欧洲药品管理局人用医药产品委员会（CHMP）建议欧盟批准Partner Therapeutics公司的IMREPLYS®（沙格司亭，重组人粒细胞-巨噬细胞集落刺激因子）用于治疗暴露于骨髓抑制剂量辐射（急性放射综合征的造血亚综合征）-动脉网

European Medicines Agency CHMP Recommends EU Approval of Partner Therapeutics IMREPLYS® (sargramostim, rhu GM-CSF) to Treat Exposure to Myelosuppressive Doses of Radiation (Haematopoietic Sub-syndrome of Acute Radiation Syndrome)

CISION 等信源发布 2025-06-20 22:03

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LEXINGTON, Mass.

马萨诸塞州列克星敦

，

June 20, 2025

2025年6月20日

/PRNewswire/ -- Partner Therapeutics, Inc. (PTx) today announced that the

/PRNewswire/ -- Partner Therapeutics, Inc. (PTx) 今天宣布

Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of PTx's Marketing Authorisation Application (MAA) for IMREPLYS

欧洲药品管理局（EMA）的人用药品委员会（CHMP）已建议批准PTx公司关于IMREPLYS的上市许可申请（MAA）。

(sargramostim, rhu GM-CSF), the same formulation that was approved by the U.S. Food and Drug Administration (FDA) under the brand name LEUKINE

（沙格司亭，重组人粒细胞-巨噬细胞集落刺激因子），与美国食品药品监督管理局（FDA）批准的同配方药物，品牌名为LEUKINE。

in 2018. IMPREPLYS would be indicated for treatment of patients of all ages acutely exposed to myelosuppressive doses of radiation with Haematopoietic Sub-syndrome of Acute Radiation Syndrome (H-ARS). Approval by the European Commission (EC) is expected within 67 days of the CHMP recommendation. EC approval will enable sales, including government procurement for stockpiling, of IMREPLYS in all European Union (EU) member states, as well as .

2018年，IMPREPLYS将被用于治疗所有年龄阶段急性暴露于骨髓抑制剂量辐射并患有急性放射综合症的造血亚综合征（H-ARS）的患者。欧洲委员会（EC）预计将在CHMP建议后的67天内批准。EC的批准将使IMREPLYS能够在所有欧盟（EU）成员国销售，包括政府为储备进行的采购。

Norway

挪威

，

Iceland

冰岛

, and

，以及

Liechtenstein

列支敦士登

。

'We are grateful to the EMA for their support leading to this approval recommendation for IMREPLYS, which will enable access to the product throughout

“我们感谢欧洲药品管理局（EMA）的支持，使得IMREPLYS获得了这一批准建议，这将使整个地区都能获得该产品。

Europe

欧洲

for treatment of citizens for radiation injuries,' said

用于治疗市民的辐射伤害，”

John L. McManus

约翰·L·麦克马纳斯

, President, Health Security and Critical Care of PTx. 'Threats of use of tactical nuclear weapons underscore the importance of preparedness. The likelihood that initial medical treatment will occur in a severe, limited-resource environment complicates response planning and requires therapies that can be effective with minimal to no supportive care.

，PTx健康安全与重症监护总裁。“使用战术核武器的威胁凸显了准备的重要性。初次医疗救治很可能发生在资源严重受限的环境中，这使应对计划复杂化，并且需要在极少或无支持性护理的情况下仍能有效的治疗方法。

Preparedness through stockpiling of such therapeutic solutions is intended to help reduce mortality if an event occurs that results in exposure to acute or sudden high-dose radiation. The approval recommendation from the EMA is an important step in PTx's international strategy for IMREPLYS to be available in the European market and beyond.'.

