Dive Brief:

潜水简介：

Massachusetts-based Syndax Pharmaceuticals won Food and Drug Administration approval Friday for a new kind of drug to treat an aggressive form of leukemia in adults and some children.

位于马萨诸塞州的Syndax Pharmaceuticals周五获得美国食品和药物管理局（FDA）的批准，开发一种新型药物，用于治疗成人和一些儿童的侵袭性白血病。

The oral drug, which Syndax will sell as Revuforj, is the first of its type, a class of compounds known as menin inhibitors. It’s cleared for patients one year or older who have relapsed or refractory acute leukemia that harbors a specific mutation: translocations in the lysine methyltransferase 2A, or KMT2A, gene..

Syndax将作为Revuforj销售的口服药物是其类型中的第一种，这类化合物被称为menin抑制剂。对于患有复发或难治性急性白血病且具有特定突变（赖氨酸甲基转移酶2A或KMT2A基因易位）的一岁或一岁以上患者，可以清除这种突变。。

People with this type of leukemia are more likely to relapse and have a median overall survival of less than one year. Syndax plans to launch two doses of the drug, which it priced at about $475,000 per year before rebates or discounts, later in November. A lower dose for patients who weigh less will be available next year..

这种白血病患者更容易复发，中位总生存期不到一年。Syndax计划在11月晚些时候推出两剂该药，在折扣或折扣之前，其价格为每年约47.5万美元。明年将为体重较轻的患者提供较低的剂量。。

Dive Insight:

潜水洞察：

The FDA’s decision comes more than a month before the agency’s Dec. 26 deadline.

FDA的决定是在该机构12月26日的最后期限前一个多月作出的。

The regulator cleared Revuforj based its analysis of data from 104 patients given Syndax’s drug in early-stage trials. Just over one-fifth of those patients experienced a complete remission, which lasted a median of about six months. Twenty-three percent of participants went on to receive a stem cell transplant after Revuforj..

监管机构根据Revuforj对104名在早期试验中服用Syndax药物的患者的数据分析，批准了Revuforj。超过五分之一的患者经历了完全缓解，持续时间中位数约为六个月。23%的参与者在Revuforj之后接受了干细胞移植。。

The drug’s labeling contains a black box warning for differentiation syndrome, which occurred in 29% of all 135 patients treated with Revuforj. Sometimes known as retinoic acid syndrome, differentiation syndrome is a potentially fatal reaction to cancer therapies in people with acute leukemia.

该药物的标签包含分化综合征的黑匣子警告，在所有135名接受Revuforj治疗的患者中，29%的患者发生了分化综合征。分化综合征有时被称为视黄酸综合征，是急性白血病患者对癌症治疗的潜在致命反应。

Jefferies analyst Kelly Shi, writing in a note to clients, said such a warning was expected for Revuforj and noted that the labeling is “similar to almost all other AML-targeted therapies.”

Jefferies分析师Kelly Shi在给客户的一封信中表示，预计Revuforj会收到这样的警告，并指出该标签“与几乎所有其他针对AML的疗法相似”

Syndax will first make 110 milligram and 160 milligram dose forms of Revuforj available, before readying a 25 milligram tablet.

Syndax将首先提供110毫克和160毫克剂量的Revuforj，然后再准备25毫克的片剂。

The company estimates about 2,000 people in the U.S. have acute leukemia with a KMT2A translocation. According to Shi, the company anticipates that “most patients will have commercial insurance coverage given [patients] younger age.”

该公司估计，美国约有2000人患有KMT2A易位的急性白血病。据施先生介绍，该公司预计“大多数患者在年龄较小的情况下都会有商业保险。”

Shi predicts peak annual sales of Revuforj to eventually reach about $200 million for the KMT2A indication alone.

Shi预测Revuforj单就KMT2A适应症而言，其年销售额峰值最终将达到约2亿美元。