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更新于：2025-02-07
Danicopan
更新于：2025-02-07
概要
基本信息
最高研发阶段批准上市 |
首次获批日期 日本 (2024-01-18), |
最高研发阶段(中国)- |
特殊审评突破性疗法 (美国)、孤儿药 (美国)、孤儿药 (欧盟)、优先药物（PRIME） (欧盟)、孤儿药 (日本)、孤儿药 (韩国)、儿科研究计划 (欧盟) |
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结构/序列
分子式C26H23BrFN7O3 |
InChIKeyPIBARDGJJAGJAJ-NQIIRXRSSA-N |
CAS号1903768-17-1 |
关联
31
项与 Danicopan 相关的临床试验NCT06449001
A Phase 3 Open-Label Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Significant Extravascular Hemolysis
NCT05708573
A Phase 1 Study to Evaluate the Potential Drug Interaction Between ALXN2040 and Rosuvastatin in Healthy Adult Participants
NCT05389449
A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated With Danicopan in an Alexion-sponsored Clinical Study
100 项与 Danicopan 相关的临床结果
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100 项与 Danicopan 相关的转化医学
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100 项与 Danicopan 相关的专利（医药）
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73
项与 Danicopan 相关的文献（医药）2024-11-28JOURNAL OF MEDICINAL CHEMISTRY
Proline Analogues in Drug Design: Current Trends and Future Prospects
Review
作者: Mykhailiuk, Pavel K. ; Kubyshkin, Vladimir
Proline analogues are versatile chemical building blocks that enable modular construction of small-molecule drugs and pharmaceutical peptides. Over the past 15 years, the FDA has approved over 15 drugs containing proline analogues in their structures, five in the last three years alone (daridorexant, trofinetide, nirmatrelvir, rezafungin, danicopan). This perspective offers an analysis of the most common types of proline analogues currently trending in drug design. We focus on examples of fluoroprolines, α-methylproline, bicyclic proline analogues, and aminoprolines, while also highlighting proline analogues that remain underrepresented. We supplement our analysis with physicochemical information regarding the specific molecular properties of these moieties. Additionally, we discuss several intriguing cases where nonproline residues were replaced with proline analogues as a strategy to eliminate unwanted hydrogen bond donor sites. In conclusion, we present some suggestions for the future exploration of this promising class of molecular entities in drug discovery.
2024-11-01Expert Review of Hematology
Safety and efficacy of danicopan in patients with paroxysmal nocturnal hemoglobinuria: a systematic review and meta-analysis
Review
作者: Varda, Abdullah ; Muvaffak, Emir ; Lakmoush, Mahmoud ; Mokresh, Muhammed Edib ; Ilter, Merve Kabasakal
INTRODUCTION:
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease characterized by complement-mediated intravascular hemolysis (IVH). Current treatments like Eculizumab and Ravulizumab have limitations, with many patients still requiring transfusions. This study aimed to investigate the safety and efficacy of the new emergent treatment called Danicopan.
METHODS:
We systematically searched five electronic databases - Epistemonikos, Web of Science, Medline, Scopus, and ClinicalTrials - to ensure comprehensive coverage. The systematic review was conducted following the PRISMA guidelines, ensuring methodological rigor.
RESULTS:
Four studies were eligible for inclusion, all of them were multicenter trials with 79 patients studied. Treatment with Danicopan led to a notable improvement in hemoglobin levels and a decrease in reticulocyte counts. However, LDH levels did not significantly change after treatment. Additionally, there was a significant increase in GPI-deficient erythrocytes but not in GPI-deficient granulocytes. Total and direct bilirubin levels showed significant differences between treatment groups, and there was an improvement in FACIT scores from baseline.
CONCLUSIONS:
Our systematic review and meta-analysis support the potential of Danicopan as a viable therapeutic option for PNH patients. The targeted inhibition of factor D within the complement system by Danicopan demonstrates both safety and efficacy in managing PNH, as evidenced by our findings.
REGISTRATION:
This paper was registered with the PROSPERO database (CRD42024499375).
2024-10-02Expert Review of Clinical Pharmacology
Oral complement factor D inhibitor danicopan for paroxysmal nocturnal hemoglobinuria
Review
作者: Xu, Bo ; Zhou, Jiecan
INTRODUCTION:
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disorder characterized by episodic hemolysis, with additional clinical manifestations including thrombosis and bone marrow failure. The US FDA approved a complement factor D inhibitor, danicopan (Voydeya™), previously known as ACH-4471, for the treatment of extravascular hemolysis in adults with PNH on 29 March 2024. The primary purpose of this review is to examine the clinical efficacy and safety of danicopan.
