November 24, 2015: Meeting of the Peripheral and Central Nervous System Drugs...

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标题的中文意思：[tr=transparent]2015年11月24日：外周和中枢神经系统的药物咨询委员会会议通知会议[tr=transparent]

CDER

November 24, 2015

8:00 a.m. to 5:30 p.m.

FDA White Oak Campus 10903 New Hampshire Avenue Building 31 Conference Center The Great Room (Rm. 1503) Silver Spring, Maryland

Agenda

The committee will discuss new drug application (NDA) 206031, drisapersen solution for injection, sponsored by BioMarin Pharmaceutical Inc., for the treatment of patients with Duchenne muscular dystrophy with mutations in the dystrophin gene that are amenable to treatment with exon 51 skipping as determined by genetic testing.

Meeting Materials

FDA intends to make background material available to the public no later than 2 business days before the meeting. If FDA is unable to post the background material on its Web site prior to the meeting, the background material will be made publicly available at the location of the advisory committee meeting, and the background material will be posted on FDA’s Web site after the meeting. Background material is available at: 2015 Meeting Materials, Peripheral and Central Nervous System Drugs Advisory Committee

Public Participation Information

Interested persons may present data, information, or views, orally or in writing, on issues pending before the committee.

• Written submissions may be made to the contact person on or before November 9, 2015.
• Oral presentations from the public will be scheduled between approximately 12:40 p.m. to 2:40 p.m. on November 24, 2015. Those individuals interested in making formal oral presentations should notify the contact person and submit a brief statement of the general nature of the evidence or arguments they wish to present, the names and addresses of proposed participants, and an indication of the approximate time requested to make their presentation on or before October 30, 2015.
  

Time allotted for each presentation may be limited. If the number of registrants requesting to speak is greater than can be reasonably accommodated during the scheduled open public hearing session, FDA may conduct a lottery to determine the speakers for the scheduled open public hearing session. The contact person will notify interested persons regarding their request to speak by November 2, 2015.

Webcast Information

CDER plans to provide a free of charge, live webcast of the November 24, 2015 meeting of the Peripheral and Central Nervous System Drugs Advisory Committee. While CDER is working to make webcasts available to the public for all advisory committee meetings held at the White Oak campus, there are instances where the webcast transmission is not successful; staff will work to re-establish the transmission as soon as possible. Further information regarding the webcast, including the web address for the webcast, will be made available at least 2 days in advance of the meeting at the following website: 2015 Meeting Materials, Peripheral and Central Nervous System Drugs Advisory Committee

CDER plans to post archived webcasts after the meeting, however, in cases where transmission was not successful, archived webcasts will not be available.

Contact Information

• Philip Bautista, PharmD
  Center for Drug Evaluation and Research
  Food and Drug Administration
  10903 New Hampshire Avenue
  WO31-2417
  Silver Spring, MD 20993-0002
  Phone: 301-796-9001
  Fax: 301-847-8533
  Email: PCNS@fda.hhs.gov
  

• FDA Advisory Committee Information Line
  1-800-741-8138
  (301-443-0572 in the Washington DC area)
  Please call the Information Line for up-to-date information on this meeting.
  

A notice in the Federal Register about last minute modifications that impact a previously announced advisory committee meeting cannot always be published quickly enough to provide timely notice. Therefore, you should always check the agency’s Web site and call the FDA Advisory Committee Information Line to learn about possible modifications before coming to the meeting.

Persons attending FDA’s advisory committee meetings are advised that the agency is not responsible for providing access to electrical outlets. FDA welcomes the attendance of the public at its advisory committee meetings and will make every effort to accommodate persons with disabilities. If you require accommodations due to a disability, please contact Philip Bautista at (301) 796-9001 at least 7 days in advance of the meeting.

Answers to commonly asked questions including information regarding special accommodations due to a disability, visitor parking, and transportation may be accessed at: Common Questions and Answers about FDA Advisory Committee Meetings.

FDA is committed to the orderly conduct of its advisory committee meetings. Please visit our Web site at Public Conduct During FDA Advisory Committee Meetings for procedures on public conduct during advisory committee meetings.

Notice of this meeting is given under the Federal Advisory Committee Act (5 U.S.C. app.2).

