泼尼松(Prednisone) - 药物靶点：GR_在研适应症：自身免疫性疾病，非转移性前列腺癌，转移性去势抵抗性前列腺癌_专利_临床

概要

基本信息

简介泼尼松（Prednisone）是一种用于治疗多种炎症性疾病，如过敏、关节炎和哮喘的药物。它属于类固醇类药物，通过抑制免疫系统和减轻炎症发挥作用。泼尼松通常口服，可用于短期或长期治疗。像所有药物一样，泼尼松可能会引起副作用，如体重增加、情绪变化和感染风险增加。与医疗保健提供者合作，确定泼尼松是否是管理您特定疾病的安全有效选择是非常重要的。

药物类型

小分子化药

别名

1,2-Dehydrocortisone、1,4-Pregnadiene-17α,21-diol-3,11,20-trione、17,21-Dihydroxypregna-1,4-diene-3,11,20-trione

+ [45]

靶点

GR

作用方式

拮抗剂

作用机制

GR拮抗剂(糖皮质激素受体拮抗剂)

治疗领域

免疫系统疾病皮肤和肌肉骨骼疾病肿瘤+ [5]

在研适应症

自身免疫性疾病非转移性前列腺癌转移性去势抵抗性前列腺癌+ [8]

非在研适应症

急性白血病前列腺腺癌先天性肾上腺增生+ [67]

原研机构

Merck Sharp & Dohme Corp.

在研机构

Janssen Research & Development LLC

Merck Sharp & Dohme Corp.

Takeda Pharmaceutical Co., Ltd.

+ [4]

非在研机构

Schering Corp.Merck KGaA

Amgen, Inc.

+ [9]

权益机构-

最高研发阶段批准上市

首次获批日期

美国 (1955-02-21),

炎症

最高研发阶段(中国)批准上市

特殊审评孤儿药 (美国)、优先审评 (中国)

登录后查看时间轴

结构/序列

分子式C21H26O5

InChIKeyXOFYZVNMUHMLCC-ZPOLXVRWSA-N

CAS号53-03-2

关联

1,813

项与泼尼松相关的临床试验

NCT06934915

/ Not yet recruiting早期临床1期IIT

Randomized, Double-Blind, Placebo-Controlled, Parallel-Groups Trial of Prednisone in Adults With an Immune-Mediated Subtype of Autism Spectrum Disorder

The goal of this clinical trial is to learn how prednisone affects adults with autism spectrum disorder (ASD). It will also learn about the safety of prednisone. The main questions it aims to answer are:
* How does prednisone affect the core features and associated target symptoms of ASD in adults with an immune-mediated subtype of ASD?
* Is prednisone safe for autistic adults without causing too many side effects?
* Does this study warrant larger trials studying anti-inflammatory drugs in this subject population?
Researchers will compare the drug prednisone to a placebo (a look-alike substance that contains no drug) to see how prednisone affects autistic adult males.
Participants will:
* Visit the clinic 2 times for a screening and baseline visit.
* Take prednisone or a placebo every day for 16 weeks.
* Visit the clinic 2 times for checkups, tests, questionnaires, and dose changes, and 1 time for a follow-up visit 4 weeks after stopping the study drug.
* Provide blood and urine samples for testing up to 4 times.
* Complete 8 remote calls every 1-2 weeks for checkups and dose changes.
* Keep a diary of the dose and times they take the study drug every day and any symptoms or side effects they experience.

开始日期2026-11-01

申办/合作机构

The Massachusetts General Hospital

NCT07654426

/ Not yet recruiting临床2期IIT

A Randomized Study of Dexrazoxane or Liposomal Doxorubicin in Newly Diagnosed Diffuse Large B-cell Lymphoma at High Risk for Heart Failure Events: the CARDIOPROTECT Trial

This trial is to evaluate if dexrazoxane is safer and more effective than liposomal doxorubicin in preventing heart failure events in participants with diffuse large B-cell lymphoma (DLBCL) undergoing either standard of care R-CHOP or pola-R-CHP treatment regiments.
The names of the study drugs involved in this study are:
* Dexrazoxane (a type of Topoisomerase II Inhibitor)
* Liposomal Doxorubicin (a type of Topoisomerase II Inhibitor)
* Standard of care R-CHOP treatment regimen (Cyclophosphamide, doxorubicin, vincristine, prednisone, rituximab)
* Standard of care pola-R-CHP treatment regimen: Cyclophosphamide, doxorubicin, polatuzumab vedotin-piiq, prednisone, rituximab

开始日期2026-10-02

申办/合作机构

Beth Israel Deaconess Medical Center, Inc.

