Recent events make our May 11th rally even more important! Let’s make sure this event is a huge success! Please register: http://www.meetup.com/ALS-Faster-Drug-Approval/events/221529700/ Important note about Genervon’s GM604 Application: We have been corrected by Genervon and now understand that they met with the FDA early February and told the FDA that they wanted to pursue the Accelerated Approval Program (AAP) as an option for going forward with GM604. Then on February 24th, they filed a formal request with supporting documents to the FDA to further the discussion. We have been advised that these pre-NDA negotiations represent the typical course of action for all drug companies, especially when the application is for AAP. As of today, Genervon has not heard from the FDA regarding the next steps. Therefore we would like to clarify that the official New Drug Application (NDA) for GM604 has not yet been submitted -- Genervon never claimed to have submitted an NDA and we take full responsibility for this confusion. Response to FDA's public statement: On Friday April 17th, the FDA posted an unusual public statement calling upon Genervon to make public, information the FDA already has in their possession: “We call upon Genervon to release all the data from their recently completed trial in order to allow a more informed discussion of the trial findings among ALS stakeholders. Such a release should include the pre- specified clinical outcome measures as assessed by change from baseline observations that were taken just prior to randomization to drug or placebo.” http://www.fda.gov/Drugs/DrugSafety/ucm443242.htm Genervon’s response to the FDA statement (scroll down to the April 17 update): http://www.genervon.com/genervon/about_pressreleases.php The April 17 public statement by the FDA may be unprecedented. When seeking approval for a drug, companies typically submit this level of data only to the FDA whose job is to evaluate it and make a decision. According to the Institute of Medicine guidelines, data sharing is not expected when there is an active application for approval. The FDA can certainly ask Genervon to share confidential data that supports their results with other medical researchers, for example, to conduct an independent analysis, but that request should be made in private negotiations - not in a public announcement that could harm a company’s reputation by implying information is being withheld. There is no evidence to support that assertion. We agree that others may analyze or interpret the trial data differently than Genervon, but that doesn’t prove the company’s analysis is incorrect. Leading ALS researchers at Massachusetts General Hospital and Columbia University Hospital recruited patients and conducted the trials; a Contract Research Organization (CRO) performed the data analyses. The data summarized in Genervon’s releases were collected from the research institutions and the CRO, independently from Genervon. The FDA is limited, by law, to communicate any concerns or questions regarding information submitted, to the drug company submitting the data. Further, the FDA is in complete control of the regulatory pathway. No one can obtain GM604 or any other unapproved drug inside or outside a clinical trial, through an expanded access program or compassionate use, without FDA approval. Genervon appears to be following the FDA’s process in good faith, and there is nothing improper about requesting the FDA to consider the eligibility of GM604 for Accelerated Approval (AAP). The decision to accept an application for AAP is made solely by the FDA. The FDA is also prohibited from publicly discussing its interactions with companies developing new medicines, or releasing any information submitted, except under special circumstances that arise later in the development process (that do not apply in this case). This begs the question, is the FDA legally allowed to post a public comment asking the drug company to release all its data? We have communicated this question to the FDA and requested they clarify that Genervon has no obligation to comply with that request. So far the FDA has refused. Is a powerful government agency stalling potential approval of a promising new ALS drug in a manner that may violate its own laws and regulations? We will continue to explore the legality of their seemingly unprecedented and unwarranted action. HopeNOWforALS.org - Our campaign has expanded. We are committed to accelerating the approval of all promising ALS drugs and treatments (not only GM604) and to bring the clinical trial process into the 21st century. It remains the prevalent position of those directly affected by ALS (PALS, their families, and