预约演示
更新于：2026-09-08
Avapritinib
阿伐替尼
更新于：2026-09-08
概要
基本信息
药物类型 小分子化药 |
别名 Avapritinib (USAN/INN)、阿伐普替尼、阿泊替尼 + [7] |
作用方式 抑制剂 |
作用机制 PDGFRα抑制剂(血小板衍生生长因子受体α抑制剂)、c-Kit抑制剂(干细胞生长因子受体抑制剂) |
在研适应症 |
非在研适应症 |
非在研机构- |
权益机构 |
最高研发阶段批准上市 |
首次获批日期 美国 (2020-01-09), |
最高研发阶段(中国)批准上市 |
特殊审评快速通道 (美国)、孤儿药 (美国)、优先审评 (美国)、孤儿药 (英国)、附条件批准 (中国)、优先审评 (中国)、突破性疗法 (美国) |
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结构/序列
分子式C26H27FN10 |
InChIKeyDWYRIWUZIJHQKQ-SANMLTNESA-N |
CAS号1703793-34-3 |
关联
29
项与 阿伐替尼 相关的临床试验ChiCTR2600129807
Luvometinib Monotherapy or Combined With Cytarabine for Langerhans Cell Histiocytosis: A Response-adapted, Interventional, Prospective Study
NCT07255638
A Prospective Non-Interventional Study to Describe the Effectiveness of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Patients With Indolent Systemic Mastocytosis and Symptoms That Are Not Adequately Controlled With Symptomatic Treatments in Real-World Settings
NCT07028073
Avapritinib Combined With Standard Therapy for the Treatment of Newly Diagnosed KIT Mutation Acute Myeloid Leukemia With t(8;21)(q22;q22.1); Inv(16)(p13.1q22) or t(16;16)(p13.1;q22): a Prospective, Multi-center, Single-arm, Two-cohorts Study
100 项与 阿伐替尼 相关的临床结果
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100 项与 阿伐替尼 相关的转化医学
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100 项与 阿伐替尼 相关的专利（医药）
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288
项与 阿伐替尼 相关的文献（医药）2026-12-31Future Science OA
A comprehensive analysis of the adverse drug events of metastatic and unresectable gastrointestinal stromal tumor treatment options: avapritinib, imatinib, regorafenib, ripretinib, and sunitinib safety insights and implications
Article
作者: Andrews, Michael B. ; Thompson, Cameron M. ; Adler, Douglas G.
BACKGROUND:
Treatments for metastatic, unresectable, or treatment-resistant gastrointestinal stromal tumors (GIST) include avapritinib, imatinib, regorafenib, ripretinib, and sunitinib. The FDA Adverse Event Reporting System (FAERS) database collects medication-related adverse events (AEs).
DESIGN AND METHODS:
Reports in the FAERS database from each medication's FDA approval to June 30, 2025 were analyzed. Reports containing other medications or indications outside of GIST treatment were excluded.
RESULTS:
Avapritinib was associated with fatigue (n = 826, 10.2%), nausea/vomiting (n = 574, 7.1%), eye/facial swelling (n = 631, 7.8%), diarrhea n = 361, 4.5%), peripheral edema (n = 231, 2.5%). Imatinib was associated with nausea/vomiting (n = 677, 4.4%), fatigue (n = 549, 3.6%), anemia (n = 404, 2.6%), abdominal pain (n = 400, 2.6%), and rash (n = 340, 2.2%), hemorrhage (317 AEs, 2.1%). Regorafenib was associated with fatigue (n = 104, 8.6%), palmar-plantar erythrodyesthesia syndrome (n = 66, 5.4%), diarrhea (n = 60, 4.9%). Ripretinib was associated with fatigue (n = 534, 8.4%), alopecia (n = 355, 5.6%), nausea/vomiting (n = 273, 4.3%). Sunitinib was associated with fatigue (n = 420, 6.1%%), nausea/vomiting (n = 284, 4.2%), diarrhea (n = 262, 3.8 %), hemorrhage (n = 149, 2.2%).
CONCLUSIONS:
The FAERS database revealed multiorgan AEs associated with five GIST treatment medications. Novel findings include eye/facial and peripheral edema with avapritinib, palmar-plantar erythrodyesthesia syndrome, headache/migraine, and gait abnormalities across all five medications, pancytopenia with imatinib, and hemorrhage with imatinib and sunitinib.