NCT07443436

/ Not yet recruiting临床2期IIT

Immunomodulatory Treatment of Interstitial Lung Disease Associated With Surfactant Related Gene Variants

Scientific justification : Variants in surfactant-related genes (SRG) explain approximately 6% of familial pulmonary fibrosis (FPF).
The pathophysiology is unknown and seems to involve endoplasmic reticulum stress in type 2 alveolar epithelial cells.
Variable improvement in the prognosis of childhood and adult interstitial lung disease (ILD) associated with a variant of a SRG, initially reported to be lethal within months of diagnosis, has been observed since the consensual use of prednisone, azithromycin and hydroxychloroquine targeting endoplasmic reticulum stress, without demonstration of the efficacy of any of these treatments alone or in combination.
The investigators hypothesize that a treatment combining prednisone, azithromycin and hydroxychloroquine is safe and could improve the prognosis of adult patients with ILD associated with SRG variant.
Main objective and primary endpoint : Main objective:
Evaluate the efficacy of triple immunomodulatory therapy (prednisone, azithromycin and hydroxychloroquine) for 12 months in patients with ILD associated with a variant of a surfactant-related gene.
Primary endpoint:
Difference in forced vital capacity decline between the 2 groups at one year.
Secondary objectives and endpoints : Secondary objectives:
1. tolerance of the triple therapy,
2. correlation between the respiratory, radiological and clinical functional response,
3. quality of life of the patients,
4. overall survival, transplant-free survival, exacerbation free-survival, hospitalization-free survival
Secondary endpoints:
1. Clinical and biological tolerance (occurrence of an adverse effect during treatment), ECG (at 3, 6, 9, 12 months after randomization) (only HCQ or AZI patients) and ophthalmological (at one year after randomization)
2. Thoracic CT scan and PFT at 6 months and one year after randomization
3. Quality of life questionnaire (EORTC QLQ-C30, v3.0) at 3 months, 6 months, 9 months and one year after randomization,
4. Collection of vital status, lung transplantation, hospitalization for pulmonary and non-pulmonary causes and episodes of exacerbation at each visit until the end of follow-up 12 months after randomization.
Design of the study : Multicenter, randomized, controlled, two-arm, parallel, open-label superiority study comparing triple immunomodulatory therapy (prednisone, azithromycin, and hydroxychloroquine) to standard of care
Category : Category 2
Population of study participants: Patients aged over 18 years with ILD and SRG variant
Number of participants included : 30
Design of the study : Multicenter, randomized, controlled, two-arm, parallel, open-label superiority study comparing triple immunomodulatory therapy (prednisone, azithromycin, and hydroxychloroquine) to standard of care.

开始日期2026-10-01

申办/合作机构

Assistance Publique des Hôpitaux de Paris SA

100 项与泼尼松相关的临床结果

登录后查看更多信息

100 项与泼尼松相关的转化医学

登录后查看更多信息

100 项与泼尼松相关的专利（医药）

登录后查看更多信息

41,275

项与泼尼松相关的文献（医药）

2026-12-31·JOURNAL OF DERMATOLOGICAL TREATMENT

Does ANA positivity affect treatment outcomes in vitiligo? A clinical evaluation of 308-nm excimer light therapy

Article

作者: Jia, Dechao ; Zeng, Wen ; Zhang, Ke ; Zhang, Wei ; Yang, Yuling ; Lu, Hongguang ; Lan, Yinghua ; Han, Ning ; Zhang, Jun ; Tan, Mengsi ; Wang, Yu ; Chen, Cheng ; Peng, Yu

OBJECTIVES:

Vitiligo is an autoimmune skin disorder characterized by melanocyte destruction and frequently associated with autoantibodies such as antinuclear antibodies (ANA). However, the clinical relevance of ANA positivity in relation to phototherapy response remains unclear. This study aimed to evaluate whether ANA positivity influences the efficacy and safety of 308-nm excimer light therapy in patients with vitiligo.

METHODS:

In this cohort study, 86 patients with vitiligo received 308-nm excimer light therapy combined with topical agents, with oral mini-pulse prednisone added for active disease when necessary. Patients were stratified by ANA status, and therapeutic response was evaluated using the Vitiligo Area Scoring Index and standardized photographs over 6 months.

RESULTS:

Of the 23 ANA-positive patients (26.7%), 19 (82.6%) had a titer of 1:100 and 4 (17.4%) had a titer of 1:320, with women comprising 73.9% of this group. ANA-positive lesions on the face and neck more frequently achieved moderate repigmentation (50-74%) but were less likely to reach excellent repigmentation (≥75%) compared with ANA-negative lesions. No significant differences were observed in cumulative treatment doses, adverse events, or the occurrence of new autoimmune conditions.

CONCLUSIONS:

In conclusion, this single-center cohort study suggests that ANA positivity does not significantly affect the efficacy or safety of 308-nm excimer light therapy in vitiligo, indicating that the impact of low-titer ANA may be limited.