friends), that given the prognosis and the fact that most patients are ineligible for standard clinical trials, the FDA must extend the Accelerated Approval Program to include treatments that have demonstrated safety and potential efficacy. The language in the 1992 FDA Accelerated Approval Program supports our position. We also want to reiterate that Genervon had no part in creating this grassroots patient-based campaign. We are PALS and their family and friends working to save the lives of the current generation of PALS. We will put our efforts into accelerated approval for any drug or treatment that demonstrates safety and potential efficacy for ALS. ALL OUR SUPPORTERS MUST TAKE ACTION! We have to push back. Our best move is to keep our energies focused on the FDA and Congress. Email the FDA…and make sure you also email/call the US Senators who represent your home state – today! Below you will find “WHAT TO INCLUDE”, email addresses and the links to Senators contact pages. Please see and understand that the last paragraph of “WHAT TO INCLUDE” should only be sent to Senators from your home state. Do not send that to the FDA. Please send this email to any of the contacts you made with Congressional Representatives during the last rally. Remember, the best way to follow breaking news items that affect our campaign is to follow us on Facebook: https://www.facebook.com/gm604forals We're in this together, Anthony Carbajal, Bethany Wolfe, Jehad Majed (jmajed@mac.com), Matthew Bellina, Nick Grillo (gm6nick@gmail.com), and the Torrino Family WHAT TO INCLUDE: #### [Start with any opening you like – BUT BE SURE TO INCLUDE]: The recent FDA public statement on their website asking drug developer Genervon to release confidential trial data to the public is an unethical move (and may also be an illegal attempt) by FDA to push back against the campaign because we are challenging their 53-year old regulatory model. I understand that until Genervon makes the specified information public, the FDA cannot legally discuss it and even if it is made public, the FDA will be constrained in terms of what they can say. By asking Genervon to share their data while GM604 is under review, the FDA is inviting their allies to take shots at it and create a circus-like atmosphere. The FDA has to stop refusing to use the Accelerated Approval Program (AAP) in the manner intended by Congress -- to meet the needs of populations just like the ALS Community. This is something that should have been done two decades ago when AAP became policy. [LAST PARAGRAPH FOR SENATORS ONLY] If you have not seen the FDA statement, here is the link: http://www.fda.gov/Drugs/DrugSafety/ucm443242.htm As your constituent, I want you to contact the FDA and tell them that you stand with the ALS Community, that you see their public post as inappropriate and irresponsible, and that new promising treatments must be considered for AAP. Please let me know when you have contacted the FDA on my behalf. ##### Email the FDA at: druginfo@fda.hhs.gov and copy these people: richard.moscicki@fda.hhs.gov janet.woodcock@fda.hhs.gov william.dunn@fda.hhs.gov stephen.ostroff@fda.hhs.gov commissioner@fda.gov eric.bastings@fda.hhs.gov US SENATORS Alabama Sessions, Jeff - (R - AL) (202) 224-4124 http://www.sessions.senate.gov/public/index.cfm/contact-jeff Shelby, Richard C. - (R - AL) (202) 224-5744 http://www.shelby.senate.gov/public/index.cfm/emailsenatorshelby Alaska Murkowski, Lisa - (R - AK) 202) 224-6665 http://www.murkowski.senate.gov/public/index.cfm/emaillisa Sullivan, Daniel - (R - AK) (202) 224-3004 http://www.sullivan.senate.gov/content/contact-dan Arizona Flake, Jeff - (R - AZ) (202) 224-4521 http://www.flake.senate.gov/public/index.cfm/contact-jeff McCain, John - (R - AZ) (202) 224-2235 http://www.mccain.senate.gov/public/index.cfm/contact-form Arkansas Boozman, John - (R - AR) (202) 224-4843 http://www.boozman.senate.gov/public/index.cfm/e-mail-me Cotton, Tom - (R - AR) 202) 224-2353 http://www.cotton.senate.gov/content/contact-tom California Boxer, Barbara - (D - CA) (202) 224-3553 https://www.boxer.senate.gov/contact/shareyourviews.html Feinstein, Dianne - (D - CA) (202) 224-3841 https://www.feinstein.senate.gov/public/index.cfm/e-mail-me Colorado Bennet, Michael F. - (D - CO) (202) 224-5852 http://www.bennet.senate.gov/?p=contact Gardner, Cory - (R - CO) (202) 224-5941 http://www.gardner.senate.gov/content/contact-cory Connecticut Blumenthal, Richard - (D - CT) (202) 224-2823 https://www.blumenthal.senate.gov/contact Murphy, Christopher - (D - CT) (202) 224-4041 http://www.murphy.senate.gov/contact Delaware Carper, Thomas R. - (D - DE) (202) 224-2441 http://www.carper.senate.gov/public/index.cfm/email-senator-carper