Takeda Pharmaceutical Co Ltd (NYSE:TAK) stock is trading higher on Wednesday. Earlier today, the European Commission approved Takeda's Adzynma (recombinant ADAMTS13) for treating ADAMTS13 deficiency in children and adult patients with congenital thrombotic thrombocytopenic purpura (cTTP).

Congenital thrombotic thrombocytopenic purpura, also known as Upshaw-Schulman syndrome, is a rare inherited disorder that affects the blood's ability to clot properly. This condition leads to the excessive formation of blood clots in small blood vessels throughout the body (a process known as thrombosis), which can cause serious health problems.

Adzynma is now the first and only enzyme replacement therapy in the European Union (EU) specifically for the treatment of cTTP.

cTTP is an ultra-rare, chronic blood clotting disorder caused by a deficiency in the ADAMTS13 enzyme.

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The EC approval was supported by the totality of evidence provided by the interim analysis of efficacy, pharmacokinetic, safety and tolerability data from the first randomized, controlled open-label, crossover Phase 3 trial in cTTP, as well as safety and efficacy data from the continuation trial.

In the Phase 3 trial, patients received 40 IU/kg Adzynma IV or plasma-based therapy every other week or weekly, based on the regimen at enrollment for months 1-6 (period 1), crossing over to the alternate treatment for months 7-12 (period 2), and all patients received Adzynma for months 13-18 (period 3).

No patient experienced an acute TTP event while receiving Adzynma prophylactic treatment (n=45), while there was one acute TTP event in a patient receiving plasma-based therapies (n=46).

One subacute TTP event was reported in one patient receiving Adzynma during the controlled comparison periods 1 and 2, compared to seven subacute TTP events in six patients receiving plasma-based therapies.

In the continuation phase (period 3), efficacy results – incidence rates of acute and subacute TTP events – were consistent with the results from periods 1 and 2.

Adzynma demonstrated a favorable safety profile compared to plasma-based therapies.

Takeda is conducting an ongoing Phase 2 b trial to investigate recombinant ADAMTS13 in adults with immune-mediated thrombotic thrombocytopenic purpura (iTTP), the acquired form of TTP.

Price Action: TAK stock is up 3.87% at $13.83 at the last check on Wednesday.

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