WASHINGTON DC IPLEX UPDATE

March 12-13, 2009

Non-stop meetings with the Health Directors of several Representatives and Senators over a two and a half-day period produced some important information and potential action that might provide some help to our cause.  Health Policy Directors and Legislative Assistants for Congressmen John Fleming of Louisiana and Frank Pallone Jr. of New Jersey; Senators Harry Reid of Nevada, Herb Kohl of Wisconsin, Dianne Feinstein of California, Orrin Hatch of Utah, Michael Enzi of Wyoming, Richard Durbin of Illinois, Debbie Stabenow of Michigan, Arlen Specter of Pennsylvania and Barbara Boxer of California all expressed concern about the difficulties the ALS community has encountered with the FDA in securing Iplex.   Some were vociferous in their distress and plan to take whatever immediate action is possible on our behalf.  Other personnel sympathetic to our needs agreed to take the matter under advisement and to present it to their Senator or Congressman in detail, but could not commit to any specific action open to them. 

We learned that the House of Representatives has little power over the FDA.  It’s possible that a meeting might be arranged between ALS WORLDWIDE, patients and their families and the FDA.  We encouraged them to pursue that plan and assured them we would be glad to meet as soon as possible.  We also learned the Senate has far greater influence over the FDA, but could not likely influence any major policy change.  It appears very possible that some of the more influential Senators will call those that can influence the FDA on our behalf. 

And while the FDA did make an announcement on Monday, March 10, that Iplex would be made available to ALS patients, the truth behind that statement is that a mere 20 patients who filed IND/IRB’s by March 6 (in contradiction to an earlier FDA announcement that all IND/IRB’s would be rejected) will have access to Iplex AFTER they re-file their applications incorporating apparently irrelevant information into an already extensive and time consuming procedure. 

The FDA must select one of the following two choices to satisfy the ALS community.

1. The immediate implementation of a simple application procedure without the March 6, 2009 filing deadline is requested. The existing FDA IND application without IRB requirement could be easily filed by ALS families, would provide direct FDA oversight of Iplex distribution, negate undue financial hardship and provide expedited access to Iplex for the ALS community. At this time, it is estimated that 100 to 200 patents would be able to afford and wish to purchase Iplex.

2. Fast track approval for the Myotonic Muscular Dystrophy Iplex trial currently underway that has had excellent results from Phase I and Phase II clinical trials and is scheduled to begin its Phase III trial, which will not be completed for another year or two at the regular pace.  If fast track approval is given, the drug would become available sooner through off label prescription by any physician.

To Accomplish This Goal, We Must Take Immediate Action

Create a media blitz. - Write, call and email anyone in the television or newspaper world.

Email, write or call your Senator and Representative. - Ask for a Congressional Hearing.  Request an open meeting with the FDA

Email, write or call the new FDA Administrator, Dr. Margaret Hamburg

Email, write or call the new FDA Deputy Commissioner, Dr. Joshua Sharfstein

Chronology of FDA Actions Regarding IPLEX

FDA Concerns and ALS Worldwide Response

FDA Definitions of Emergency Life Threatening Diseases

FDA Misconceptions Regarding Myotrophin

Sample Letter to Send to the Media