中文意思：

[tr=transparent]CDER

[tr=transparent]2015年11月24日

[tr]上午8:00到下午5:30。

[tr]美国白橡木校园 [tr]10903新罕布什尔州大道 [tr]31楼会议中心 [tr]房间的伟大的（RM。[tr]1503） [tr]马里兰州银泉，

[tr]日程

[tr=transparent]委员会将讨论新药申请（NDA）206031，注射drisapersen溶液，通过BioMarin制药公司赞助，用于治疗Duchenne型肌营养不良症患者与抗肌萎缩蛋白基因，可与51外显子跳跃通过基因测试确定的治疗基因突变。

[tr]会议材料

[tr]FDA打算背景材料提供给会议前公开不迟于2个工作日。[tr]如果FDA无法张贴在其网站的背景资料，在会议之前，背景材料将被公布在咨询委员会会议的地点，和背景材料将公布在FDA的网站后会议。[tr]背景材料是可用的：[tr]2015会议材料，外周和中枢神经系统药物咨询委员会

[tr]公众参与信息

[tr]当事人可能提出的数据，信息，或意见，口头或书面，在待决的问题在委员会。

• [tr]书面意见可在2015年11月9日或之前的联络人。
• [tr]口头报告从公众将安排约12:40下午2:40之间的2015年11月24日。[tr]在正式的口头报告感兴趣的那些人应该通知联络人和提交的证据他们想的一般性质的简短声明中，参与者的名字和地址了，和近似的时间指示请于2015年10月30日或之前他们的表现。
  

[tr]分配给每个演示文稿的时间可能是有限的。[tr]如果注册请求说大于可以合理安排计划的公开听证会议期间的数目，FDA可能通过抽签确定预定公开的听证会议发言。[tr]联系人会通知利害关系人对于他们的要求说2015年11月2日。

[tr]网络直播信息

[tr]CDER计划提供一个免费的，对中枢和外周神经系统药物咨询委员会2015年11月24日会议网上直播。[tr]而正在使网络广播中心向公众提供所有咨询委员会会议上，白橡木校园举行，有一些情况下，网络传输不成功；工作人员将重新传输尽快。[tr]对于网络的进一步信息，包括网络直播的网址，将在以下网站提供至少2天的时间在会议前：[tr]2015会议材料，外周和中枢神经系统药物咨询委员会

[tr]CDER计划后存档网播会议之后，然而，在传输没有成功的案例，存档网播将不可用。

[tr]联系方式

• [tr]菲利普包蒂斯塔，药学博士
  [tr=transparent]药物评价和研究中心
  [tr=transparent]食品和药物管理局
  [tr=transparent]10903新罕布什尔州大道
  [tr]wo31-2417
  [tr=transparent]银泉，医师20993-0002
  [tr=transparent]电话：301-796-9001
  [tr]传真：301-847-8533
  [tr=transparent]电子邮件[tr=transparent]pcns@fda.hhs.gov
  

• [tr]FDA咨询委员会信息线
  [tr=transparent]1-800-741-8138
  [tr]（301-443-0572在华盛顿地区）
  [tr]请在本次会议的最新信息，信息系。
  

[tr=rgb(194, 217, 233)]在联邦注册通知，最后一分钟的修改影响此前公布的咨询委员会会议，总是不能尽快公布够提供及时的通知。[tr=transparent]因此，你应该经常检查机构的网站和呼叫的FDA咨询委员会的信息了解可能的修改之前，会议。

[tr=transparent]参加FDA咨询委员会会议建议的机构是不负责提供电源插座的人。[tr]FDA欢迎市民参加咨询委员会会议，并将尽一切努力满足残疾人。[tr=transparent]如果你需要因残疾住宿，请联系菲利普包蒂斯塔在（301）796-9001至少在会议前7天。

[tr=transparent]一般要求包括信息关于特别住宿因残疾，访客停车问题的答案，和运输可以在：[tr]常见问题及回答FDA咨询委员会会议[tr]。

[tr]FDA致力于其顾问委员会会议有序进行。[tr]请访问我们的网站[tr=transparent]公众在FDA咨询委员会会议上进行[tr]对公众行为的过程中咨询委员会会议。

[tr]本次会议通知联邦咨询委员会法案（5 U.S.C. 2应用程序。）。

[tr]信息来源：http://www.fda.gov/AdvisoryCommittees/Calendar/ucm467180.htm

≧亚亚≦

关键是剖析ALS的病因，然后，才研制药物和治疗

轻松依旧

不明白意思