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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

Take Action

Letter Signers as of 2/3/2016



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


  • Matt Salmon
  • Ann Kirkpatrick



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



  • Joe Courtney
  • Elizabeth Esty



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



  • Lynn Westmoreland
  • Doug Collins



  •  Bill Foster



  • Marlin Stutzman
  • Todd Young



  • Rod Blum



  • Andy Barr


  • Ralph Abraham, M.D.



  • 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



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


  • Gregg Harper



  • Blaine Luetkemeyer



  • 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



  •  Joyce Beatty
  • David Joyce
  • Steve Stivers


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


South Carolina

  • Mick Mulvaney



  • Steve Cohen
  • Stephen Fincher


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


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



  •  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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