Thanks to a Takeda approval Thursday, patients now have a drug to tackle a rare inherited blood clotting disorder.

由于周四获得武田批准，患者现在可以使用药物治疗罕见的遗传性凝血障碍。

The U.S. FDA has given a green light to Takeda’s Adzynma, the first recombinant protein product as a preventative or on demand enzyme replacement therapy in adults and kids with congenital thrombotic thrombocytopenic purpura (cTTP).

美国FDA为武田的Adzynma发出了绿灯，这是第一种重组蛋白产品，可作为成人和患有先天性血栓性血小板减少性紫癜（cTTP）的儿童的预防性或按需酶替代疗法。

According to the FDA, cTTP is a very rare inherited blood clotting disorder caused by a disease-causing mutation in the ADAMTS13 gene, which makes an enzyme responsible for regulating blood clotting. Patients with a deficiency in this enzyme create blood clots in the small blood vessels throughout the body.

根据FDA的说法，cTTP是一种非常罕见的遗传性凝血疾病，由ADAMTS13基因的致病突变引起，该突变使一种负责调节凝血的酶。这种酶缺乏的患者会在整个身体的小血管中产生血栓。

The disease is estimated to affect fewer than 1,000 people in the U.S..

据估计，这种疾病在美国影响不到1000人。。

While symptoms of cTTP typically develop in infancy or early childhood, they can also crop up in adulthood and may manifest first during pregnancy, the FDA said. People with cTTP can suffer from severe bleeding episodes, strokes and damage to vital organs.

FDA表示，虽然cTTP的症状通常发生在婴儿期或幼儿期，但它们也可以在成年期出现，并可能在怀孕期间首先出现。cTTP患者可能患有严重的出血事件，中风和重要器官损伤。

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武田在Alofisel失败后花费了7.7亿美元的时间，做出退出决定

Until now, treatment has typically revolved around prophylactic plasma-based therapy, the regulator added.

到目前为止，监管机构补充说，治疗通常围绕预防性血浆治疗。

Takeda’s Adzynma, for its part, is a recombinant form of the ADAMTS13 gene that helps patients by replacing low levels of the deficient enzyme. For protective enzyme replacement therapy, the drug is given to help reduce the risk of disease symptoms, though it can also be given on demand when patients are dealing with acute events..

武田的Adzynma本身就是ADAMTS13基因的重组形式，可通过替换低水平的缺陷酶来帮助患者。对于保护性酶替代疗法，尽管在患者处理急性事件时也可以根据需要给予该药物，以帮助降低疾病症状的风险。。

Adzynma is administered through the vein once every other week in its protective form, and it’s given once daily for on-demand enzyme replacement therapy.

Adzynma以保护形式每隔一周静脉注射一次，每天给予一次按需酶替代疗法。

The FDA based its approval on a trial of 46 cTTP patients who either got six months of treatment with Adzynma or plasma-based therapies. Patients crossed over to the other drug after the study’s six-month mark, the FDA said.

FDA的批准基于对46名cTTP患者的试验，这些患者接受了Adzynma或基于血浆的疗法治疗6个月。FDA表示，在研究六个月后，患者转入另一种药物。

Efficacy was determined based on incidence of thrombotic thrombocytopenic purpura (TTP) events and TPP manifestations, plus the incidence of the need for supplemental doses.

根据血栓性血小板减少性紫癜（TTP）事件和TPP表现的发生率以及需要补充剂量的发生率确定功效。

For on-demand enzyme replacement therapy, Takeda looked at the proportion of acute TTP events responding to its drug in both the prophylactic and on-demand cohorts throughout the study’s run. All acute and subacute TTP events were resolved after treatment with either Adzynma or plasma-based therapies, the FDA explained..

对于按需酶替代疗法，武田在整个研究过程中研究了预防和按需队列中对其药物有反应的急性TTP事件的比例。FDA解释说，用Adzynma或基于血浆的疗法治疗后，所有急性和亚急性TTP事件均得到解决。。

In terms of safety, the most common side effects associated with Adzynma were headache, diarrhea, migraine, abdominal pain, nausea, upper respiratory tract infection, dizziness and vomiting.

在安全性方面，与Adzynma相关的最常见副作用是头痛，腹泻，偏头痛，腹痛，恶心，上呼吸道感染，头晕和呕吐。

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The green light for Adzynma marks Takeda’s second approval this week. The company has also received an FDA all-clear on its VEGFR inhibitor Fruzaqla, also known as fruquintinib, in adults with metastatic colorectal cancer as a third-line treatment.

Adzynma的绿灯标志着武田本周的第二次批准。作为三线治疗，该公司还获得了VEGFR抑制剂Fruzaqla（也称为fruquintinib）在转移性结直肠癌患者中的全部FDA。

Takeda pushed fruquintinib over the FDA finish line after acquiring its rights outside of China in January. The Tokyo company paid Hutchmed $400 million up front, with the potential for $730 million in milestone payments.

武田在1月份在中国境外获得权利后，将fruquintinib推向FDA终点线。东京公司提前支付了4亿美元，里程碑付款可能达到7.3亿美元。