Macugen是目前美国FDA批准用于治疗年龄相关性黄斑变性的唯一的抗血管内皮生长因子的适体。临床试验显示,该药玻璃体内注射后具有一定疗效,副作用较少。 美国FDA批准用于治疗视网膜老年黄斑病变（AMD）的新药Macugen（商品名；通用名：pegaptanib sodium[哌加他尼钠]）注射液  12月20日，FDA宣布批准哌加他尼钠注射液（Macugen）用于治疗眼科疾病新生血管型（湿性）老年黄斑病变（Age-Related Macular Degeneration，AMD）患者的视力缓慢丧失。哌加他尼钠是一种选择性血管内皮生产因子（vascular endothelial growth factor，VEGF）拮抗剂。 “Macugen是靶向湿性AMD的生物机制（underlying biology）的第一类治疗药物之一，”FDA代理局长Lester M. Crawford博士说。“Macugen为该病患者提供了另一种所需的治疗手段。” AMD是一种导致严重和不可逆的视力丧失的视网膜疾病，是造成55岁以上人群失明的一个主要原因。如不予治疗，大多数受湿性AMD影响的眼睛可能恶化为机能性失明。湿性AMD占AMD的近10％，其病因是异常渗漏性血管的生长最终损害了负责中心视力的眼底区域，而中心视力是大多数精细视觉活动所必需的，包括阅读、驾驶和认人。两项关于Macugen的安全性和有效性的临床试验在湿性AMD患者中开展了两年。在这两项临床试验中，接受Macugen治疗的患者发生视力丧失的人数显著减少。与注射过程相关的严重不良事件包括感染、视网膜脱落和创伤性白内障。以Macugen治疗的患者中其它常被报告的不良事件是眼刺激、眼痛、眼结膜下出血和视力模糊。 Macugen的新药申请被收到后在6个月内得到批准。 MACUGEN 0,3MG 1 JERINGA PRECARGADA SOLUCION INYECTABLE 90 MICROLITROS PFIZER, S.A. General Information Other Useful Resources Macugen at Drugs.com Macugen at RX-List Web Search for Macugen Wikipedia Search for Macugen Macugen (pegaptanib) is a selective vascular endothelial growth factor (VEGF) antagonist. Macugen is indicated for the treatment of neovascular (wet) age-related macular degeneration. According to the present product label, Macugen (0.3 mg) should be administered once every six weeks by intravitreous injection into the eye to be treated. There are 15 million people in the United States living with some form of AMD, with more than 1.6 million experiencing the active blood vessel growth and blood vessel leakage associated with neovascular AMD. -------------------------------------------------------------------------------- Clinical Results FDA approval of Macugen was based on results from two controlled, double-masked, randomized trials (study EOP1003 & EOP1004) enrolling a total of 1,200 patients with neovascular AMD. Subjects received 0.3 mg, 1 mg or 3 mg if Macugen by injection or a control (sham) treatment every 64 days for 48 weeks. The median age of the patients was 77 years. All subjects enrolled had classic, occult, or mixed lesions of up to 12 disc areas and baseline visual acuity in the study eye between 20/40 and 20/320. Results showed that treatment with Macugen demonstrated statistically significant less vision loss compared to sham treatment. Results showed that among patients receiving 0.3 mg of Macugen, 70% lost less than three lines of vision on the eye chart, compared with 55% of patients receiving control treatment (P<.0001). The results demonstrated a 27% relative treatment effect for Macugen treated patients compared to controls with respect to three line loss. Macugen also helped limit progression to legal blindness, by 50 % compared to controls, in the study eye.More sham treated patients (75/296) received PDT than Macugen 0.3 mg treated patients (58/294). On average, Macugen 0.3 mg treated patients and sham treated patients continued to experience vision loss. The rate of vision decline in the Macugen treated group was slower than the rate in the patients who received sham treatment. In both studies, the primary endpoint was the proportion of patients losing less than 15 letters of visual acuity, from baseline up to 54 week assessment. -------------------------------------------------------------------------------- Side Effects Adverse events associated with the use of Macugen may include (but are not limited to) the following: Ocular Discomfort Eye Pain Endophthalmitis Reduced Visual Acuity Visual Disturbance Corneal Edema Blurred Vision Dizziness -------------------------------------------------------------------------------- Mechanism of Action Pegaptanib is a selective vascular endothelial growth factor (VEGF) antagonist. VEGF is a secreted protein that selectively binds and activates its receptors located primarily on the surface of vascular endothelial cells. VEGF induces angiogenesis, and increases vascular permeability and inflammation, all of which are thought to contribute to the progression of the neovascular (wet) form of age-related macular degeneration (AMD), a leading cause of blindness. -------------------------------------------------------------------------------- Literature References Bayes M, Rabasseda X, Prous JR. Gateways to clinical trials. Methods Find Exp Clin Pharmacol. 2002 Nov;24(9):615-43. Gragoudas ES, Adamis AP, Cunningham ET Jr, Feinsod M, Guyer DR; Pegaptanib for neovascular age-related macular degeneration. N Engl J Med. 2004 Dec 30; 351(27):2805-16. Vinores SA. Technology evaluation: pegaptanib, Eyetech/Pfizer. Curr Opin Mol Ther. 2003 Dec; 5(6):673-9. -------------------------------------------------------------------------------- Additional Information For additional information regarding Macugen or macular degeneration, please visit The Macugen Web Site Macugen治疗糖尿病视网膜病变的有效性 近期在视力和眼病研究协会的年会上，一项II期临床试验结果表明，Macugen（一种血管内皮生长因子类似物）在治疗糖尿病视网膜病变方面有广泛的应用前景。 172名平均年龄为62岁的入组糖尿病患者临床上均明显的视网膜增厚，至少有半个区域及黄斑中心的受累。眼底荧光造影和摄片均可见眼底有渗出性表现。将患者随机分为Macugen 0.3mg治疗组（44人）、1.0mg治疗组（44人）、3.0mg治疗组（42人）及安慰剂对照组（42人）。四组在视力、视网膜厚度、病程、糖尿病类型和严重程度及其他并发症方面均具有可比性。结果发现，0.3mg和1.0mg治疗组中分别约有60%和45%的患者至少能看见视力表上的一行。3.0mg治疗组和安慰剂组中约有30%的患者视力有了一行以上的进步。此外，0.3mg治疗组中有49%的患者视网膜厚度变薄了75纳米，该比例在1.0mg治疗组中为28%。两组患者视网膜厚度变薄的平均值分别为68纳米和23纳米。各剂量治疗组中需要激光治疗的患者人数明显少于安慰剂组。Macugen的副作用较少见，有1例患者发生了内眼炎（0.2%），另有5例患者发生了玻璃体出血（5%）。未发现新发白内障或原有白内障加重的患者。研究人员指出，与常规治疗相比，36周的Macugen治疗能有效改善糖尿病视网膜病变患者的视力，视网膜厚度有明显的变薄，可安全有效地用于糖尿病视网膜病变的治疗。