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ACTION ALERT: Sign-On Extended! Urge House Member - Apply FDASIA Tools to Duchenne

Action Alert

PPMD and a coalition of Duchenne foundations continue to work together on preparing for the Sarepta Ad Comm. As of today, we are still waiting on a new confirmed date. With the postponement of the Ad Comm, the Congressional letter to FDA remains open. Has your House Members signed on yet? Below is the list of updated signatures. If your House member is not on the list, please reach out today.  If you do see your member listed please send them a thank you. If you have questions please reach out to Ryan Fischer

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Letter Signers as of 2/3/2016

 

Alabama

  • Bradley Byrne
  • Terri Sewell
  • Mike D. Rogers
  • Gary Palmer
  • Mo Brooks
  • Martha Roby


Arizona

  • Matt Salmon
  • Ann Kirkpatrick

 

California

  • Lucille Roybal-Allard
  • Ted Lieu
  • Eric Swalwell
  • Mark DuSaulnier

 

Connecticut

  • Joe Courtney
  • Elizabeth Esty

 

Florida

  • Ted Deutch
  • Richard Nugent
  • Ander Crenshaw
  • Alcee L. Hastings
  • Gus Bilirakis
  • Ileana Ros-Lehtinen

 

Georgia

  • Lynn Westmoreland
  • Doug Collins

 

Illinois

  •  Bill Foster

 

Indiana

  • Marlin Stutzman
  • Todd Young

 

Iowa

  • Rod Blum

 

Kentucky

  • Andy Barr

Louisiana

  • Ralph Abraham, M.D.

 

Maine

  • Bruce Poliquin
  • Chellie Pingree
  • Massachusetts
  • Bill Keating
  • Michael Capuano
  • Stephen F. Lynch
  • James P. McGovern
  • Niki Tsongas
  • Michigan
  • Brenda L. Lawrence
  • Mike Bishop
  • John Conyers

 

Minnesota

  • Erik Paulsen
  • Collin C. Peterson
  • Tim Walz
  • John Kline


Mississippi

  • Gregg Harper

 

Missouri

  • Blaine Luetkemeyer

 

Nebraska

  • Brad Ashford

 

New Jersey

  • Scott Garrett
  • Christopher Smith
  • Leonard Lance

 

New York

  • Peter King
  • Daniel M. Donovan, Jr
  • Brian Higgins
  • Grace Meng
  • Charles Rangel

 

North Carolina

  • Patrick McHenry

 

North Dakota

  • Kevin Cramer

 

Ohio

  •  Joyce Beatty
  • David Joyce
  • Steve Stivers


Pennsylvania

  • Tom Marino
  • Ryan A. Costello
  • Mike Doyle
  • Lou Barletta
  • Robert Brady
  • Mike Kellly

 

South Carolina

  • Mick Mulvaney

 

Tennessee

  • Steve Cohen
  • Stephen Fincher


Texas

  • Bill Flores
  • Pete Sessions
  • Ted Poe
  • John Culberson
  • Roger Williams
  • Joe Barton
  • Al Green
  • Randy Weber
  • Beto O’Rourke


Washington

  • Adam Smith
  • Derek Kilmer
  • Rick Larsen
  • Denny Heck

 

Wisconsin

  •  Ron Kind

About our Congressional letter to FDA


There has never been a moment like this in our Duchenne community. Today the FDA released briefing documents on the application for eteplirsen. It is clear that we continue to face a serious regulatory challenge as a community.

The clinical and research community have accelerated therapy development to our first-ever product reviews. Congress has ensured that regulators have the proper resources and authorities to review these products. Our families have spent years participating in the natural history studies and clinical trials needed to generate evidence of these products safety and efficacy profiles. And our Duchenne community has helped build a regulatory infrastructure complete with regulatory guidance and quantifiable patient preference data.

Please reach out to your Representative and urge them to sign on to the congressional letter being circulated by Representatives Fitzpatrick, Keating, and King on the Hill in support of the FDA’s continued use of the employment of the tools Congress included in the Food and Drug Administration Safety and Innovation Act (FDASIA) and the broad flexibility referenced in the Code of Federal Regulations Title 21.

This letter underscores FDASIA’s focus on accelerating the approval of drugs that treat unmet medical needs, prioritizing the patient perspective in evaluating new drugs and treatments, and providing regulators with flexibility when evaluating drugs for a life-threatening illness. Our community is heartened to know that our congressional champions remain committed to ensuring the FDA has the tools, authorities, and latitude necessary to review and clear safe, effective treatments for rare disease as quickly as possible.

Please urge your members to join by signing on and asking that the agency fully utilize these tools and authorities, especially where there is a favorable benefit-risk equation, to provide patients and physicians with new options to treat rare and deadly diseases like Duchenne.


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