2026-12-31·JOURNAL OF INVESTIGATIVE SURGERY

Laparoscopic Ultrasound-Guided Biopsy: A Safe and Effective Approach for Diagnosing Idiopathic Retroperitoneal Fibrosis

Article

作者: Li, Zhi-De ; Ma, Zhi-Gang ; Chen, Xiong ; Zhuo, Qian ; Meng, Yuan ; Li, Yu-Peng

BACKGROUND:

Idiopathic retroperitoneal fibrosis (IRPF) is a rare fibroinflammatory condition that is challenging to distinguish from retroperitoneal malignancies based solely on imaging. We evaluated laparoscopic ultrasound-guided biopsy for diagnosing IRPF.

METHODS:

We retrospectively analyzed 12 patients with suspected retroperitoneal fibrosis who underwent laparoscopic ultrasound-guided biopsy. The surgical technique involved systematic laparoscopic exploration, intraoperative ultrasound to delineate the mass and its relation to great vessels, and targeted biopsy.

RESULTS:

Preoperative imaging suggested retroperitoneal masses in all 12 patients but failed to provide a definitive diagnosis in any case. Laparoscopic biopsy was successfully performed in all patients without conversion to laparotomy. Pathological examination confirmed IRPF in 11 patients and retroperitoneal adenocarcinoma in one. The procedure was safe, with minimal blood loss (20.4 ± 14.8 mL) and a short operative time (68.3 ± 19.0 min). All IRPF patients were treated with a combination of prednisone, tamoxifen, and mycophenolate mofetil, leading to significant lesion regression on follow-up imaging (median 37.9 months).

CONCLUSIONS:

Laparoscopic ultrasound-guided biopsy is a safe and highly effective diagnostic procedure for IRPF. It provides definitive pathological diagnosis to guide appropriate therapy and should be considered when percutaneous biopsy is not feasible.

2026-12-31·Future Science OA

Fulminant disseminated strongyloidiasis mimicking inflammatory bowel disease: a case report

Article

作者: Nfikha, Zeineb ; Ismail, Samar ; Harbi, Raida ; Akkari, Imen ; Ben Jazia, Elhem ; Trabelsi, Oussama ; Mrabet, Soumaya ; Cherif, Emna

BACKGROUND:

Strongyloidiasis is a gastrointestinal parasitic infection caused by Strongyloides stercoralis (S. stercoralis). Its autoinfection capability allows the parasite to persist lifelong in the absence of treatment, exposing patients to the risk of hyperinfection and fatal dissemination in cases of immunosuppression.

RESULTS:

A 33-year-old man, presented in December 2024 with severe acute colitis. Endoscopy and histopathology findings were consistent with inflammatory bowel disease (IBD). The patient was treated with intravenous corticosteroids and antibiotics, then maintained on tapering oral corticosteroids. Three months later, while on 20 mg/day prednisone equivalent, he was readmitted for another episode of severe acute colitis. Stool parasitology, stool culture, tissue cytomegalovirus (CMV) polymerase chain reaction (PCR), and Clostridium difficile toxin assays were all negative. Intravenous corticosteroids were restarted with partial response. The patient subsequently developed Enterococcus faecium meningitis, ileal obstruction requiring surgical resection, and hypoxemic respiratory distress. Gastric biopsies revealed numerous Strongyloides larvae. Stool parasitology confirmed disseminated strongyloidiasis. The clinical course rapidly deteriorated to septic shock and multiorgan failure, causing death.

DISCUSSION:

This case highlights the potential diagnostic confusion between IBD and strongyloidiasis. It underscores the potentially fatal consequences of unrecognized chronic strongyloidiasis and supports systematic screening prior to immunosuppressive therapy in at-risk populations.

