2015年3月30日讯 /生物谷BIOON/ --自古以来，民告官就是难于上青天。许多人面对“有关部门”滥用权力时都选择了息事宁人，正所谓是做人留一线，日后好相见。而FDA作为在美国生物医药领域负责审批药物临床研究和上市事宜的最高机构，在众多生物医药公司看来可谓是执掌了生杀大权。
不过，较真的日本人最近还真就把FDA告上了法庭。消息显示，日本Otsuka Pharmaceuticals公司最近在美国联邦地方法院起诉FDA滥用监管权，损害了旗下Abilify的市场权益，最终决定和FDA对簿公堂。
说来这一案件也够离奇，一般而言，对于生物医药公司来说，当他们的药物上市时候，为了扩大销量唯恐FDA所批准的适应症不够多，覆盖的患者群体不够广。而在这起案件中，Otsuka Pharmaceuticals却希望这一药物的适用症范围尽可能限制在一定人群中。
原来，此次双方面临的焦点在于Otsuka公司开发的Abilify此前被批准应用于治疗妥瑞症、躁郁症以及精神分裂症。这种药物已经上市，就为Otsuka公司带来超过650万美元的收入。不过相关资料显示，Abilify将于今年四月20日失去专利保护，并将面临仿制药的挑战。可以预想，Abilify的销售额必将大受影响。
不过，Otsuka公司并不希望轻易放弃公司的这棵摇钱树。Otsuka公司表示由于Abilify此前是作为罕见疾病妥瑞症的治疗方案申请上市的，因此Abilify应当享受孤儿药所具有的一切优惠措施。这就意味着Abilify的排他性经营权将再延长7年之久。
然而，FDA却于最近将Abilify的适用范围扩大至图雷特综合征（Tourette syndrome ，TS是一种相对普通的神经紊乱，特征是无意识地快速抽搐，突然移动，或者发出声音，并且以相同的方式重复）的治疗，根据相关规定，这也意味着Abilify将无法获得延长的排他性经营权。
Otsuka公司起诉FDA在缺乏相关临床数据的基础上，未能充分考虑到Abilify对图雷特综合征患者的安全性和有效性等方面就做出了这一决定，涉及滥用其审批权利，因而请求法院驳回FDA的这一决定。（小编想问一句，是不是你们拿错剧本了？）
Otsuka公司与FDA的这一争执目前为止尚属罕见。不过，无论结果如何，小编窃以为这样的做法必将有利于规范生物医药企业以及医药管理机构双方的行为规范。这种不畏公共权力，敢于一切从法律角度出发和行政部门“叫板”的精神值得国内的管理机构和医药行业从业者学习。
详细英文报道：
Japan's Otsuka Pharmaceuticals accused the U.S. FDA in a unique court suit of manipulating its regulatory procedures to kill off its 7-year exclusivity to market Abilify (aripiprazole) for treating Tourette syndrome in children.
Under normal circumstances, a drugmaker wants as broad an indication or as many of them as possible to widen the scope of possible sales. In this case, Otsuka wants to keep its narrow, pediatric indication.
Otsuka filed the suit in a federal district court, which could issue an injunction soon, seeking to reverse the FDA's action, which the Japan-based company called "arbitrary, capricious and unlawful abuse of its authority." Up to $45.5 million could be hanging on the suit's outcome.
Otsuka obtained 7-year exclusivity to market Abilify in December under the FDA's orphan drug procedures, based on the Tourette pediatric indication. Up to now, Abilify had been earning Otsuka $6.5 million a year as one of the leading antipsychotic drugs, including treatment for bipolar disorder and schizophrenia.
In an almost Kafkaesque twist and why Otsuka is upset, Abilify's patent protection is set to expire April 20, opening it to generic competition. The orphan drug approval for treating Tourette's was Otsuka's way of keeping Abilify on the market as well as its new indication for an additional 7 years without a generic challenge.
That is what Otsuka saw as the drug's near future until February, when the agency added what Otsuka might consider a Machiavellian twist. Apparently, the FDA stepped in and decided to lift the restriction of the new indication to pediatric use and give it a new indication for treating Tourette's at all ages. That had the effect, Otsuka argued in its suit, of eliminating the 7-year exclusivity intended for pediatric use, and thus opening that indication to generic competition next month.
The argument Otsuka laid out in the suit and what the court must determine is whether the agency can approve Abilify for a broader segment of the population when no clinical trials have been conducted to test its safety and effectiveness in treating Tourette's in those age groups.