通过储备此类治疗方案来做好准备，旨在如果发生导致暴露于急性或突然高剂量辐射的事件时帮助降低死亡率。欧洲药品管理局（EMA）的批准建议是PTx国际战略的重要一步，有助于IMREPLYS进入欧洲市场及其他地区。

ABOUT IMREPLYS / LEUKINE (sargramostim)

关于 IMREPLYS / LEUKINE（沙格司亭）

Sargramostim is a human granulocyte-macrophage colony-stimulating growth factor (GM-CSF) produced by recombinant DNA technology in a yeast (S. cerevisiae) expression system. The binding to GM-CSF receptors expressed on the surface of target cells (haematopoietic progenitors and mature immune cells), initiates an intracellular signalling cascade which induces the cellular responses (i.e., division, maturation, activation).

沙格司亭是一种通过重组DNA技术在酵母（酿酒酵母）表达系统中产生的粒细胞-巨噬细胞集落刺激生长因子（GM-CSF）。它与靶细胞（造血祖细胞和成熟的免疫细胞）表面表达的GM-CSF受体结合后，会启动细胞内信号级联反应，从而诱导细胞反应（如分裂、成熟、激活）。

GM-CSF is a multilineage factor and, in addition to dose-dependent effects on the myelomonocytic lineage, it can promote the proliferation and maturation of megakaryocytic and erythroid progenitors. Sargramostim (LEUKINE) was approved by the US Food and Drug Administration in 2018 to increase survival in adult and pediatric patients from birth to 17 years of age acutely exposed to myelosuppressive doses of radiation (Hematopoietic Syndrome of Acute Radiation Syndrome [H-ARS]). .

GM-CSF是一种多谱系因子，除了对髓单核细胞谱系有剂量依赖性效应外，它还可以促进巨核细胞和红系祖细胞的增殖和成熟。沙格司亭（LEUKINE）于2018年获得美国食品药品监督管理局批准，用于提高从出生到17岁的成人和儿童患者在急性暴露于骨髓抑制剂量辐射（急性放射综合症的造血综合征[H-ARS]）后的生存率。

The product is commercially available in

该产品已上市销售

the United States

美国

under the brand name LEUKINE. LEUKINE will continue to be accessible through a named patient program operated by Tanner Pharma Group outside of

品牌名称为LEUKINE。 LEUKINE将继续通过Tanner Pharma集团运营的指定患者计划在范围之外获得。

the United States

美国

, including in the European Union for named patient use outside of the H-ARS indication.

，包括在欧洲联盟用于H-ARS适应症之外的命名患者使用。

In the European Union,

在欧盟，

Iceland

冰岛

，

Norway

挪威

, and

，以及

Liechtenstein

列支敦士登

, upon approval by the EC, IMPREPLYS would be indicated:

经EC批准后，IMPREPLYS将被指示：

For treatment of patients of all ages acutely exposed to myelosuppressive doses of radiation with Haematopoietic Sub-syndrome of Acute Radiation Syndrome (H-ARS).

用于治疗所有年龄段急性暴露于骨髓抑制剂量辐射且伴有急性放射综合征造血亚综合征（H-ARS）的患者。

在

the United States

美国

, LEUKINE is indicated:

，LEUKINE的适应症包括：

To shorten time to neutrophil recovery and to reduce the incidence of severe and life-threatening infections and infections resulting in death following induction chemotherapy in adult patients 55 years and older with acute myeloid leukemia (AML).

缩短中性粒细胞恢复时间，减少55岁及以上急性髓系白血病（AML）成人患者在诱导化疗后严重和危及生命的感染以及导致死亡的感染的发生率。

For the mobilization of hematopoietic progenitor cells into peripheral blood for collection by leukapheresis and autologous transplantation in adult patients.

用于动员造血祖细胞进入外周血，以便通过白细胞分离术采集并用于成年患者的自体移植。

For the acceleration of myeloid reconstitution following autologous bone marrow or peripheral blood progenitor cell transplantation in adult and pediatric patients 2 years of age and older.

用于加速成人和2岁及以上儿童患者自体骨髓或外周血祖细胞移植后的髓系重建。

For the acceleration of myeloid reconstitution following allogeneic bone marrow transplantation in adult and pediatric patients 2 years of age and older.

用于加速2岁及以上儿童和成年患者在同种异体骨髓移植后的髓系重建。

For treatment of delayed neutrophil recovery or graft failure after autologous or allogeneic bone marrow transplantation in adult and pediatric patients 2 years of age and older.