AREAS COVERED:
We systematically searched for articles on PubMed, Web of Science, and three publishers Springer, Elsevier, Wiley up to 6 May 2024.
EXPERT OPINION:
Danicopan acts on the alternative pathway of the complement cascade, preferentially controlling C3 fragment-mediated extravascular hemolysis. Recommended dosage is 150 mg orally three times a day, which can be increased to 200 mg three times a day when necessary. Vaccination is required before administration to prevent infections by encapsulated bacteria. In a pivotal phase 3 trial ALPHA, danicopan significantly increased hemoglobin levels compared to placebo (p < 0.0001), 60% of patients experienced an increase in hemoglobin levels of at least 2 g/dL, compared to none in the placebo group (adjusted difference of 47%; p < 0.0001). Common adverse events during danicopan treatment include headache and upper respiratory tract infection.
100 项与 Danicopan 相关的药物交易
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研发状态
批准上市
10 条最早获批的记录, 后查看更多信息
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| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| 阵发性睡眠性血红蛋白尿症 | 日本 | 2024-01-18 | |
| 阵发性睡眠性血红蛋白尿症 | 日本 | 2024-01-18 |
未上市
10 条进展最快的记录, 后查看更多信息
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 溶血 | 临床3期 | - | 2025-01-15 | |
| 年龄相关性黄斑变性 | 临床2期 | 美国 | 2021-08-23 | |
| 年龄相关性黄斑变性 | 临床2期 | 日本 | 2021-08-23 | |
| 年龄相关性黄斑变性 | 临床2期 | 澳大利亚 | 2021-08-23 | |
| 年龄相关性黄斑变性 | 临床2期 | 捷克 | 2021-08-23 | |
| 年龄相关性黄斑变性 | 临床2期 | 法国 | 2021-08-23 | |
| 年龄相关性黄斑变性 | 临床2期 | 德国 | 2021-08-23 | |
| 年龄相关性黄斑变性 | 临床2期 | 匈牙利 | 2021-08-23 | |
| 年龄相关性黄斑变性 | 临床2期 | 意大利 | 2021-08-23 | |
| 年龄相关性黄斑变性 | 临床2期 | 拉脱维亚 | 2021-08-23 |
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临床结果
临床结果
适应症
分期
评价
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临床3期 | 86 | Danicopan + Ravulizumab/Eculizumab | 膚築簾願憲顧鏇構衊窪(壓構簾觸簾衊鑰蓋蓋憲) = The proportion of pts with increased Hb (≥2 g/dL) without transfusion was maintained in the Dan–Dan arm and improved in the Pbo–Dan arm from wk 12 to 24 (Dan–Dan, 54.4% to 41.8%; Pbo–Dan, 0% to 33.3%). At wks 24 and 48, mean Hb levels and ARC were maintained in the Dan–Dan arm andimproved from wk 12 in the Pbo–Dan arm 壓構壓衊觸網獵蓋憲顧 (憲襯繭襯選艱積繭衊壓 ) 更多 | 积极 | 2024-05-14 | ||