3,390

项与泼尼松相关的新闻（医药）

2026-07-11

·Biotech前瞻

全球首个B7-H3 ADCⅢ期OS阳性：翰森率先破局，赛道进入兑现期

点击蓝字关注我们本期看点在此前发布的安全阴影下的逆袭：首款B7H3 ADC凭疗效数据获FDA优先审评中，我们曾分析第一三共与默沙东联合开发的ifinatamab deruxtecan（I-DXd）进入FDA优先审评，以及B7-H3 ADC距离首款上市还有多远。不到三个月，竞争格局再次发生变化。 2026年7月10日，翰森制药宣布，自主研发的B7-H3 ADC——risvutatug rezetecan（Ris-Rez，HS-20093）在关键性Ⅲ期ARTEMIS-008研究中达到总生存期主要终点。这是全球首次有B7-H3 ADC在随机Ⅲ期研究中取得OS阳性结果。B7-H3 ADC由此不再只是早期研究中缓解率较高的新兴赛道，而开始进入生存获益、上市申报和全球商业化的兑现阶段。 01 首个Ⅲ期OS阳性，意义不止于翰森领先 ARTEMIS-008是一项在中国开展的随机、开放标签Ⅲ期研究，纳入一线含铂治疗后进展或复发的小细胞肺癌患者，比较Ris-Rez与拓扑替康。预设期中分析显示，Ris-Rez显著改善OS，PFS等关键次要终点也取得积极结果；安全性与既往研究一致，未发现新的安全信号。翰森将据此推进中国上市申报准备工作。目前，具体的风险比、中位OS、中位PFS、脑转移亚组以及间质性肺病发生情况尚未公布，因此还不能判断绝对生存获益幅度，也不适合与其他产品进行直接跨研究比较。但从证据等级看，这一结果已经完成了关键跨越。早期研究中，Ris-Rez在53例可评估小细胞肺癌患者中显示出较高缓解率：8.0 mg/kg和10.0 mg/kg剂量组ORR分别为61.3%和50.0%，中位PFS分别为5.9个月和7.3个月。ARTEMIS-008进一步证明，这些缓解信号能够转化为随机对照研究中的生存改善。这也是翰森此次阳性结果最重要的价值：验证的并非单一靶点，而是抗体、连接子、载荷、剂量选择和安全管理共同构成的完整ADC产品。 02 I-DXd抢占上市窗口，Ris-Rez率先完成生存验证当前最接近上市的B7-H3 ADC，仍是第一三共与默沙东联合开发的I-DXd。 2026年4月，美国FDA受理I-DXd用于经含铂治疗后进展的广泛期小细胞肺癌上市申请，并授予优先审评，PDUFA日期为2026年10月10日。该申请主要基于Ⅱ期IDeate-Lung01研究，而非Ⅲ期结果。在最新公布的137例12 mg/kg剂量患者中，I-DXd确认ORR为48.2%，中位缓解持续时间为5.3个月，中位PFS为4.9个月，中位OS为10.3个月。二线治疗亚组ORR为56.3%，中位OS为12.0个月。这也修正了早期文章中引用的54.8% ORR和11.8个月OS数据。前者来自较早期的剂量优化分析，随着扩展队列纳入，最终主要分析数据已更新为ORR 48.2%、中位OS 10.3个月。 I-DXd的主要风险仍是间质性肺病。在上述研究中，药物相关ILD/肺炎发生率为12.4%，包括4例3级事件和2例5级事件。因此，两款产品目前形成了不同的领先位置： I-DXd率先进入FDA审评，有望抢占首款获批的时间窗口；Ris-Rez则率先取得随机Ⅲ期OS阳性结果，证据等级暂时领先。不过，ARTEMIS-008主要基于中国患者。Ris-Rez能否在全球人群中重复这一结果，还需要GSK正在开展的全球Ⅲ期EMBOLD SCLC-301研究验证。GSK已获得大中华区以外的开发和商业化权益，该交易包括1.85亿美元首付款及最高15.25亿美元里程碑付款。 03 五款头部产品进入Ⅲ期，中国企业占据四席截至2026年7月，公开进入Ⅲ期开发的B7-H3 ADC头部产品主要有五款，其中四款源自中国企业。值得注意的是，YL201早于Ris-Rez公布了B7-H3 ADC的首个Ⅲ期阳性结果。其鼻咽癌TAISHAN-301研究在期中分析中达到确认ORR共同主要终点，显著优于化疗，但另一共同主要终点OS尚未成熟。因此，更准确的表述是： YL201是全球首个取得Ⅲ期疗效终点阳性结果的B7-H3 ADC；Ris-Rez是全球首个取得Ⅲ期OS阳性结果的B7-H3 ADC。 2026年1月，宜联生物将YL201在多个实体瘤中的开发和商业化权益授权给罗氏。YL201在中国已有小细胞肺癌和鼻咽癌两项注册性Ⅲ期研究。映恩生物与BioNTech的BNT324/DB-1311则选择前列腺癌作为首个Ⅲ期突破口。BNT324-03计划纳入736例患者，与多西他赛联合泼尼松或泼尼松龙进行比较，主要关注影像学PFS和OS。明慧医药的MHB088C已将大中华区权益授予齐鲁制药，目前正在推进二线小细胞肺癌和二线食管鳞癌两项Ⅲ期研究。从第一三共/默沙东，到翰森/GSK、宜联/罗氏、映恩/BioNTech和明慧/齐鲁，五款头部产品全部采用联合开发或授权合作模式。B7-H3 ADC已经成为中国创新药对外合作最集中的赛道之一。 04 总结与展望 ARTEMIS-008阳性后，B7-H3 ADC的竞争标准正在发生变化。首先是OS。小细胞肺癌进展快、患者异质性高，单臂研究中的高ORR并不必然转化为生存改善。Ris-Rez率先跨过Ⅲ期OS门槛，但完整数据仍需关注绝对生存延长、风险比和不同复发间隔亚组。其次是安全性。B7-H3 ADC普遍搭载拓扑异构酶Ⅰ抑制剂载荷，主要毒性包括中性粒细胞减少、贫血和血小板减少。不同产品之间真正能够形成差异的，可能不是短期ORR相差几个百分点，而是严重骨髓抑制、ILD、减量和停药比例。第三是全球重复性。中国拥有小细胞肺癌、鼻咽癌和食管鳞癌等疾病的患者资源和临床开发效率，但中国Ⅲ期阳性并不自动等同于全球注册成功。Ris-Rez、YL201和MHB088C最终仍需要更多国际多中心证据。第四是生物标志物。B7-H3在多种实体瘤中广泛表达，但现有早期研究尚未建立稳定的表达水平与疗效相关性。这一特点扩大了潜在治疗人群，也意味着不同产品可能难以通过简单的IHC阈值完成患者筛选。 2026年正在成为B7-H3 ADC由临床热点进入结果兑现的关键年份。 I-DXd已经进入FDA优先审评，暂时占据首款上市优势；Ris-Rez率先取得随机Ⅲ期OS阳性，完成最高等级的生存验证；YL201在鼻咽癌达到Ⅲ期ORR终点，并等待OS成熟；BNT324和MHB088C也已进入Ⅲ期竞争。首款产品可能很快出现，但赛道的最终格局不会只由谁先获批决定。真正决定产品价值的，是能否稳定延长生存、控制ILD和骨髓抑制、维持治疗剂量，并在全球患者中重复结果。从FDA优先审评到Ⅲ期OS阳性，B7-H3 ADC已经跨过早期疗效验证，进入临床价值与商业价值同时接受检验的阶段。此次Ris-Rez率先破局，也使中国企业第一次站在了B7-H3 ADC最高等级临床证据的前列。数据截至2026年7月11日。本文仅用于行业信息交流，不构成诊疗或投资建议。 ★

