Takeda has announced that the Japanese Ministry of Health, Labour and Welfare (MHLW) has approved ADZYNMA Intravenous Injection 1500 (apadamtase alfa/cinaxadamtase alfa) to treat congenital thrombotic thrombocytopenic purpura (cTTP), a rare blood clotting disorder.
A human recombinant A disintegrin and metalloproteinase with thrombospondin motifs ADAMTS13, ADZYNMA is indicated for use in those aged 12 years and above.
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It is the first recombinant ADAMTS13 protein to obtain approval for the treatment of cTTP.
The approval is based on interim analysis data from a Phase III clinical trial and long-term safety and efficacy results from a continuation study.
The controlled, randomised, open-label, crossover Phase III trial enrolled cTTP patients between 12 and 68 years, including five Japanese patients, to assess the efficacy, pharmacokinetic, safety and tolerability of ADZYNMA.
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No acute TTP events were reported in patients under prophylactic treatment with ADZYNMA during the interim analysis.
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By GlobalDataAn acute TTP event was reported in a patient who received plasma-based therapies during the Phase III study-controlled comparison periods 1 and 2.
Treatment-emergent adverse events (TEAEs) linked to ADZYNMA were significantly lower than on plasma-based therapies.
Only 10.3% of patients treated with ADZYNMA experienced TEAEs, while 50% of those on plasma-based therapy did so.
Constipation, abnormal ADAMTS13 activity, headache, pruritus and hypertension were the TEAEs reported in the ADZYNMA arm.
ADZYNMA is already approved by the US Food and Drug Administration (FDA) for both prophylactic and on-demand treatment of cTTP in adults and children.
Takeda research and development head for Japan Yasushi Kajii stated: “The approval of ADZYNMA is an important milestone for people living with cTTP in Japan, who had limited treatment options and now have the first treatment option specifically approved to treat this ultra-rare condition.
“Developing innovative treatments that make a difference in the lives of patients is at the heart of what we do. With this approval, we are proud to support the cTTP community with new possibilities and continue our 70-plus year commitment to the rare disease community.”
The development follows Takeda’s recent FDA approval for EOHILIA, an oral suspension for treating eosinophilic esophagitis.
