SAN FRANCISCO, Dec. 15, 2025 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) today announced that the Office of Orphan Products Development of the U.S. Food and Drug Administration (FDA) has granted roxadustat Orphan Drug Designation for the treatment of myelodysplastic syndromes (MDS).

旧金山，2025年12月15日（环球新闻社）—— FibroGen公司（纳斯达克股票代码：FGEN）今日宣布，美国食品药品监督管理局（FDA）孤儿产品开发办公室已授予罗沙司他（roxadustat）用于治疗骨髓增生异常综合征（MDS）的孤儿药资格。

“The Orphan Drug Designation granted to roxadustat for MDS underscores the significant treatment gap in this indication, and highlights patients’ need for additional convenient treatments that can provide durable response,” said Thane Wettig, Chief Executive Officer of FibroGen. “Roxadustat showed an improvement in transfusion-independence in a subset of patients with high transfusion burden in a post-hoc analysis from the Phase 3 MATTHERHORN trial, which along with its favorable tolerability profile and oral route of administration has the ability to set it apart from current second-line treatments.

“罗沙司他针对MDS获得的孤儿药资格认证突显了该适应症中存在的显著治疗空白，并强调了患者对于额外便捷且能提供持久疗效的治疗方案的需求，”FibroGen首席执行官Thane Wettig表示。“在3期MATTHERHORN试验的事后分析中，罗沙司他在一部分高输血负担患者中显示出改善输血独立性的效果，加之其良好的耐受性特征和口服给药方式，使其有潜力区别于当前的二线治疗方案。”

Our team is finalizing the Phase 3 protocol in this patient population for submission to the FDA in the fourth quarter of 2025.”.

我们的团队正在最终确定这一患者群体的第三阶段方案，计划于2025年第四季度提交给FDA。

There are approximately 58,000 patients diagnosed with LR-MDS in the U.S. with 85% of them suffering from anemia. Anemia in patients with MDS is associated with increased risk of cardiovascular complications and the need for blood transfusions. Transfusion-dependent patients suffer higher rates of complications and decreased quality of life.

美国大约有58,000名患者被诊断为低危骨髓增生异常综合征（LR-MDS），其中85%的患者患有贫血。MDS患者的贫血与心血管并发症风险增加以及输血需求相关。依赖输血的患者并发症发生率更高，生活质量下降。

Current first-line treatments lead to transfusion independence in less than 50% of patients and relief is often temporary with limited options for second line and beyond treatments. In a post-hoc analysis from the Phase 3 MATTERHORN trial, roxadustat demonstrated transfusion independence benefits compared to placebo in patients with high transfusion burden..

当前的一线治疗使不到50%的患者脱离输血依赖，且缓解通常是暂时的，二线及后续治疗选择有限。在对3期MATTERHORN试验进行的事后分析中，罗沙司他相比安慰剂，在高输血负担的患者中显示出脱离输血依赖的益处。

The FDA Orphan Drug Designation is granted to drugs intended for the treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Benefits of the designation may include exemption from certain FDA fees, financial incentives for qualified clinical development, and seven years of market exclusivity in the U.S.

FDA孤儿药资格授予用于治疗、诊断或预防美国境内少于20万人的罕见疾病的药物。该资格的好处可能包括免除某些FDA费用、合格临床开发的财政激励以及在美国七年的市场独占权。

following drug approval..

药品批准后...

About Myelodysplastic Syndromes Anemia

关于骨髓增生异常综合征贫血

Myelodysplastic syndromes (MDS) are a group of disorders characterized by dysfunctional progenitor blood cells and stem cells, resulting in chronic anemia in most patients. Annual incidence rates of MDS are estimated to be 4.9/100,000 adults in the U.S, of which 77% are considered lower-risk MDS. Approximately 80% of patients with MDS have anemia at the time of diagnosis, and around 60% of patients with MDS will experience severe anemia (hemoglobin <8 g/dL) at some point during the course of their disease.