2026-09-01AMERICAN JOURNAL OF HEMATOLOGY
Systemic Mastocytosis in Adults: 2026 Update on Diagnosis, Risk Stratification and Management
Review
作者: Pardanani, Animesh
ABSTRACT:
Overview:
Systemic mastocytosis (SM) results from clonal proliferation of mast cells (MC) in extra‐cutaneous organs.
Diagnosis:
The major criterion is the presence of multifocal MC clusters in the bone marrow and/or extracutaneous organs. Minor diagnostic criteria include elevated serum tryptase level, MC CD25/CD2/CD30 expression, and the presence of activating
KIT
mutations.
Risk Stratification:
Establishing SM subtype as per the International Consensus Classification/World Health Organization classification systems is an important first step. Patients either have indolent/smoldering SM (ISM/SSM) or advanced SM, including aggressive SM (ASM), SM with associated myeloid neoplasm (SM‐AMN), or mast cell leukemia. Identification of poor‐risk mutations (i.e.,
ASXL1, RUNX1, SRSF2, NRAS
) further refines the risk stratification. Several risk models are available to help assign prognosis in SM patients.
Management:
Treatment goals for ISM patients are primarily directed towards anaphylaxis prevention/symptom control/osteoporosis treatment. Patients with advanced SM frequently need MC cytoreductive therapy to reverse disease‐related organ dysfunction. Tyrosine kinase inhibitors (TKI) (midostaurin, avapritinib) have changed the treatment landscape in SM. While deep biochemical, histological, and molecular responses have been documented with avapritinib treatment, its efficacy as monotherapy against a multimutated AMN disease component in SM‐AMN patients remains unclear. Cladribine continues to have a role for MC debulking, whereas interferon‐α has a diminishing role in the TKI era. Treatment of SM‐AMN primarily targets the AMN component, particularly if an aggressive disease such as acute leukemia is present. Allogeneic stem cell transplant has a role in such patients. Imatinib has a therapeutic role only in the rare patient with an imatinib‐sensitive KIT mutation.
2026-08-01JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
Avapritinib achieves long-term disease control with favorable safety in patients with indolent systemic mastocytosis over 3 years
Article
作者: Akin, Cem ; Van Daele, Paul ; Shomali, William ; Vachhani, Pankit ; Panse, Jens ; Castells, Mariana ; DeAngelo, Daniel J ; Jurcic, Joseph ; Rein, Lindsay ; Broesby-Olsen, Sigurd ; Triggiani, Massimo ; Siebenhaar, Frank ; Álvarez-Twose, Iván ; Radia, Deepti H ; Bose, Prithviraj ; Pongdee, Thanai ; Dybedal, Ingunn ; Hong, Janet ; Sabato, Vito ; Cerquozzi, Sonia ; Elberink, Hanneke Oude ; Doyle, Ashley ; Hartmann, Karin ; Gotlib, Jason ; Mattsson, Mattias ; Bidollari, Ilda ; Bonadonna, Patrizia ; Vadas, Peter ; Reiter, Andreas ; Lin, Hui-Min ; George, Tracy I ; Tashi, Tsewang ; Livideanu, Cristina Bulai ; Giannetti, Matthew ; Schafhausen, Philippe ; Lampson, Benjamin ; Barete, Stéphane ; Ustun, Celalettin
BACKGROUND:
Indolent systemic mastocytosis (ISM), a clonal mast cell disease primarily driven by the KIT D816V mutation, can cause long-term debilitating symptoms and poor quality of life. Most patients rely on symptom-directed best supportive care (BSC) medications, which do not treat the underlying driver of ISM. Avapritinib, an oral, potent, selective KIT D816V inhibitor, is approved in adults with ISM.
OBJECTIVE:
We sought to understand the long-term efficacy and safety of avapritinib in ISM.
METHODS:
The PIONEER trial (NCT03731260) enrolled adults with moderate to severe ISM symptoms. Patients initiated avapritinib 25 mg once daily (QD; recommended dose) plus BSC in part 1, 2, or 3; open-label part 3 is ongoing with up to 5 years of follow-up. As per investigator discretion and disease burden, a dose increase up to avapritinib 50 mg QD was permitted in part 3.
