Industry News: FDA Announces Advisory Committee Meeting to Review Ataluren.

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The Food and Drug Administration (FDA) has published the date, location, and time of the Central and Peripheral Nervous System (PCNS) Advisory Committee Meeting to review ataluren and request for public comments to the Federal Register. The Advisory Committee will discuss  ataluren's new drug application by PTC Therapeutics, for the treatment of patients with Duchenne muscular dystrophy due to a nonsense mutation in the dystrophin gene. To learn more about what an advisory committee is and the role it plays in drug development, please see FDA's website. 

 

Date: September 28, 2017

Time: 9:00am-4:30pm EST

Location: Tommy Douglas Conference Center

The Ballroom

10000 New Hampshire Ave.

Silver Spring, MD 209903

There are two ways that patients and families affected by Duchenne can participate in ataluren's AdComm.

 

1.    WRITTEN TESTIMONY

  • Written testimony from patients and families affected by Duchenne, especially those with experience with ataluren may be submitted to the docket electronically or by mail on or before September 27, 2017. Comments submitted prior to September 14, 2017 will be shared with the Advisory Committee Members.

 

  • Electronic submission can be submitted via the Federal eRulemaking Portal: https://www.regulations.gov. Follow the instructions for submitting comments. Comments submitted electronically, including attachments, to https://www.regulations.gov will be posted to the docket unchanged. Because your comment will be made public, you are solely responsible for ensuring that your comment does not include any confidential information that you or a third party may not wish to be posted, such as medical information, your or anyone else’s Social Security number, or confidential business information, such as a manufacturing process. Please note that if you include your name, contact information, or other information that identifies you in the body of your comments, that information will be posted on https://www.regulations.gov.

 

  • If you want to submit a comment with confidential information that you do not wish to be made available to the public, submit the comment as a written/paper submission and in the manner detailed (see “Written/Paper Submissions” and “Instructions”).

 

  • All submissions received must include the Docket No. FDA-2017-N-4835 for “Peripheral and Central Nervous System Drugs Advisory Committee; Notice of Meeting; Establishment of a Public Docket; Request for Comments.” Received comments, those filed in a timely manner (see ADDRESSES), will be placed in the docket and, except for those submitted as “Confidential Submissions,” publicly viewable at https://www.regulations.gov or at the Dockets Management Staff between 9 a.m. and 4 p.m., Monday through Friday.

 

  • Confidential Submissions--To submit a comment with confidential information that you do not wish to be made publicly available, submit your comments only as a written/paper submission. You should submit two copies total. One copy will include the information you claim to be confidential with a heading or cover note that states “THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.” The Agency will review this copy, including the claimed confidential information, in its consideration of comments. The second copy, which will have the claimed confidential information redacted/blacked out, will be available for public viewing and posted on https://www.regulations.gov. Submit both copies to the Dockets Management Staff. If you do not wish your name and contact information to be made publicly available, you can provide this information on the cover sheet and not in the body of your comments and you must identify this information as “confidential.” Any information marked as “confidential” will not be disclosed except in accordance with 21 CFR 10.20 and other applicable disclosure law. For more information about FDA’s posting of comments to public dockets, see 80 FR 56469, September 18, 2015, or access the information at: https://www.gpo.gov/fdsys/pkg/FR-2015-09-18/pdf/2015- 23389.pdf.

 

2.    ORAL TESTIMONY

  • During the AdComm meeting, the FDA has scheduled time between 1:15 and 2:15 for patients, families, and other interested members of the public to provide public testimony. This is called the Open Public Hearing (OPH). For those of us in the Duchenne patient community, OPH is an opportunity to provide FDA with the perspective of patients and families affected by Duchenne as it relates to ataluren.

 

  •  Parent Project Muscular Dystrophy is working to organize those in the Duchenne community’s public testimony in an attempt to avoid a speaker slot lottery and that messages are not duplicated and are impactful. For those interested in collaborating, please contact Ryan Fisher at PPMD ryan@parentprojectmd.org to speak about the OPH portion of AdComm and learn about travel support.

 

  • Other individuals interested in speaking during OPH should contact Moon Hee V. Choi at PCNS@fda.hhs.gov and submit a brief statement on the general nature of their testimony, the names and addresses of all speakers, the amount of time needed on or before September 6, 2017. You will be notified regarding your request to speak by September 7, 2017.

 

BACKGROUND MATERIALS

FDA will publish background material 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 http://www.fda.gov/AdvisoryCommittees/Calendar/default.htm. Scroll down to the appropriate advisory committee meeting link.

 

SPECIAL ACCOMODATIONS

If you require special accommodations due to a disability, please contact Moon Hee V. Choi at least 7 days in advance of the meeting at PCNS@fda.hhs.gov or 301-796-9001.

 

ADDITIONAL QUESTIONS & CONCERNS

Contact Moon Hee V. Choi

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)