Takeda Announces European Commission Approval of ADZYNMA® (Recombinant ADAMTS13) as First and Only Enzyme Replacement Therapy for Congenital Thrombotic Thrombocytopenic Purpura

August 7, 2024 (Osaka and Cambridge, Massachusetts)

Takeda Pharmaceutical Company Limited announced that the European Commission (EC) has approved ADZYNMA®▼(recombinant ADAMTS13) for the treatment of ADAMTS13 deficiency in pediatric and adult patients with congenital thrombotic thrombocytopenic purpura (cTTP).

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

ADZYNMA（recombinant ADAMTS13）

apadamtase alfa /cinaxadamtase alfa

It is the first and only approved recombinant "A Disintegrin And Metalloproteinase with Thrombospondin type 1 motif, member 13" (ADAMTS13) enzyme replacement therapy for the treatment of pediatric and adult patients with congenital thrombotic thrombocytopenic purpura (cTTP) due to ADAMTS13 deficiency.

• ADZYNMA has also received approval from the U.S. Food and Drug Administration (FDA) and Japan's Ministry of Health, Labour and Welfare (MHLW) for both prophylactic and on-demand treatment in patients with cTTP.

• ADZYNMA Granted Orphan Drug Designation (ODD) by the U.S. FDA for the Treatment and Prevention of TTP, Including Its Acquired Idiopathic and Secondary Forms, as well as Fast Track and Rare Pediatric Disease Designations.

• U.S. FDA Grants Takeda Rare Pediatric Disease Priority Review Voucher for Approval of ADZYNMA.

• ADZYNMA has also been granted ODD by the European Medicines Agency (EMA) and Japan's Ministry of Health, Labour and Welfare for the treatment of TTP.

Rare Neurological Disorders Communication Group

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