Speak Up for the Duchenne Community!

After waiting for a very long time, the Duchenne community now has clarity on the Advisory Committee meetings dates for BioMarin and Sarepta.  BioMarin’s date is November 24, 2015 and Sarepta’s date is January 22, 2016.  Please remember, these dates are tentative until they actually appear on the Federal Registry.  Both of the Ad Comm dates are approximately 30 days prior to the PDUFA date, or the date by which the FDA is committed to announce its decision on approval of the drug.

CureDuchenne has been working actively with the companies to assist them as they prepare for their respective Ad Comm meetings, and we have felt that it was prudent to wait until an announcement of the dates to discuss this publically.  As the early supporter of drisapersen’s development and an early contributor to the development of eteplirsen, we are anxious and ready to hopefully see these drugs approved.

CureDuchenne has overcome many obstacles during this last decade to bring exon skipping to this point, and we will do everything possible to see that our loved ones will have a treatment soon.

Here is the important information you should know about the Ad Comm:

The public advisory committee meeting of the Food and Drug Administration (FDA) to discuss BioMarin’s drisapersen will be held on November 24 in Silver Spring, MD from 8 a.m. to 5:30 p.m. The meeting is open to the public. Tomorrow the document will be published in the Federal Register.

The committee will discuss the new drug application for drisapersen for the treatment of patients with Duchenne muscular dystrophy that is amenable to treatment with exon 51 skipping.

Share your story and your wishes with the FDA. Tell them how important it is for the Duchenne community to have approved treatments for this devastating disease.

Key dates:

October 30, 2015 – deadline to submit request to make a formal oral presentation. Those interested in making a formal oral presentation should submit a brief statement of the general nature of what they wish to present, the names and address of proposed participants and the approximate amount of time requested to make their presentation to the contact person listed below.

November 2, 2015 – FDA contact will notify interested persons regarding their request to speak.

November 9, 2015 – deadline for written submission may be made to the contact person listed below.

November 24, 2015 – Ad Comm meeting.  Location: MD. FDA White Oak Campus, 10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great Room (rm. 1503), Silver Spring, MD 20993-0002. Oral presentations from the public will be scheduled between approximately 12:40 p.m. and 2:40 p.m.

Contact Person: Philip Bautista, Center for Drug Evaluation and Research, Food and

Drug Administration, 10903 New Hampshire Ave., Bldg. 31, Rm. 2417, Silver Spring, MD 20993-0002, 301-796-9001, FAX: 301-847-8533, PCNS@fda.hhs.gov, or FDA Advisory Committee Information Line, 1-800-741-8138 (301-443-0572 in the Washington, DC area).

Answers to commonly asked questions including information regarding special accommodations due to a disability, visitor parking, and transportation may be accessed at: http://www.fda.gov/AdvisoryCommittees/AboutAdvisoryCommittees/ucm408555.htm.

FDA is committed to the orderly conduct of its advisory committee meetings. Please visit http://www.fda.gov/AdvisoryCommittees/AboutAdvisoryCommittees/ucm111462.htm for procedures on public conduct during advisory committee meetings

For more information about attending the Ad Comm or providing written testimony, please contact katie@cureduchenne.org or call 949-872-2552.