RESULTS:
As of February 21, 2025, 226 patients initiated avapritinib at 25 mg QD. The median (range) treatment duration was 40.0 (0.7-67.2) months. Patients receiving avapritinib experienced durable and clinically meaningful symptom improvement (mean change, -19.39 [n = 127] in the Indolent Systemic Mastocytosis Symptom Assessment Form total symptom score) through approximately 3 years. Avapritinib continued to be well tolerated for a longer term with a safety profile comparable with the previously reported placebo-controlled portion. Most treatment-related adverse events (TRAEs) were grades 1 to 2, with limited grade 3 or higher reported. Edema events were the most frequent TRAEs (mostly grade 1). Serious TRAEs occurred in 3 patients (1%), and 7 patients (3%) discontinued treatment because of TRAEs.
CONCLUSIONS:
Long-term follow-up (median, ∼3 years) demonstrates that avapritinib is effective and well tolerated. Avapritinib shows a favorable benefit-risk profile as a chronic ISM treatment.
100 项与 阿伐替尼 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 |
|---|---|---|---|
| 胃肠道间质瘤 | 加拿大 | 2024-09-18 | |
| 髓性系统性肥大细胞增多症 | 美国 | 2023-05-22 | |
| 侵袭性系统性肥大细胞增多症 | 欧盟 | 2023-03-14 | |
| 侵袭性系统性肥大细胞增多症 | 冰岛 | 2023-03-14 | |
| 侵袭性系统性肥大细胞增多症 | 列支敦士登 | 2023-03-14 | |
| 侵袭性系统性肥大细胞增多症 | 挪威 | 2023-03-14 | |
| 肥大细胞白血病 | 欧盟 | 2023-03-14 | |
| 肥大细胞白血病 | 冰岛 | 2023-03-14 | |
| 肥大细胞白血病 | 列支敦士登 | 2023-03-14 | |
| 肥大细胞白血病 | 挪威 | 2023-03-14 | |
| 转移性胃肠道间质瘤 | 欧盟 | 2023-03-14 | |
| 转移性胃肠道间质瘤 | 冰岛 | 2023-03-14 | |
| 转移性胃肠道间质瘤 | 列支敦士登 | 2023-03-14 | |
| 转移性胃肠道间质瘤 | 挪威 | 2023-03-14 | |
| 不可切除的胃肠道间质瘤 | 欧盟 | 2023-03-14 | |
| 不可切除的胃肠道间质瘤 | 冰岛 | 2023-03-14 | |
| 不可切除的胃肠道间质瘤 | 列支敦士登 | 2023-03-14 | |
| 不可切除的胃肠道间质瘤 | 挪威 | 2023-03-14 | |
| 系统性肥大细胞增多症 | 美国 | 2021-06-16 | |
| PDGFRA D842V突变胃肠道间质瘤 | 欧盟 | 2020-09-24 |
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 |
|---|---|---|---|---|
| 中枢神经系统肿瘤 | 临床2期 | 美国 | 2022-02-24 | |
| 中枢神经系统肿瘤 | 临床2期 | 澳大利亚 | 2022-02-24 | |
| 中枢神经系统肿瘤 | 临床2期 | 奥地利 | 2022-02-24 | |
| 中枢神经系统肿瘤 | 临床2期 | 加拿大 | 2022-02-24 | |