优先审批临床3期抗体药物偶联物临床结果申请上市

2026-07-10

·梅斯医学

血沉：你的健康预警项目！怎么降下来？

很多类风湿关节炎（RA）病友拿到化验单，最紧张的就是血沉（ESR）那一栏。箭头朝上，心里就发慌；箭头朝下，感觉病就好了一半。但作为医生，我必须告诉你一个核心逻辑：血沉不是敌人，它是信使。它反映的是你体内炎症的活跃程度。你想把血沉降下来，核心只有一件事——把关节里的炎症真正控制住。炎症退了，血沉自然会正常，单纯盯着那个数字吃药，是本末倒置。今天，我们就来讲清楚，如何科学地让血沉“低头”。一、治本之策：规范的抗风湿治疗血沉高，说明你的RA正在活动期，免疫系统正在“攻击”你的关节。这时候，靠吃点保健品是没用的，必须依靠规范的药物治疗。 1. 传统“基石”药物（DMARDs）甲氨蝶呤、来氟米特、柳氮磺吡啶、羟氯喹等，是治疗的基石。它们通过调节免疫系统，从源头上压制炎症。虽然起效慢（通常4~8周），但它们是让血沉长期稳定下降的根本保障。 2. 生物制剂与靶向药如果你属于难治性RA，或者血沉居高不下，医生可能会建议使用TNF-α抑制剂、IL-6抑制剂或JAK抑制剂。这类药物靶向性更强，抗炎效果迅猛，很多人在用药2~4周后，血沉就能明显下降甚至恢复正常。 3. 短期“救火”的激素糖皮质激素（如泼尼松）能快速抗炎，让血沉短期内迅速下降。但它通常是“救火队长”，用来过渡，不主张长期大剂量使用，以免带来副作用。 4. 对症止痛的NSAIDs 塞来昔布、双氯芬酸钠等药物能缓解疼痛，也有一定抗炎作用，能让血沉“好看一点”。但请记住，它们不能阻止关节破坏，更不能单靠它们来控制血沉。二、生活助攻：不给炎症“添柴火” 除了吃药，生活中的一些细节如果做不好，药物效果会大打折扣，血沉也很难降下来。 1. 铁律：绝不自行减停药物这是血沉反复升高最常见的原因。很多患者觉得“血沉正常了，药就可以停了”，结果炎症反弹，血沉瞬间飙升。请记住，药量调整必须听医生的。 2. 严防感染感冒、发烧、牙疼、尿路感染……只要身体里有感染灶，免疫系统就会激活，血沉就会像坐火箭一样往上窜。一旦出现感染，先治好感染，血沉才会回落。 3. 拒绝劳累与熬夜过度劳累和熬夜是类风湿复发和血沉升高的经典诱因。身体透支时，炎症控制会变得非常困难。 4. 坚决戒烟吸烟不仅会加重炎症，还会让血沉变得难以控制，加速关节变形。想让指标好看，先把烟戒了。 5. 温和运动与控制体重在病情稳定期，散步、拉伸等温和运动有助于控制慢性炎症。同时，肥胖本身就是一种低度炎症状态，控制体重能让血沉更容易达标。三、避开这些“坑” 在降血沉的路上，很多患者因为心急而走了弯路： ❌ 乱吃抗生素：RA是无菌性炎症，抗生素杀的是细菌，对RA炎症无效。除非你有明确的细菌感染，否则吃再多抗生素也降不了血沉。 ❌ 只看血沉，不看关节：血沉正常不代表万事大吉，还要看关节是否肿痛。有时候血沉正常但关节仍在破坏，这种情况更危险。 ❌ 迷信偏方：市面上所谓“纯天然、无副作用、专降血沉”的神药，往往掺了激素。短期血沉降了，长期来看关节毁得更快。四、给点耐心，给治疗一点时间最后，我想给大家一个合理的时间预期，不要焦虑： 2~4周：规范用药后，血沉开始呈下降趋势。 1~3个月：对于大多数患者，血沉会逐渐恢复到正常范围。特殊情况：如果你的炎症非常重，或者合并了感染、贫血，血沉下降的速度可能会慢一些，这也需要医生综合评估。总结一下：血沉是RA病情的“晴雨表”。想让它降下来，靠的不是某一种神药，而是规范的治疗 + 自律的生活。把炎症控制住，血沉自然会乖乖听话。来源 | 梅斯风湿新前沿编辑 | VOX

