Syndax Pharmaceuticals白血病药物获得美国食品药品监督管理局（FDA）一流批准-动脉网

Syndax Pharmaceuticals Leukemia Drug Wins a First-in-Class FDA Approval

MedCity News 等信源发布 2024-11-19 02:14

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Syndax Pharmaceuticals has won FDA approval for a drug addressing advanced cases of acute leukemia carrying a particular genetic signature that leads to an aggressive form of the disease. The regulatory decision makes the Syndax drug the first therapy in a new class of medicines for blood cancers.

Syndax制药公司（Syndax Pharmaceuticals）已获得美国食品和药物管理局（FDA）的批准，该药物用于治疗晚期急性白血病病例，该病例携带特定的遗传特征，导致该疾病的侵袭性形式。监管决定使Syndax药物成为治疗血癌的新型药物中的第一种。

The FDA approval specifically covers the treatment of adults and children age 1 and older. The Syndax drug, a twice-daily pill known in development as revumenib, will be marketed under the brand name Revuforj. The product’s late Friday approval came six weeks ahead of the target date for a regulatory decision..

FDA的批准特别涵盖了成人和1岁及以上儿童的治疗。Syndax药物是一种每日两次的避孕药，在开发过程中被称为revumenib，将以Revuforj品牌销售。该产品于周五晚些时候获得批准，比监管决定的目标日期提前了六周。。

In leukemia, the proliferation of abnormal white blood cells prevents bone marrow from producing red blood cells and platelets. Chemotherapy is a standard treatment. Blood transfusions do not cure the disease, but they can boost a patient’s levels of red blood cells and platelets.

在白血病中，异常白细胞的增殖阻止骨髓产生红细胞和血小板。化疗是一种标准治疗方法。输血不能治愈疾病，但可以提高患者的红细胞和血小板水平。

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PurpleLab®通过向医疗保健和制药公司的多个不同用户群体提供数据分析服务，在该领域脱颖而出。

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Waltham, Massachusetts-based Syndax designed Revuforj to inhibit a protein called menin. In patients who have a rearrangement of the KMT2A gene, menin activates pathways that drive cancer growth. This particular genetic rearrangement drives an estimated 10% of acute leukemias, Syndax said in an investor presentation.

位于马萨诸塞州沃尔瑟姆的Syndax设计了Revuforj来抑制一种名为menin的蛋白质。在KMT2A基因重排的患者中，menin激活驱动癌症生长的途径。Syndax在投资者介绍中表示，这种特殊的基因重排导致了大约10%的急性白血病。

Patients whose cancer carries this genetic signature have poor prognoses and high rates of drug resistance and relapse. Revuforj is a small molecule that blocks the interaction of menin with KMT2A fusion proteins..

携带这种基因特征的癌症患者预后不佳，耐药性和复发率很高。Revuforj是一种小分子，可阻断menin与KMT2A融合蛋白的相互作用。。

Syndax evaluated Revuforj in a single-arm, open-label Phase 1/2 study that enrolled 104 participants, both adults and pediatric patients. Results showed that treatment led to complete remission or complete remission with partial hematologic recovery in 21.2% of study participants. The median duration of the response was 6.4 months.

。结果显示，21.2%的研究参与者的治疗导致完全缓解或完全缓解，部分血液学恢复。反应的中位持续时间为6.4个月。

Syndax said 23% (24 out of 104) of patients in the study underwent stem cell transplants after treatment with Revuforj. This procedure can restore a patient’s ability to produce blood cells. Results from the study were published in August in the Journal of Clinical Oncology. The company said more data will presented next month during the annual meeting of the American Society of Hematology..

Syndax说，23%（104名患者中的24名）在接受Revuforj治疗后接受了干细胞移植。这种手术可以恢复患者产生血细胞的能力。这项研究的结果发表在八月的《临床肿瘤学杂志》上。该公司表示，下个月将在美国血液学会年会上提供更多数据。。

Revuforj’s label carries a black box warning for the risk of differentiation syndrome, a complication in which a cancer drug triggers an excessive immune response from the affected leukemia cells. Differentiation syndrome can become fatal if it leads to failure of key organs, such as the heart. According to the Revuforj’s label, if differentiation syndrome is suspected, clinicians should start corticosteroid therapy and monitor the patient’s blood circulation and heart until symptoms resolve..