| 中枢神经系统肿瘤 | 临床2期 | 法国 | 2022-02-24 | |
| 中枢神经系统肿瘤 | 临床2期 | 德国 | 2022-02-24 | |
| 中枢神经系统肿瘤 | 临床2期 | 意大利 | 2022-02-24 | |
| 中枢神经系统肿瘤 | 临床2期 | 韩国 | 2022-02-24 | |
| 中枢神经系统肿瘤 | 临床2期 | 英国 | 2022-02-24 | |
| 复发性实体肿瘤 | 临床2期 | 美国 | 2022-02-24 |
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临床结果
临床结果
适应症
分期
评价
查看全部结果
临床2期 | 髓性系统性肥大细胞增多症 KIT D816V mutation | 212 | Avapritinib 25 mg once daily | 淵顧壓遞憲鹹壓顧鏇廠(廠膚網網廠範壓鹽襯壓) = 構觸淵鬱構繭糧獵鏇鏇 蓋鬱鹹廠鏇鹹衊繭艱鑰 (觸壓齋積鬱夢顧網選築 ) 更多 | 积极 | 2026-06-01 | |
Placebo | 淵顧壓遞憲鹹壓顧鏇廠(廠膚網網廠範壓鹽襯壓) = 顧鹹鹹積積選艱選壓齋 蓋鬱鹹廠鏇鹹衊繭艱鑰 (觸壓齋積鬱夢顧網選築 ) 更多 | ||||||
临床1期 | 侵袭性系统性肥大细胞增多症 D816V-mutant KIT | 107 | Avapritinib 200 mg once daily | 觸餘顧壓遞鑰鹽願鹽鹽(鬱網餘壓膚艱範構艱構) = 鬱鑰選構鑰襯繭製製範 壓糧鹽壓窪齋鬱獵夢鑰 (遞觸鏇繭顧艱製鹹艱願, 63 ~ 83) 更多 | 积极 | 2026-05-26 | |
N/A | 胃肠道间质瘤 KIT gene mutation | PDGFRA gene mutation | BRAF kinase mutation | 14 | 餘製願繭獵構鹹鑰鏇夢(齋積範鹽憲鬱鬱餘窪繭) = 鹽鬱築壓構積窪衊遞鬱 襯衊網淵餘艱鏇衊構襯 (築繭獵廠鏇築遞壓鏇願, 72 ~ 84) 更多 | 积极 | 2026-05-02 | ||
餘製願繭獵構鹹鑰鏇夢(齋積範鹽憲鬱鬱餘窪繭) = 觸憲齋衊糧蓋膚糧廠繭 襯衊網淵餘艱鏇衊構襯 (築繭獵廠鏇築遞壓鏇願 ) 更多 | |||||||
临床2期 | 107 | 鏇顧簾壓衊蓋觸膚顧鏇 = 構壓夢衊願網鏇鏇構鑰 簾網壓遞鹹夢願衊齋繭 (範構淵鏇願鏇製積廠齋, 憲積積顧糧夢齋憲觸選 ~ 選蓋範壓顧膚衊選鏇廠) 更多 | - | 2026-04-24 | |||
临床1期 | 髓性系统性肥大细胞增多症 D816V-mutant KIT | 67 | (Indolent Systemic Mastocytosis) | 襯鬱窪築選夢選構齋範(獵網網淵壓廠醖艱夢齋) = Mean SSD and individual symptoms score improvements from baseline were sustained with ~3.5 years of follow-up. 夢網製構襯觸蓋繭廠憲 (淵衊築鬱餘構網蓋鬱糧 ) 更多 | 积极 | 2026-03-27 | |
N/A | 16 | (Gastrointestinal Stromal Tumours) | 願壓淵襯範餘鏇鏇衊窪(壓鑰蓋鹽築壓簾鹹製蓋) = 選膚願糧選繭繭鬱繭鏇 糧蓋窪窪鹹積襯糧醖鑰 (網繭築簾願衊餘蓋範鬱 ) 更多 | 不佳 | 2026-03-09 | ||
临床2期 | 髓性系统性肥大细胞增多症 D816V-mutant KIT | 226 | 窪鏇顧積夢夢憲蓋繭鹽(選構廠繭鏇蓋壓鬱膚蓋) = 構糧範憲憲餘繭製鏇構 膚壓襯選鑰襯鑰衊廠願 (醖艱顧衊獵願蓋醖繭壓 ) 更多 | 积极 | 2026-02-27 | ||
临床1期 | 髓性系统性肥大细胞增多症 KIT D816V mutation | 226 | 鹹構積構憲醖衊廠選鏇(製鹹齋齋醖夢製觸醖願) = 糧願構蓋積蓋餘願壓襯 餘艱膚範遞窪衊鏇醖壓 (構構膚淵鏇夢鹹顧艱獵, 0.7 ~ 67.2) | 积极 | 2026-02-27 | ||
临床1期 | 髓性系统性肥大细胞增多症 KIT D816V mutation | 90 | 鏇顧艱積鬱繭糧餘觸選(夢餘製構廠鹹遞夢窪艱) = 鹽遞糧襯淵衊構壓夢艱 醖淵壓糧獵願鬱憲鏇願 (淵鹹蓋襯遞憲餘夢構簾, 5.57) 更多 | 积极 | 2026-02-27 | ||
N/A | 18 | 遞獵廠鏇膚餘選鹹繭鹽(醖獵築壓廠積築鏇壓鏇) = 顧衊鹹襯窪觸憲夢壓窪 鬱選憲網鹽膚鏇壓膚遞 (憲鹽淵鬱願遞觸餘觸壓 ) | 积极 | 2026-02-27 |
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