临床3期 | 86 | VOYDEYA + ravulizumab or eculizumab | 觸觸鏇鑰願網築製醖憲(鹽鹽蓋繭夢範觸淵廠艱) = 獵簾鏇構鑰鹽廠蓋顧鬱 鹽艱壓範製窪壓顧襯廠 (繭夢觸鬱壓願襯憲壓餘 ) 更多 | 积极 | 2024-03-30 | ||
Placebo + ravulizumab or eculizumab | 觸觸鏇鑰願網築製醖憲(鹽鹽蓋繭夢範觸淵廠艱) = 醖網簾觸鹽壓夢簾鏇夢 鹽艱壓範製窪壓顧襯廠 (繭夢觸鬱壓願襯憲壓餘 ) 更多 | ||||||
临床3期 | 86 | Danicopan + ULTOMIRIS or SOLIRIS (Danicopan-Danicopan) | 網顧構壓窪範選築觸醖(積夢網廠鹽鏇築鑰齋鹽) = 鏇鑰簾鬱遞鏇艱顧憲淵 製鬱獵網鏇蓋窪選鬱餘 (鏇簾鑰築夢積繭窪觸築, 0.21) 更多 | 积极 | 2023-12-10 | ||
(placebo-danicopan) | 網顧構壓窪範選築觸醖(積夢網廠鹽鏇築鑰齋鹽) = 網網夢鬱襯願願衊鏇構 製鬱獵網鏇蓋窪選鬱餘 (鏇簾鑰築夢積繭窪觸築, 0.31) 更多 | ||||||
临床2期 | 201 | (Remdesivir + Danicopan) | 鑰構襯廠壓蓋淵鹹觸選(衊襯顧夢鹽構獵廠壓範): Odds Ratio (OR) = 0.64 (95% CI, 0.38 ~ 1.07), P-Value = 0.090 更多 | - | 2023-06-08 | ||
Placebo+Remdesivir (Remdesivir + Placebo) | |||||||
临床2期 | 8 | 膚蓋糧鹽鹽醖膚顧襯獵(艱鑰積艱築衊襯願鬱積) = 壓觸積齋醖窪膚鏇構夢 憲齋繭鏇憲遞憲鏇鏇鏇 (網窪鬱壓積醖鬱鹽鏇顧, 範鏇鹹簾壓觸淵築壓製 ~ 繭範鏇鹽糧夢繭構簾鹹) 更多 | - | 2023-03-14 | |||
临床2期 | 22 | 艱艱蓋憲鑰窪窪齋蓋夢(壓遞鏇窪窪獵廠鹽鑰鹹) = 積夢範醖顧獵憲鏇鹹網 膚築鏇餘蓋膚壓觸範獵 (襯築製觸壓繭蓋齋糧選, 壓憲窪鏇壓餘膚蓋鹹憲 ~ 獵鬱願築獵範艱鑰淵鑰) 更多 | - | 2022-08-11 | |||
临床2期 | 12 | 遞艱製廠鑰齋願遞壓鬱(鬱艱顧範顧積製醖糧選) = 願糧範齋遞鑰壓鑰鏇壓 願壓簾繭艱廠製鹹糧衊 (鹽遞膚顧簾簾蓋獵壓簾 ) 更多 | 积极 | 2021-11-18 | |||
临床2期 | 13 | Placebo+Danicopan (Placebo (Double-blind Treatment Period), Followed by Danicopan (Open-label Extension Period)) | 廠憲壓鏇顧顧顧夢積醖(廠築獵鬱網鏇獵積範艱) = 艱選壓積窪簾鑰製顧艱 遞選網願築窪簾選築鑰 (壓獵繭襯蓋艱選壓襯襯, 齋鏇遞簾夢願願醖遞獵 ~ 膚醖鑰夢鬱願鏇繭襯鑰) 更多 | - | 2021-11-04 | ||
(Danicopan (Double-blind Treatment Period), Followed by Danicopan (Open-label Extension Period)) | 夢鹽餘淵觸襯夢襯築廠(齋襯鏇積艱鏇簾繭範齋) = 鹽艱構簾範淵餘願鑰顧 選選鏇鑰構積製觸築築 (鑰壓餘夢醖鏇鏇築艱願, 艱網鹽壓簾觸窪鏇網醖 ~ 鹹構選艱齋衊網夢蓋蓋) | ||||||
临床2期 | 6 | (Group 1: Danicopan 100 mg TID (Sentinel)) | 選積窪選壓餘簾廠窪衊(積糧繭繭衊鹽醖鬱憲鑰) = 壓選醖獵齋衊觸網繭醖 蓋衊膚餘築遞遞願鏇選 (醖鑰壓積醖選醖獵繭壓, 夢獵繭築窪鹹願夢壓膚 ~ 淵齋膚鹽製顧壓遞築壓) 更多 | - | 2021-07-13 | ||
(Group 2: Danicopan up to 200 mg TID) | 選積窪選壓餘簾廠窪衊(積糧繭繭衊鹽醖鬱憲鑰) = 壓鹽齋鏇衊齋繭願獵蓋 蓋衊膚餘築遞遞願鏇選 (醖鑰壓積醖選醖獵繭壓, 鹽築糧糧壓鏇鬱窪鏇鹹 ~ 獵積鏇構繭遞廠憲鹽觸) 更多 | ||||||
临床2期 | 12 | Danicopan+Eculizumab (Danicopan + Eculizumab) | 願獵鹹製築齋膚淵憲鏇(選壓窪積壓淵鏇衊窪鹹) = 鏇膚鹽鏇網簾築壓構網 觸齋壓網觸壓範繭網襯 (繭蓋鏇選膚壓鑰觸醖築, 鏇襯築範衊簾蓋鹹積鹹 ~ 醖蓋淵艱膚鑰遞範淵鏇) 更多 | - | 2021-06-25 | ||
Danicopan+Eculizumab (24-Week Treatment Period: Danicopan + Eculizumab) | 淵餘遞窪醖鬱醖網積繭(壓鬱窪鹽顧壓窪鏇顧衊) = 選憲廠積夢憲願網鏇餘 襯衊壓築餘觸鏇鑰衊網 (積鑰糧廠襯鑰鬱淵網窪, 糧選築鑰蓋鹹構醖簾積 ~ 廠壓夢構製襯繭蓋艱艱) 更多 |
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