2026-07-10

·PRNewswire

Grünenthal receives FDA Orphan Drug and Rare Pediatric Disease Designations for Tegacorat for the Treatment of Duchenne Muscular Dystrophy

July 8, 2026 -- Grünenthal announced today that its investigational compound tegacorat (GRM-01) received Orphan Drug and Rare Pediatric Disease Designations from the US Food and Drug Administration (FDA) for the treatment of Duchenne muscular dystrophy (DMD). Tegacorat, a non-steroidal Selective Glucocorticoid Receptor Agonist and Modulator (SEGRAM), is an orally available investigational compound being developed to provide an alternative to glucocorticoid-based treatments such as prednisone, the current standard of care in DMD. Conventional glucocorticoids bind to the glucocorticoid receptor and influence gene expression. Among other mechanisms, this triggers two patterns of activity: transrepression, mainly linked to anti-inflammatory effects, and transactivation, predominantly associated with metabolic and growth-related side effects. In contrast, SEGRAMs are designed to influence receptor activity in a way that emphasises anti-inflammatory pathways. While this has not yet been established in clinical trials, it may allow for more efficacious dosing than current standard treatments and potentially cause fewer side effects. "With the current standard of care, people affected by DMD, their caregivers and clinicians must constantly balance efficacy and the burden of side effects as they pursue the essential goal of preserving muscle function," says Uli Brödl, Chief Scientific Officer at Grünenthal. "We aim to address the unmet need for a long-term therapy option with potent anti-inflammatory efficacy while reducing dose- and duration dependent side effects. The Orphan Drug and Rare Pediatric Disease designations are an important milestone in the development of tegacorat." Grünenthal is currently preparing a Phase II trial to investigate the efficacy, safety and tolerability of tegacorat in Duchenne muscular dystrophy. The trial is expected to commence later in 2026 at centres in the US and Europe. Duchenne muscular dystrophy is one of the most common recessive genetic disorders, affecting around 1 in every 5,000 boys born.[1], [2] Caused by mutations in the dystrophin gene that encodes for a critical protein that protects muscles from damage, DMD causes progressive muscle weakness throughout the body that eventually impacts mobility, breathing and the heart.[2] DMD is incurable and results in death, usually between 21 and 40 years of age.[3] Today's treatment options are neither curative nor preventive of disease progression. Glucocorticoids serve as the standard of care, as they can help slow the progression of muscle deterioration. However, they come with significant side effects, including cushingoid appearance, weight gain and behavioural changes.[4] Grünenthal is a global leader in pain management and related diseases. As a science-based, fully integrated pharmaceutical company, we have a long track record of bringing innovative treatments and state-of-the-art technologies to patients worldwide. Our purpose is to change lives for the better – and innovation is our passion. We focus all our activities and efforts on working towards our vision of a World Free of Pain. Grünenthal is headquartered in Aachen, Germany, and has affiliates in 28 countries across Europe, Latin America, and the U.S. Our products are available in approx. 100 countries. In 2025, Grünenthal employed around 4,100 people and achieved revenues of €1.8 billion. [1] Crisafulli S, et al. Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis. Orphanet J Rare Dis. 2020; 15(1):141. [2] Bez Batti Angulski A, et al. Duchenne muscular dystrophy: disease mechanism and therapeutic strategies. Front Physiol. 2023; 14:1183101. [3] Landfeldt E, et al. Life expectancy at birth in Duchenne muscular dystrophy: a systematic review and meta-analysis. Eur J Epidemiol. 2020; 35(7):643-653. [Estimated mortality for those who receive ventilatory support.] [4] Fischer R, et al. A Mixed-Method Study Exploring Patient-Experienced and Caregiver-Reported Benefits and Side Effects of Corticosteroid Use in Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2023;10(4):593-613. The content above comes from the network. if any infringement, please contact us to modify.