骨髓增生异常综合征（MDS）是一组以造血祖细胞和干细胞功能异常为特征的疾病，大多数患者因此患有慢性贫血。据估计，美国每年每10万成年人中MDS的发病率为4.9例，其中77%被认为是低危MDS。大约80%的MDS患者在诊断时即患有贫血，约60%的MDS患者在病程中的某个阶段会出现严重贫血（血红蛋白<8 g/dL）。

Anemia in patients with MDS is associated with increased risk of cardiovascular complications and the need for blood transfusion. Approximately 50% of patients with MDS require regular red blood cell transfusions. Transfusion-dependent MDS patients suffer higher rates of cardiac events, infections, and iron overload with the related complications.

骨髓增生异常综合征（MDS）患者的贫血与心血管并发症风险增加以及输血需求相关。大约50%的MDS患者需要定期进行红细胞输注。依赖输血的MDS患者发生心脏事件、感染和铁过载及相关并发症的风险更高。

In addition, anemia frequently leads to significant fatigue, cognitive dysfunction, and decreased quality of life. Today, patients are routinely treated with erythropoiesis-stimulating agents (ESAs), luspatercept, imetelstat, or lenalidomide in lower-risk MDS with isolated del(5q), and hypomethylating agents (HMAs) in higher-risk disease.

此外，贫血常导致显著的疲劳、认知功能障碍和生活质量下降。目前，低危MDS伴孤立del(5q)的患者通常接受促红细胞生成剂（ESAs）、鲁沙肽、伊美司他或来那度胺治疗，而高危疾病则常用低甲基化剂（HMAs）进行治疗。

Only 35-40% of patients respond to current treatments and the durability of response is short. Moreover, these treatments are challenging to dose-calibrate and can only be administered via subcutaneous injection or through IV infusion. There remains a high unmet need for the treatment of anemia associated with MDS, and new strategies that provide durable response and the convenience of oral administration are highly desired in managing patients with MDS..

目前只有35-40%的患者对现有治疗有反应，且反应持续时间较短。此外，这些治疗方案难以进行剂量调整，只能通过皮下注射或静脉输注给药。对于与骨髓增生异常综合征（MDS）相关的贫血治疗，仍然存在高度未满足的需求，而能够提供持久反应且方便口服的新策略在管理MDS患者时备受期待。

About Roxadustat

关于罗沙司他

Roxadustat, an oral medication, is the first in a new class of medicines comprising HIF-PH inhibitors that promote erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin, improved iron absorption and mobilization, and downregulation of hepcidin.

罗沙司他是一种口服药物，属于新型缺氧诱导因子脯氨酰羟化酶抑制剂类药物，通过增加内源性促红细胞生成素的产生、改善铁的吸收和动员以及下调铁调素来促进红细胞的生成。

Roxadustat is approved in Europe, Japan, and numerous other countries for the treatment of anemia of CKD in adult patients on dialysis (DD) and not on dialysis (NDD). FibroGen has the sole rights to roxadustat in the United States, Canada, Mexico, and in all markets not held by AstraZeneca or licensed to Astellas.

Roxadustat 已在欧洲、日本及众多其他国家获得批准，用于治疗接受透析（DD）和未接受透析（NDD）的成年慢性肾病贫血患者。FibroGen 在美国、加拿大、墨西哥以及所有未由阿斯利康持有或授权给安斯泰来的市场中拥有 roxadustat 的独家权利。

Astellas and FibroGen are collaborating on the commercialization of roxadustat for the treatment of anemia in territories including Japan, Europe, Turkey, Russia, and the Commonwealth of Independent States, the Middle East, and South Africa..

