Takeda's Adzynma (apadamtase alfa) Becomes First FDA-Approved Enzyme Replacement Therapy for cTTP, a Disorder with Over 90% Untreated Mortality

▎Edited by the WuXi AppTec content team

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Today, Takeda announced that the U.S. FDA has approved the marketing application for its enzyme replacement therapy Adzynma (apadamtase alfa).According to the press release, this is the FDA-approved treatment forAdult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP)The First ProductEnzyme Replacement Therapy (ERT).

This application is supported by efficacy, pharmacokinetic, safety, and tolerability data from the first randomized controlled trial in cTTP, along with positive long-term safety and effectiveness data. This Phase 3 trial aims to evaluate the benefits of TAK-755 across multiple clinically relevant endpoints. Interim results announced by Takeda in January 2023 showed,Compared with plasma-based therapies, TAK-755 reduced the incidence of thrombocytopenia events by 60% (95% CI, 30%-70%)., which is an important indicator of disease activity in cTTP and also the most frequently observed clinical symptom.

In terms of safety,The incidence of treatment-related adverse events was significantly lower in subjects treated with TAK-755 than in those treated with plasma (8.9% vs 47.7%).. In addition, the interim analysis results of a Phase 3b extension study indicate that,TAK-755 Preventive Therapy Shows Good Safety, No Neutralizing Antibodies Detected.

TAK-755 is an ADAMTS13ReorganizationProtein can replace the missing or malfunctioning ADAMTS13 enzyme, the lack of which is the root cause of cTTP.By supplementing the function of the ADAMTS13 enzyme, it can effectively improve both acute and chronic symptoms of cTTP, as well as other complications caused by ADAMTS13 deficiency, thereby enhancing the quality of life and lifespan of patients with cTTP. Previously, TAK-755 also received Fast Track designation and Orphan Drug designation from the U.S. FDA, and in May this year, it obtainedPriority Review Status

, for the prevention and treatment of cTTP.

References:

[1] FDA Approves First Treatment for Patients with Rare Inherited Blood Clotting Disorder. Retrieved November 9, 2023 from https://www.prnewswire.com/news-releases/fda-approves-first-treatment-for-patients-with-rare-inherited-blood-clotting-disorder-301983923.html

[2] U.S. Food & Drug Administration Grants Priority Review of TAK-755 for the Treatment of Congenital Thrombotic Thrombocytopenic Purpura (cTTP). Retrieved November 8, 2023, from https://www.businesswire.com/news/home/20230516006066/en

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