Takeda Wins First FDA Approval for Ultra-Rare Blood Clotting Disease

2023-11-10

临床结果临床3期上市批准孤儿药快速通道

Pictured: Takeda's office in Cambridge, Massachusetts/iStock, hapabapa The FDA on Thursday approved Takeda’s recombinant ADAMTS13, now to be marketed as Adzynma, for the treatment of the rare blood disorder congenital thrombotic thrombocytopenic purpura—the biopharma company’s second approval in as many days. Adzynma is indicated as an intravenous prophylactic and on-demand therapy of congenital thrombotic thrombocytopenic purpura (cTTP) in both adults and children. Afflicting two to six patients in every million people, cTTP is an ultra-rare and debilitating clotting disorder characterized by thrombocytopenia, red blood cell destruction, headaches and abdominal pain. When left unchecked, cTTP can lead to widespread organ damage and reach mortality rates of almost 90%. According to Julie Kim, president of Takeda’s U.S. business unit, Adzynma is the “first FDA-approved therapeutic option for people with cTTP,” who often face “serious, life-threatening health challenges” and are left with no treatment options specifically designed to treat their conditions. The disease is caused by a deficiency in ADAMTS13, an enzyme that cleaves the glycoprotein von Willebrand factor (VWF). Faulty ADAMST13 results in the accumulation of ultra-large VWF complexes in the blood, which in turn leads to the uncontrolled aggregation and adhesion of platelets. Adzynma addresses the underlying cause of cTTP by delivering a functional human recombinant ADAMTS13 protein, restoring the body’s ability to break down the large VWF complexes and prevents the formation of clots. This mechanism of action won Adzynma the FDA’s Orphan Drug, Fast Track and Rare Pediatric Disease designations for the treatment and prevention of cTTP. The FDA’s approval on Thursday was supported by data from a pivotal Phase III trial, as well as additional evidence from a continuation trial. In June 2023, Takeda presented findings from the Phase III trial at the 2023 Congress of the International Society on Thrombosis and Hemostasis. Results showed that Adzynma cut the incidence of thrombocytopenia by 60% compared to a plasma-based control therapy. None of the patients receiving prophylactic Adzynma experienced an acute TTP event. Adzynma’s data package also showed a favorable safety pro the therapy. The most common side effects were headache, migraine, diarrhea, nausea, abdominal pain and vomiting. None of the patients treated with Adzynma developed neutralizing antibodies. While Adzynma’s label does not carry a boxed warning, it comes with precautions against hypersensitivity reactions as well as the risk of developing antibodies against Adzynma, which might lead to a “decreased or lack of response” to the treatment. Thursday’s approval comes just a day after Takeda also won the FDA’s greenlight for its fruquintinib, now being marketed as Fruzaqla, the “first and only selective inhibitor of all three VEGF receptor kinases” for previously treated metastatic colorectal cancer regardless of biomarker status. Tristan Manalac is an independent science writer based in Metro Manila, Philippines. He can be reached at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

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