IMETELSTAT

主成分: IMETELSTAT (C148H211N68O53P13S13: 4610公克/莫耳(g/mol))

商品名: RYTELO

中文名: 尚無中文譯名

美國食品藥物管理局首次核准時間與編號: 2024.06.06 [NDA #217779]

研發廠商: GERON藥廠

IUPAC名: N-[3-[[(2S,3S,5R)-3-[[[(2S,3S,5R)-3-[[[(2S,3S,5R)-3-[[[(2S,3S,5R)-3-[[[(2S,3S,5R)-3-[[[(2S,3S,5R)-3-[[[(2S,3S,5R)-3-[[[(2S,3S,5R)-3-[[[(2S,3S,5R)-3-[[[(2S,3S,5R)-3-[[[(2S,3S,5R)-3-[[[(2S,3S,5R)-3-[[[(2S,3S,5R)-3-amino-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]amino]-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]amino]-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]amino]-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]amino]-5-(2-amino-6-oxo-1H-purin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]amino]-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]amino]-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]amino]-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]amino]-5-(2-amino-6-oxo-1H-purin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]amino]-5-(2-amino-6-oxo-1H-purin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]amino]-5-(2-amino-6-oxo-1H-purin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]amino]-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]amino]-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]oxy-2-hydroxypropyl]hexadecanamide 

分子線性簡式(SMILES): CCCCCCCCCCCCCCCC(=O)NCC(COP(=S)(O)OCC1C(CC(O1)N2C=C(C(=O)NC2=O)C)NP(=S)(O)OCC3C(CC(O3)N4C=NC5=C(N=CN=C54)N)NP(=S)(O)OCC6C(CC(O6)N7C=NC8=C7N=C(NC8=O)N)NP(=S)(O)OCC9C(CC(O9)N1C=NC2=C1N=C(NC2=O)N)NP(=S)(O)OCC1C(CC(O1)N1C=NC2=C1N=C(NC2=O)N)NP(=S)(O)OCC1C(CC(O1)N1C=C(C(=O)NC1=O)C)NP(=S)(O)OCC1C(CC(O1)N1C=C(C(=O)NC1=O)C)NP(=S)(O)OCC1C(CC(O1)N1C=NC2=C(N=CN=C21)N)NP(=S)(O)OCC1C(CC(O1)N1C=NC2=C1N=C(NC2=O)N)NP(=S)(O)OCC1C(CC(O1)N1C=NC2=C(N=CN=C21)N)NP(=S)(O)OCC1C(CC(O1)N1C=CC(=NC1=O)N)NP(=S)(O)OCC1C(CC(O1)N1C=NC2=C(N=CN=C21)N)NP(=S)(O)OCC1C(CC(O1)N1C=NC2=C(N=CN=C21)N)N)O 

公共化合物資料庫(PubChem): 

• Imetelstat: 72941969/CAS編號: 868169-64-6

英文仿單: U.S. FDA英文仿單 ORIG-1

給藥途徑: 靜脈輸注(intravenous infusion)

劑量: 45 mg凍乾粉；188 mg凍乾粉 [根據體重自冰箱中取出合適劑量的瓶數，靜置回溫10~15分鐘後，與符合美國藥典標準的1.8毫升或6.3毫升0.9%氯化鈉注射液重構為每瓶濃度31.4 mg/mL，再轉移1.5毫升或6毫升重構液至含有500毫升符合美國藥典標準的0.9%氯化鈉注射液的靜脈注射袋中稀釋，溫和翻轉靜脈注射袋至少五次，確保含有合適劑量的IMETELSTAT混和均勻後輸注]

機制: IMETELSTAT可藉由與人類端粒酶RNA構件(telomerase RNA component)的模板區(template region)結合，抑制端粒酶活性並阻止與端粒結合，使端粒酶常表現增加的骨髓增生不良症候群(MDS, myelodysplastic syndromes)、惡性幹細胞與前驅細胞等的端粒因而縮短長度、減少增生與誘發細胞凋亡(apoptosis)。

適應症:

1. 血液淋巴惡性腫瘤|骨髓增生不良症候群(MDS, myelodysplastic syndromes):

用於治療成人伴有造血刺激劑(erythropoiesis-stimulating agents)無效或不合適之8週需要4個以上紅血球單位的輸血依賴性貧血(transfusion-dependent anemia)之低中度風險(low- to intermediate-1 risk)的骨髓增生不良症候群(MDS, myelodysplastic syndromes)；

潛在靶點與作用機轉: 整理中，敬請期待。 

中華民國藥品許可與健保價格(中文的用法、劑量、副作用等資訊詳見各連結內之仿單資料連結): 食品藥物管理署尚未核准IMETELSTAT