用于治疗2岁及以上成人和儿童患者自体或异体骨髓移植后中性粒细胞恢复延迟或移植物失败。

To increase survival in adult and pediatric patients from birth to 17 years of age acutely exposed to myelosuppressive doses of radiation (Hematopoietic Syndrome of Acute Radiation Syndrome [H-ARS]).

为了提高从出生到17岁的成人和儿童患者在急性暴露于骨髓抑制剂量的辐射（急性放射综合症的造血系统综合症[H-ARS]）后的生存率。

Important Safety Information for LEUKINE (sargramostim)

LEUKINE（沙格司亭）的重要安全信息

Contraindications

禁忌症

Do not administer LEUKINE to patients with a history of serious allergic reaction, including anaphylaxis, to human granulocyte-macrophage colony-stimulating factor, sargramostim, yeast-derived products, or any other component of LEUKINE.

不要对有过敏反应史的患者使用LEUKINE，包括对人类粒细胞-巨噬细胞集落刺激因子、沙格司亭、酵母衍生产品或LEUKINE任何其他成分产生过的严重过敏反应或过敏性休克。

Warnings and Precautions

警告和注意事项

Serious hypersensitivity reactions, including anaphylactic reactions, have been reported with LEUKINE. If a serious allergic or anaphylactic reaction occurs, immediately discontinue LEUKINE therapy, and institute medical management. Discontinue LEUKINE permanently for patients with serious allergic reactions..

已有报告显示LEUKINE可引起严重的超敏反应，包括过敏性反应。如果发生严重的过敏或过敏性反应，立即停止LEUKINE治疗，并进行医学处理。对于出现严重过敏反应的患者，永久停止使用LEUKINE。

LEUKINE can cause infusion-related reactions that may be characterized by respiratory distress, hypoxia, flushing, hypotension, syncope, and/or tachycardia. Observe closely during infusion, particularly in patients with preexisting lung disease; dose adjustment or discontinuation may be needed.

LEUKINE可能引起输注相关反应，其特征可能表现为呼吸困难、低氧血症、潮红、低血压、晕厥和/或心动过速。在输注期间应密切观察，特别是对于已有肺部疾病的患者；可能需要调整剂量或停止用药。

LEUKINE should not be administered simultaneously with or within 24 hours preceding cytotoxic chemotherapy or radiotherapy or within 24 hours following chemotherapy.

LEUKINE 不应与细胞毒性化疗或放疗同时使用，也不应在化疗或放疗前 24 小时内或化疗后 24 小时内使用。

Edema, capillary leak syndrome, and pleural or pericardial effusions have been reported in patients after LEUKINE administration. LEUKINE should be used with caution in patients with preexisting fluid retention, pulmonary infiltrates, or congestive heart failure. Such patients should be monitored.

已有报告称，患者在使用LEUKINE后出现了水肿、毛细血管渗漏综合征以及胸膜或心包积液。对于存在先前液体潴留、肺部浸润或充血性心力衰竭的患者，应谨慎使用LEUKINE。应对这些患者进行监测。

Supraventricular arrhythmia has been reported in uncontrolled studies during LEUKINE administration, particularly in patients with a history of cardiac arrhythmia. Use LEUKINE with caution in patients with preexisting cardiac disease.

在使用LEUKINE治疗期间，未受控制的研究中已有报告出现室上性心律失常，尤其是有心律失常病史的患者。对于已有心脏疾病的患者，应谨慎使用LEUKINE。

If absolute neutrophil count (ANC) > 20,000 cells/mm3 or if white blood cell (WBC) counts > 50,000/mm3, LEUKINE administration should be interrupted, or the dose reduced by half. Monitor complete blood counts (CBC) with differential twice per week.

如果绝对中性粒细胞计数 (ANC) > 20,000 cells/mm3 或白细胞计数 (WBC) > 50,000/mm3，应中断LEUKINE的使用或剂量减半。每周两次监测全血细胞计数 (CBC) 及其分类。

Discontinue LEUKINE therapy if tumor progression, particularly in myeloid malignancies, is detected during LEUKINE treatment.