Revuforj的标签上有一个关于分化综合征风险的黑匣子警告，分化综合征是一种癌症药物引发受影响白血病细胞过度免疫反应的并发症。如果分化综合征导致心脏等关键器官衰竭，则可能致命。根据Revuforj的标签，如果怀疑有分化综合征，临床医生应该开始皮质类固醇治疗，并监测患者的血液循环和心脏，直到症状消失。。

“The FDA approval of the first menin inhibitor is a major breakthrough for patients with [relapsed/refractory] acute leukemia with a KMT2A translocation, a genetic alteration associated with a very poor prognosis,” Dr. Ghayas Issa, associate professor of leukemia at The University of Texas MD Anderson Cancer Center,” said in Syndax’s announcement of the approval.

德克萨斯大学MD安德森癌症中心白血病副教授Ghayas Issa博士在Syndax的批准公告中表示：“FDA批准第一种menin抑制剂对于患有KMT2A易位的[复发/难治性]急性白血病患者是一项重大突破，KMT2A易位是一种与预后极差相关的基因改变。”。

“The significant clinical benefit and robust efficacy seen with Revuforj represents a substantial improvement over what has been historically observed in these patients with previously available therapies and has the potential to be an important new treatment option for patients.”.

“Revuforj所带来的显着临床益处和强大的疗效代表了对这些先前可用疗法的患者历史上观察到的显着改善，并且有可能成为患者重要的新治疗选择。”。

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随着技术的进步，人工智能工具将越来越多地减轻医疗保健提供者的行政负担。

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While Revuforj is now the first FDA-approved menin inhibitor, potential competitors are on its heels. Kura Oncology has reached the Phase 2 portion of a Phase 1/2 test of ziftomenib in advanced acute myeloid leukemia. Icovamenib, a menin inhibitor from Biomea Fusion, is in early clinical development in both liquid and solid tumors.

虽然Revuforj现在是FDA批准的第一种menin抑制剂，但潜在的竞争对手紧随其后。库拉肿瘤学已经达到了ziftomenib治疗晚期急性髓细胞白血病的1/2期试验的2期部分。Icovamenib是Biomea Fusion的menin抑制剂，在液体和实体瘤中均处于早期临床开发阶段。

Other companies developing small molecule menin inhibitors include Johnson & Johnson, Sumitomo Dainippon, and Daiichi Sankyo..

其他开发小分子menin抑制剂的公司包括强生公司、住友大力邦公司和第一三共公司。。

Meanwhile, Syndax is running additional studies that could support expanding Revuforj to use as an earlier line of treatment for leukemia characterized by a KMT2A translocation as well as for leukemias driven by a genetic mutation to mNPM1 gene, which is found in about 30% of acute myeloid leukemia cases.

同时，Syndax正在进行更多的研究，这些研究可能支持扩大Revuforj作为以KMT2A易位为特征的白血病以及由mNPM1基因突变驱动的白血病的早期治疗方案，mNPM1基因突变在约30%的急性髓性白血病病例中被发现。

Last week, Syndax reported positive preliminary Phase 2 data in acute myeloid leukemia driven by mNPM1. In the first half of 2025, the company it plans to publish and present these results at a medical conference and seek FDA approval in this indication..

上周，Syndax报告了由mNPM1驱动的急性骨髓性白血病的阳性初步2期数据。2025年上半年，该公司计划在医学会议上公布并展示这些结果，并在该适应症中寻求FDA的批准。。

Syndax has priced Revuforj at $39,500 a month, which works out to $474,000 annually before any rebates or discounts. Dosing of Revuforj is according to a patient’s weight. The company said it expects the 110 mg and 160 mg tablets of the drug will become available later this month through specialty distributors and specialty pharmacies.

Syndax将Revuforj定价为每月39500美元，在任何回扣或折扣之前，每年的价格为474000美元。Revuforj的剂量取决于患者的体重。该公司表示，预计该药的110毫克和160毫克片剂将于本月晚些时候通过专业分销商和专业药店上市。

The lowest dose, 25 mg, is for patients who weigh less than 40 kg (about 88 pounds). Syndax expects this dose will become commercially available next year, either late in the first quarter or early in the second quarter. Until then, Syndax will supply an oral solution of this dose via an expanded access program..

最低剂量为25毫克，适用于体重小于40公斤（约88磅）的患者。Syndax预计，这种剂量将于明年上市，要么在第一季度末，要么在第二季度初。在此之前，Syndax将通过扩展访问程序提供该剂量的口服溶液。。

Photo: Getty Images, Sarah Silbiger

照片：盖蒂图片社，莎拉·西尔比格

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