拜耳/Regeneron糖尿病性视网膜病变药Eylea获FDA突破性疗法认定

拜耳/Regeneron糖尿病性视网膜病变药Eylea获FDA突破性疗法认定

                               
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发布日期：2014-09-18  来源：生物谷

Eylea有望成为首个糖尿病性视网膜病变（DR）治疗药物，上月Eylea获FDA和欧盟批准用于糖尿病性黄斑水肿（DME），是该药的第3个适应症。

                               
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拜耳（Bayer）和合作伙伴Regeneron制药近日宣布，FDA已授予眼科药物Eylea（aflibercept，阿柏西普注射液）治疗糖尿病性黄斑水肿（DME）患者糖尿病性视网膜病变（DR）的突破性疗法认定。该突破性疗法的授予，是基于2项III期研究（VIVID-DME和VISTA-DME）的积极数据，这些研究中，经过2年治疗后，Eylea使DME患者DR病情得到了统计学意义的显著改善。

目前，尚未有任何药物批准用于治疗糖尿病性视网膜病变（DR），如果获批，Eylea将成为首个DR治疗药物。拜耳和Regeneron计划在今年晚些时候提向FDA提交Eylea治疗糖尿病性黄斑水肿（DME）患者糖尿病性视网膜病变（DR）的补充生物制品许可（sBLA），并期待与FDA密切合作，使Eylea尽快上市造福患者。

“突破性疗法”认定，旨在加快某些重症治疗新药的开发和审评。目前，“突破性疗法”新药研发正呈上升势头，已有多个“突破性疗法”认定药物获得FDA批准上市。

Eylea已于今年7月底和8月初分别获FDA和欧盟批准，用于糖尿病性黄斑水肿（DME）的治疗，这是Eylea获批的第3个适应症。此前，Eylea已获美国、欧盟及其他国家批准，治疗湿性年龄相关性黄斑变性（wet-AMD）、视网膜中央静脉阻塞（CRVO）的治疗。此外，拜耳和Regeneron已向FDA和欧盟提交了Eylea治疗视网膜分支静脉阻塞（BRVO）继发黄斑水肿（ME）的上市申请，这也代表着Eylea的第4个新适应症申请。

Eylea是一种新型玻璃体内注射用VEGF抑制剂，是一种重组融合蛋白，由人体血管内皮细胞生长因子（VEFG）受体1和2的胞外区与人体免疫球蛋白G1的可结晶片段融合而成。

Eylea作为VEGF家族各成员（包括VEGF-A）及胎盘生长因子（PIGF）的一种可溶性诱饵受体发挥作用，与这些因子具有极高的亲和力，从而抑制这些因子与同源VEGF受体的结合，因此Eylea可抑制异常的血管生成及渗漏。

目前，拜耳和Regeneron正在合作Eylea的全球开发。Regeneron保留Eylea在美国的独家权利，拜耳则授权获得该药在美国以外国家和地区的独家销售权，这2家公司将平分Eylea在未来销售的利润。

英文原文：FDA grants Breakthrough Therapy designation to EYLEA Injection for treatment of diabetic retinopathy

Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the U.S. Food and Drug Administration (FDA) has granted EYLEA® (aflibercept) Injection Breakthrough Therapy designation for the treatment of diabetic retinopathy in patients with diabetic macular edema (DME). The designation is based on positive results in two Phase 3 trials (VIVID-DME and VISTA-DME), in which EYLEA demonstrated a statistically significant improvement in a pre-specified measure of diabetic retinopathy in patients with DME after two years of treatment.

"Millions of people in the U.S. are living with diabetic eye diseases that can cause vision loss and even blindness," said George D. Yancopoulos, M.D., Ph.D., Chief Scientific Officer of Regeneron and President of Regeneron Laboratories. "There are no FDA-approved medicines for diabetic retinopathy and we look forward to working closely with the FDA to potentially bring EYLEA to these patients as soon as possible. We plan to submit a supplemental Biologics License Application (sBLA) in the U.S. for diabetic retinoapthy in patients with DME later this year."

In the Phase 3 VIVID-DME trial in diabetic retinopathy patients with DME, 29 percent of evaluable patients in the 2Q4 group (monthly) and 33 percent of evaluable patients in the 2Q8 group (every two months, after 5 initial monthly injections) treated with EYLEA experienced at least a 2-step improvement on the diabetic retinopathy severity scale (DRSS), a grading system measuring the degree of retinopathy, compared to 8 percent of patients in the laser control group (p less than 0.001). In the Phase 3 VISTA-DME trial in diabetic retinopathy patients with DME, 40 percent of evaluable patients in both the 2Q4 and 2Q8 groups treated with EYLEA experienced at least a 2-step improvement on the DRSS compared to 17 percent of patients in the laser control group (p less than 0.0001). The most frequent ocular adverse events (AEs) observed in the VIVID-DME trial were conjunctival hemorrhage, cataract, and increased intraocular pressure. The most frequent ocular AEs observed in the VISTA-DME trial were conjunctival hemorrhage, eye pain, and vitreous floaters. The two-year results from these trials on the primary endpoint of best-corrected visual acuity (BCVA) and overall safety have been previously announced and are available here.

The Breakthrough Therapy designation was created by the FDA to expedite the development and review of drugs for serious or life-threatening conditions. Drugs qualifying for this designation must show credible evidence of a substantial improvement on a clinically significant endpoint over available therapies, or over placebo if there is no available therapy. The designation includes all of the fast track program features, as well as more intensive FDA guidance and discussion. The Breakthrough Therapy designation is distinct from both accelerated approval and priority review, which can also be granted to the same drug if relevant criteria are met.

EYLEA is approved in the United States, European unio (EU) and other countries for the treatment of wet age-related macular degeneration (AMD), macular edema following central retinal vein occlusion (CRVO), and DME. Regulatory submissions have been made for EYLEA in the U.S. and EU for macular edema following branch retinal vein occlusion (BRVO).

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