如果在LEUKINE治疗期间检测到肿瘤进展，特别是髓系恶性肿瘤，应停止LEUKINE治疗。

Treatment with LEUKINE may induce neutralizing anti-drug antibodies. Use LEUKINE for the shortest duration needed.

使用LEUKINE治疗可能会诱导产生中和抗药抗体。请在最短时间内使用LEUKINE。

Avoid administration of solutions containing benzyl alcohol (including LEUKINE for injection reconstituted with Bacteriostatic Water for Injection, USP [0.9 % benzyl alcohol]) to neonates and low birth weight infants.

避免给新生儿和低出生体重婴儿使用含苯甲醇的溶液（包括用含0.9%苯甲醇的注射用抑菌水复溶的LEUKINE注射剂）。

Drug Interactions

药物相互作用

Avoid the concomitant use of LEUKINE and products that induce myeloproliferation. Monitor for clinical and laboratory signs of excess myeloproliferative effects.

避免同时使用LEUKINE和诱导骨髓增殖的产品。监测骨髓增殖过度效应的临床和实验室体征。

Adverse Reactions

不良反应

Adverse events occurring in >10% of patients receiving LEUKINE in controlled clinical trials and reported at a higher frequency than in placebo patients are:

在控制性临床试验中，接受LEUKINE治疗的患者中发生率超过10%且比安慰剂组报告频率更高的不良事件包括：

In recipients of autologous bone marrow transplantation (BMT)–asthenia, malaise, diarrhea, rash, peripheral edema, urinary tract disorder

自体骨髓移植（BMT）接受者 - 乏力、不适、腹泻、皮疹、外周水肿、泌尿系统疾病

In recipients of allogeneic BMT–abdominal pain, chills, chest pain, diarrhea, nausea, vomiting, hematemesis, dysphagia, GI hemorrhage, pruritus, bone pain, arthralgia, eye hemorrhage, hypertension, tachycardia, bilirubinemia, hyperglycemia, increased creatinine, hypomagnesemia, edema, pharyngitis, epistaxis, dyspnea, insomnia, anxiety, high glucose, low albumin.

异基因骨髓移植受者-腹痛、寒战、胸痛、腹泻、恶心、呕吐、呕血、吞咽困难、胃肠道出血、瘙痒、骨痛、关节痛、眼出血、高血压、心动过速、胆红素血症、高血糖、肌酐升高、低镁血症、水肿、咽炎、鼻衄、呼吸困难、失眠、焦虑、高血糖、低白蛋白。

In patients with AML–fever, weight loss, nausea, vomiting, anorexia, skin reactions, metabolic laboratory abnormalities, edema

在AML患者中出现发热、体重减轻、恶心、呕吐、厌食、皮肤反应、代谢实验室异常、水肿等症状

Please see full Prescribing Information for LEUKINE at

请参阅LEUKINE的完整处方信息 tại

www.leukine.com

。

ABOUT PARTNER THERAPEUTICS

关于合作伙伴治疗学

Partner Therapeutics, Inc. (PTx), an integrated biotechnology company, focuses on development and commercialization of late-stage therapeutics to improve health outcomes in treatment of cancer and other serious diseases. The company believes in delivering products and supporting medical teams with the purpose of achieving superior outcomes for patients and their families.

Partner Therapeutics, Inc. (PTx)是一家综合生物技术公司，专注于开发和商业化晚期治疗药物，以改善癌症和其他严重疾病的治疗效果。公司致力于提供产品并支持医疗团队，旨在为患者及其家属实现更优的治疗结果。

Visit .

访问。

www.partnertx.com

。

Reference

参考文献

LEUKINE (sargramostim) injection - Full Prescribing Information available at:

LEUKINE（沙格司亭）注射液 - 完整处方信息可在此处获取：

https://www.leukine.com/prescribing-information.pdf

PARTNER THERAPEUTICS

合作伙伴治疗学