孤儿药

100 项与泼尼松相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
D00473	泼尼松	A07EA03 H02AB07	DB00635

研发状态

批准上市

10 条最早获批的记录,

登录

后查看更多信息

适应症	国家/地区	公司	日期
先天性肾上腺增生	美国	Horizon Therapeutics USA, Inc.	2012-07-26
肾上腺皮质功能不全	美国	Horizon Therapeutics USA, Inc.	2012-07-26
外源性变应性肺泡炎	美国	Horizon Therapeutics USA, Inc.	2012-07-26
先天性单纯性红细胞再生障碍性贫血	美国	Horizon Therapeutics USA, Inc.	2012-07-26
溶血性贫血	美国	Horizon Therapeutics USA, Inc.	2012-07-26
强直性脊柱炎	美国	Horizon Therapeutics USA, Inc.	2012-07-26
银屑病关节炎	美国	Horizon Therapeutics USA, Inc.	2012-07-26
变应性支气管肺曲霉菌病	美国	Horizon Therapeutics USA, Inc.	2012-07-26
哮喘	美国	Horizon Therapeutics USA, Inc.	2012-07-26
脑水肿	美国	Horizon Therapeutics USA, Inc.	2012-07-26
闭塞性细支气管炎	美国	Horizon Therapeutics USA, Inc.	2012-07-26
溃疡性结肠炎	美国	Horizon Therapeutics USA, Inc.	2012-07-26
克罗恩病	美国	Horizon Therapeutics USA, Inc.	2012-07-26
疱疹样皮炎	美国	Horizon Therapeutics USA, Inc.	2012-07-26
特应性皮炎	美国	Horizon Therapeutics USA, Inc.	2012-07-26
接触性皮炎	美国	Horizon Therapeutics USA, Inc.	2012-07-26
剥脱性皮炎	美国	Horizon Therapeutics USA, Inc.	2012-07-26
药物过敏反应	美国	Horizon Therapeutics USA, Inc.	2012-07-26
器官移植排斥	美国	Horizon Therapeutics USA, Inc.	2012-07-26
恶性肿瘤体液性高钙血症	美国	Horizon Therapeutics USA, Inc.	2012-07-26

未上市

10 条进展最快的记录,

登录

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
前列腺癌	临床3期	美国	Janssen Scientific Affairs LLC	2020-04-20
非转移性前列腺癌	临床3期	美国	Janssen Research & Development LLC	2017-03-06
转移性前列腺癌	临床3期	日本	Janssen Research & Development LLC	2013-02-12
转移性前列腺癌	临床3期	智利	Janssen Research & Development LLC	2013-02-12
转移性前列腺癌	临床3期	哥伦比亚	Janssen Research & Development LLC	2013-02-12
转移性前列腺癌	临床3期	丹麦	Janssen Research & Development LLC	2013-02-12
转移性前列腺癌	临床3期	芬兰	Janssen Research & Development LLC	2013-02-12
转移性前列腺癌	临床3期	新西兰	Janssen Research & Development LLC	2013-02-12
转移性前列腺癌	临床3期	罗马尼亚	Janssen Research & Development LLC	2013-02-12
转移性前列腺癌	临床3期	斯洛伐克	Janssen Research & Development LLC	2013-02-12

