ADAMTS13 enzyme in people with cTTP.

The FDA has approved Adzynma for the prophylactic and on-demand treatment of adult and paediatric patients with cTTP.

The approval was based on the totality of the evidence provided by the analysis of efficacy, pharmacokinetic, safety and tolerability data from the Phase 3 trial in cTTP as well as by data from the continuation trial.

In the first randomised, controlled, open-label, crossover late-stage study, the participants received 40 IU/kg Adzynma IV or plasma-based therapy.

The treatment was administered every other week or weekly based on the regimen at enrolment for months 1-6, crossing over to the alternate treatment for months 7-12, and all patients received Adzynma for months 13-18.

As per the results, no patient experienced an acute thrombotic thrombocytopenic purpura (TTP) event during Adzynma prophylactic treatment, while there was one acute TTP event in a patient receiving plasma-based therapies.

Additionally, no subacute TTP events were observed in patients receiving the therapy during months 1-6 and months 7-12, compared to five subacute TTP events in four patients receiving plasma-based therapies.

In the continuation period, two patients who were administered Adzynma prophylaxis had two subacute events.

Furthermore, the human recombinant protein showed a favourable safety profile in comparison to plasma-based therapies, the Japanese pharmaceutical firm said.

Takeda US country head and business unit president Julie Kim said: “As we strive to help patients with limited or no treatment options, developing innovative treatments in rare diseases is an inspiring challenge and one we have taken on for 70-plus years as a leader in haematology.

“Today, we are proud to further support the rare disease community by delivering Adzynma as the first FDA-approved therapeutic option for people with cTTP.”

Previously, Adzynma was granted Orphan Drug Designation (ODD) by the US FDA for the treatment and prevention of TTP.