Takeda Receives Positive CHMP Opinion for Recombinant ADAMTS13 rADAMTS13 in Congenital Thrombotic Thrombocytopenic Purpura cTTP


Osaka, Japan & Cambridge, Mass., United States:

    • cTTP Is an Ultra-rare, Potentially Fatal Blood-Clotting Disorder with Limited Treatment Options; Untreated, Acute TTP Events Have a Mortality Rate of >90%1,2
    • If Approved in the European Union, rADAMTS13 Will Be the First and Only Recombinant ADAMTS13 Enzyme Replacement Therapy for cTTP
    • Positive Opinion Based on Totality of Evidence, Including Results from the First Randomized, Controlled, Open-label, Crossover Phase 3 cTTP Trial

Takeda (TSE:4502/NYSE:TAK) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval, under exceptional circumstances, of recombinant ADAMTS13 (rADAMTS13) for the treatment of ADAMTS13 deficiency in children and adult patients with cTTP. The European Commission (EC) will consider the CHMP positive opinion when determining the potential marketing authorization for rADAMTS13 throughout the European Union (EU). If approved, rADAMTS13 will be the first and only enzyme replacement therapy in the EU for the treatment of cTTP.3

“People living with cTTP experience serious, potentially fatal health challenges and have limited treatment options in the European Union,” said Obi Umeh, M.D., M.Sc., Vice President, Franchise Global Program Leader at Takeda. “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. We look forward to the European Commission’s decision as we aspire to transform the standard of care for cTTP for more patients around the world.”

cTTP is an ultra-rare, chronic blood clotting disorder caused by a deficiency in the ADAMTS13 enzyme. It is associated with acute events and debilitating chronic symptoms or thrombotic thrombocytopenic purpura (TTP) manifestations, which can include thrombocytopenia, microangiopathic hemolytic anemia, renal manifestations, stroke and abdominal pain. Untreated, acute TTP events have a mortality rate of >90%.

The Committee’s positive opinion was supported by the totality of evidence including the interim analysis of efficacy, pharmacokinetic, safety, and tolerability data from the first randomized, controlled open-label, crossover Phase 3 trial in cTTP. Data from this trial (NCT03393975) were published in The New England Journal of Medicine in May 2024. rADAMTS13 is also being investigated in adults with immune-mediated thrombotic thrombocytopenic purpura (iTTP), the acquired form of TTP, in an ongoing Phase 2b trial (NCT05714969).