赛诺菲（Sanofi）及旗下健赞（Genzyme）4月7日宣布，经过与FDA之间富有建设性的讨论，计划于第二季度重新向FDA提交单抗药物Lemtrada（alemtuzumab，阿伦单抗）的补充生物制品许可申请（sBLA），寻求批准该药用于复发型多发性硬化症的治疗。此次sBLA的提交，将提供对FDA于2013年12月27日完整回应函（CRL）中所指出问题的回复。
去年12月，FDA审查后发现，Lemtrada可能会导致系列自身免疫性疾病并潜在引发甲状腺癌、皮肤癌、乳腺癌，因此对该药的安全性问题表达了严重关注，并认为赛诺菲需要进一步补充III期临床数据，以证明该药的安全性及疗效。而FDA顾问委员会则认为，Lemtrada的安全性问题，并不妨碍该药的批准，但对相关临床研究的质量表达了担忧。
Lemtrada是一种单克隆抗体，靶向于CD52，这是一种位于T细胞及B细胞上的蛋白。Lemtrada被认为能够选择性消耗T细胞和B细胞，同时保留免疫系统中的其他抗感染成分，通过抗炎作用，有助于再次平衡免疫系统。
Lemtrada已于2013年9月获欧盟批准，用于活动性复发缓解型多发性硬化症（RRMS）成人患者的治疗。如果获FDA批准，Lemtrada将与百健艾迪（Biogen Idec）的Tysabri展开激烈竞争，该药是市面上最有效的多发性硬化症（MS）治疗药物，但可能引发一种潜在致死的脑部感染，即进行性多灶性白质脑病(PML)。这种风险，已将Tysabri的使用限制至经其他MS药物治疗失败的患者群体，若Lemtrada获批，也可能被限制至这一患者群体。根据汤姆森路透数位分析师预测，若Lemtrada获FDA获批，预计截止2018年的销售峰值仅为7.52亿美元。
Sanofi: Genzyme to Resubmit Lemtrada Application for FDA Review
PARIS, April 7, 2014 (GLOBE NEWSWIRE) -- Sanofi (EURONEXT: SAN and NYSE: SNY) and its subsidiary Genzyme announced today that following constructive discussions with the U.S. Food and Drug Administration (FDA) the company plans to resubmit in the second quarter its supplemental Biologics License Application (sBLA) seeking approval of Lemtrada (alemtuzumab) for the treatment of relapsing forms of multiple sclerosis. The resubmission will provide information to specifically address issues noted by the FDA in its December 27, 2013 Complete Response Letter.
Genzyme had previously announced its intention to appeal the FDA's Complete Response Letter. In light of the planned resubmission, the company does not expect to pursue an appeal at this time.
ab0ut Genzyme, a Sanofi Company
Genzyme has pioneered the development and delivery of transformative therapies for patients affected by rare and debilitating diseases for over 30 years. We accomplish our goals through world-class research and with the compassion and commitment of our employees. With a focus on rare diseases and multiple sclerosis, we are dedicated to making a positive impact on the lives of the patients and families we serve. That goal guides and inspires us every day. Genzyme's portfolio of transformative therapies, which are marketed in countries around the world, represents groundbreaking and life-saving advances in medicine. As a Sanofi company, Genzyme benefits from the reach and resources of one of the world's largest pharmaceutical companies, with a shared commitment to improving the lives of patients.
Genzyme holds the worldwide rights to alemtuzumab and has primary responsibility for its development and commercialization in multiple sclerosis. Bayer HealthCare holds the right to co-promote alemtuzumab in MS in the United States. Upon commercialization, Bayer will receive contingent payments based on global sales revenue.
Genzyme(R) is a registered trademark and Lemtrada is a trademark of Genzyme Corporation.
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