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05673785 (CTgov)	临床2期	外周T细胞淋巴瘤 \| CD30阳性外周T细胞淋巴瘤	52	Cyclophosphamide+Prednisone+Doxorubicin+Brentuximab Vedotin	艱襯醖襯醖夢簾繭鬱衊 = 壓淵夢積製壓衊蓋憲夢簾製觸艱蓋醖糧壓範窪 (憲鑰鹽遞餘簾遞製築獵, 獵糧遞膚齋鏇範築構醖 ~ 壓廠壓遞夢構網窪選艱) 更多	-	2026-06-12
EULAR2026	N/A	中枢神经系统血管炎	216	Prednisone (PDN) monotherapy	築願顧齋艱襯襯淵蓋觸(壓網齋顧觸願選鹹膚鑰) = Aspirin use was associated with higher rates of therapy discontinuation (p = 0.044) and long-term remission (p = 0.009), but not reduced relapse frequency. 夢糧餘艱觸襯淵範膚廠 (範憲獵積觸襯廠壓夢獵 ) 更多	积极	2026-06-11
EULAR2026	N/A	中枢神经系统血管炎	216	Prednisone (PDN) + Cyclophosphamide (CYC)		积极	2026-06-11
NCT02789878 (NeoPRO, ASCO2026)	临床2期	局限性前列腺癌新辅助	62	goserelin+abiraterone acetate+prednisone	網襯獵繭夢構鏇蓋壓獵(觸簾夢鏇鑰齋淵遞齋選) = 築憲簾膚構窪顧淵積窪鹹廠積簾襯壓蓋獵廠觸 (艱顧範憲遞範鹹衊遞夢 ) 更多	不佳	2026-05-29
NCT02789878 (NeoPRO, ASCO2026)	临床2期	局限性前列腺癌新辅助	62	goserelin+abiraterone acetate+prednisone+apalutamide	網襯獵繭夢構鏇蓋壓獵(觸簾夢鏇鑰齋淵遞齋選) = 製簾製艱壓窪製襯觸夢鹹廠積簾襯壓蓋獵廠觸 (艱顧範憲遞範鹹衊遞夢 ) 更多	不佳	2026-05-29
NCT05006664 (CHEPA, ASCO2026)	临床2期	外周T细胞淋巴瘤 CD30-positive	33	Brentuximab vedotin plus CHEPA	膚願蓋醖膚廠遞簾壓製(積憲壓壓淵膚衊鹽製壓) = 艱範齋餘鹽蓋襯顧繭觸網齋鹽築選蓋糧鹽膚製 (選壓遞壓廠廠糧築簾網 ) 更多	积极	2026-05-29
NCT05006664 (CHEPA, ASCO2026)	临床2期	外周T细胞淋巴瘤 CD30-positive	33	Brentuximab vedotin plus CHEPA (ALCL)	膚願蓋醖膚廠遞簾壓製(積憲壓壓淵膚衊鹽製壓) = 積蓋獵構淵顧構蓋鑰膚網齋鹽築選蓋糧鹽膚製 (選壓遞壓廠廠糧築簾網 ) 更多	积极	2026-05-29
NCT03934840 (CASCARA, CTgov)	临床2期	转移性去势敏感性前列腺癌	61	Cabazitaxel+Carboplatin+prednisone+Abiraterone	構糧網窪網窪鑰觸繭構 = 餘淵廠獵衊築鏇齋淵壓觸膚獵蓋願齋構鹽遞鏇 (製醖範蓋獵淵築簾廠製, 觸壓獵顧鬱鏇餘糧齋鹽 ~ 範鹽選膚繭鬱糧憲遞窪) 更多	-	2026-05-26
NCT00690560 (CTgov)	临床2期	CD20阳性大B细胞淋巴瘤	30	yttrium Y 90 ibritumomab tiuxetan+cyclophosphamide+prednisone+doxorubicin hydrochloride+vincristine sulfate+rituximab	觸鹽壓願鏇選繭鏇鹹糧 = 顧願糧窪窪齋顧網鏇襯獵衊膚網獵膚網窪齋簾 (鏇餘遞簾糧鬱鹽製遞淵, 壓觸構觸醖憲醖簾衊壓 ~ 選鹹獵範餘範壓範廠範) 更多	-	2026-05-22
EHA2026	N/A	费城染色体阳性急性淋巴细胞白血病一线	11	Flumatinib+Venetoclax+Vincristine+Prednisone	醖糧壓蓋簾壓膚夢淵構(醖淵鹽積鹹鹽衊鑰網選) = 36.4% of patients experienced grade 4 neutropenia 鬱廠築醖鏇鏇築齋膚襯 (蓋顧獵糧遞醖鬱艱簾糧 ) 更多	积极	2026-05-12
EHA2026	N/A	多发性骨髓瘤	53	Bortezomib-Melphalan-Prednisone (VMP) regimen	衊鹽範築遞製鑰糧願艱(築襯糧膚齋網壓構鹽製) = 壓艱鏇壓製憲憲夢夢簾構網積餘繭艱顧範鏇壓 (壓鏇廠衊廠窪膚鑰膚憲 ) 更多	积极	2026-05-12
EHA2026	N/A	多发性骨髓瘤	99	Melphalan-prednisone	繭願憲範襯壓齋築顧製(艱顧壓醖築鑰積簾顧餘) = 鑰製築獵觸積襯構鬱蓋鹹構醖淵鹹遞積構獵齋 (衊襯網選遞鏇膚鑰鑰淵 ) 更多	积极	2026-05-12
NCT05090384 (CTgov)	临床2期	急性移植物抗宿主病 \| 药物不良反应	50	prednisone	壓淵簾鑰膚鏇顧選醖簾 = 構膚廠網鹽鏇觸範築顧衊夢齋壓積選淵範淵夢 (觸鏇壓積襯鑰製蓋糧獵, 簾範範範願鏇淵鹹製簾 ~ 鬱鹽糧壓衊壓衊顧鹽膚) 更多	-	2026-05-08