安斯泰来和FibroGen正在合作，在包括日本、欧洲、土耳其、俄罗斯、独联体、中东和南非在内的地区将罗沙司他商业化，用于治疗贫血。

About FibroGen

关于 FibroGen

FibroGen, Inc. is a biopharmaceutical company focused on development of novel therapies at the frontiers of cancer biology and anemia. Roxadustat (爱瑞卓

FibroGen, Inc. 是一家生物制药公司，专注于癌症生物学和贫血领域的前沿新疗法的开发。罗沙司他（爱瑞卓）

®

®

, EVRENZO

，EVRENZO

TM

商标符号 (™)

) is currently approved in Europe, Japan, and numerous other countries for the treatment of anemia in chronic kidney disease (CKD) patients on dialysis and not on dialysis. The Company continues to evaluate the development plan for the Phase 3 trial of roxadustat in anemia associated with lower-risk myelodysplastic syndrome (LR-MDS) in the U.S.

）目前在欧洲、日本和许多其他国家获批用于治疗接受透析和未接受透析的慢性肾病（CKD）患者的贫血。该公司继续评估罗沙司他针对较低风险骨髓增生异常综合征（LR-MDS）相关贫血在美国进行的3期试验的开发计划。

FG-3246 (also known as FOR46), a first-in-class antibody-drug conjugate (ADC) targeting CD46, is in Phase 2 development for the treatment of metastatic castration-resistant prostate cancer. This program also includes the development of FG-3180, an associated CD46-targeted PET biomarker. For more information, please visit www.fibrogen.com.

FG-3246（也称为FOR46）是一种首创的靶向CD46的抗体药物偶联物（ADC），目前处于治疗转移性去势抵抗性前列腺癌的二期临床开发阶段。该项目还包括FG-3180的开发，这是一种相关的CD46靶向PET生物标志物。欲了解更多信息，请访问www.fibrogen.com。

.

。

Forward-Looking Statements

前瞻性声明

This release contains forward-looking statements regarding FibroGen’s strategy, future plans and prospects, including statements regarding its commercial products and clinical programs and those of it and its collaboration partners Fortis and UCSF. These forward-looking statements include, but are not limited to, statements regarding the efficacy, safety, convenience, and potential clinical or commercial success of FibroGen products and product candidates, statements about regulatory interactions, and statements about FibroGen’s plans and objectives.

本发布包含关于FibroGen战略、未来计划和前景的前瞻性声明，包括有关其商业产品和临床项目以及其与合作方Fortis和UCSF的项目声明。这些前瞻性声明包括但不限于关于FibroGen产品及其候选产品的功效、安全性、便利性及潜在临床或商业成功的声明，关于监管互动的声明，以及关于FibroGen计划和目标的声明。

These forward-looking statements are typically identified by use of terms such as “may,” “will”, “should,” “on track,” “could,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “predict,” “potential,” “continue” and similar words, although some forward-looking statements are expressed differently.

这些前瞻性陈述通常通过使用诸如“可能”、“将”、“应该”、“按计划”、“可能”、“预期”、“计划”、“预期”、“相信”、“估计”、“预测”、“潜在”、“继续”等术语以及类似的词语来识别，尽管有些前瞻性陈述的表述方式有所不同。

FibroGen’s actual results may differ materially from those indicated in these forward-looking statements due to risks and uncertainties related to the continued progress and timing of its various programs, including the enrollment and results from ongoing and potential future clinical trials, and other matters that are described in FibroGen’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q, each as filed with the Securities and Exchange Commission (SEC), including the risk factors set forth therein.

由于与 FibroGen 各项计划的持续进展和时间安排相关的风险和不确定性，包括正在进行和未来潜在临床试验的入组和结果，以及 FibroGen 最近提交给美国证券交易委员会 (SEC) 的年度报告（Form 10-K）和季度报告（Form 10-Q）中描述的其他事项，特别是其中列出的风险因素，FibroGen 的实际结果可能与这些前瞻性声明中表明的内容有重大差异。

Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release, and FibroGen undertakes no obligation to update any forward-looking statement in this press release, except as required by law. .

投资者应注意不要对这些前瞻性声明施加过度依赖，这些声明仅截至本发布日期有效，除非法律要求，FibroGen不承担更新本新闻稿中任何前瞻性声明的义务。

Source: FibroGen, Inc.

来源：FibroGen公司。

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