Coons, Christopher A. - (D - DE) (202) 224-5042 http://www.coons.senate.gov/contact/ Florida Nelson, Bill - (D - FL) (202) 224-5274 http://www.billnelson.senate.gov/contact-bill Rubio, Marco - (R - FL) (202) 224-3041 http://www.rubio.senate.gov/public/index.cfm/email-senator-rubio?p=Contact Georgia Isakson, Johnny - (R - GA) (202) 224-3643 http://www.isakson.senate.gov/public/index.cfm/email-me Perdue, David - (R - GA) (202) 224-3521 http://www.perdue.senate.gov/content/contact-david Hawaii Hirono, Mazie K. - (D - HI) (202) 224-6361 http://www.hirono.senate.gov/contact/email Schatz, Brian - (D - HI) (202) 224-3934 http://www.schatz.senate.gov/contact Idaho Crapo, Mike - (R - ID) (202) 224-6142 http://www.crapo.senate.gov/contact/email.cfm Risch, James E. - (R - ID) (202) 224-2752 http://www.risch.senate.gov/public/index.cfm/email Illinois Durbin, Richard J. - (D - IL) (202) 224-2152 http://www.durbin.senate.gov/contact/email Kirk, Mark - (R - IL) (202) 224-2854 http://www.kirk.senate.gov/?p=comment_on_legislation Indiana Coats, Daniel - (R - IN) (202) 224-5623 http://www.coats.senate.gov/contact/ Donnelly, Joe - (D - IN) (202) 224-4814 http://www.donnelly.senate.gov/contact/email-joe Iowa Ernst, Joni - (R - IA) (202) 224-3254 http://www.ernst.senate.gov/content/contact-joni Grassley, Chuck - (R - IA) (202) 224-3744 http://www.grassley.senate.gov/constituents/questions-and-comments Kansas Moran, Jerry - (R - KS) (202) 224-6521 http://www.moran.senate.gov/public/index.cfm/e-mail-jerry Roberts, Pat - (R - KS) (202) 224-4774 http://www.roberts.senate.gov/public/index.cfm?p=EmailPat Kentucky McConnell, Mitch - (R - KY) (202) 224-2541 http://www.mcconnell.senate.gov/public/index.cfm?p=ContactForm Paul, Rand - (R - KY) (202) 224-4343 http://www.paul.senate.gov/?p=contact Louisiana Cassidy, Bill - (R - LA) (202) 224-5824 http://www.cassidy.senate.gov/content/contact-bill Vitter, David - (R - LA) (202) 224-4623 http://www.vitter.senate.gov/contact/email-senator-vitter Maine Collins, Susan M. - (R - ME) (202) 224-2523 http://www.collins.senate.gov/public/index.cfm/email King, Angus S., Jr. - (I - ME) (202) 224-5344 http://www.king.senate.gov/contact Maryland Cardin, Benjamin L. - (D - MD) (202) 224-4524 http://www.cardin.senate.gov/contact/ Mikulski, Barbara A. - (D - MD) (202) 224-4654 http://www.mikulski.senate.gov/contact Massachusetts Markey, Edward J. - (D - MA) (202) 224-2742 http://www.markey.senate.gov/contact Warren, Elizabeth - (D - MA) (202) 224-4543 http://www.warren.senate.gov/?p=email_senator Michigan Peters, Gary - (D - MI) (202) 224-6221 http://www.peters.senate.gov/content/contact-gary Stabenow, Debbie - (D - MI) (202) 224-4822 http://www.stabenow.senate.gov/?p=contact Minnesota Franken, Al - (D - MN) (202) 224-5641 http://www.franken.senate.gov/?p=email_al Klobuchar, Amy - (D - MN) (202) 224-3244 http://www.klobuchar.senate.gov/public/email-amy Mississippi Cochran, Thad - (R - MS) (202) 224-5054 http://www.cochran.senate.gov/public/index.cfm/email-me Wicker, Roger F. - (R - MS) (202) 224-6253 http://www.wicker.senate.gov/public/index.cfm/contact Missouri Blunt, Roy - (R - MO) (202) 224-5721 http://www.blunt.senate.gov/public/index.cfm/contact-roy McCaskill, Claire - (D - MO) (202) 224-6154 http://www.mccaskill.senate.gov/contact Montana Daines, Steve - (R - MT) (202) 224-2651 http://www.daines.senate.gov/content/contact-steve Tester, Jon - (D - MT) (202) 224-2644 http://www.tester.senate.gov/?p=email_senator Nebraska Fischer, Deb - (R - NE) (202) 224-6551 http://www.fischer.senate.gov/public/?p=email-deb Sasse, Ben - (R - NE) (202) 224-4224 http://www.sasse.senate.gov/content/contact-ben Nevada Heller, Dean - (R - NV) (202) 224-6244 http://www.heller.senate.gov/public/index.cfm/contact-form Reid, Harry - (D - NV) (202) 224-3542 http://www.reid.senate.gov/contact New Hampshire Ayotte, Kelly - (R - NH) (202) 224-3324 http://www.ayotte.senate.gov/?p=contact Shaheen, Jeanne - (D - NH) (202) 224-2841 http://www.shaheen.senate.gov/contact/ New Jersey Booker, Cory A. - (D - NJ) (202) 224-3224 http://www.booker.senate.gov/?p=contact Menendez, Robert - (D - NJ) (202) 224-4744 http://www.menendez.senate.gov/contact New Mexico Heinrich, Martin - (D - NM) (202) 224-5521 http://www.heinrich.senate.gov/contact/write-martin Udall, Tom - (D - NM) (202) 224-6621 http://www.tomudall.senate.gov/?p=contact New York Gillibrand, Kirsten E. - (D - NY) (202) 224-4451 http://www.gillibrand.senate.gov/contact/ Schumer, Charles E. - (D - NY) (202) 224-6542 http://www.schumer.senate.gov/contact/email-chuck North Carolina Burr, Richard - (R - NC) (202) 224-3154 http://www.burr.senate.gov/public/index.cfm?FuseAction=Contact.ContactForm Tillis, Thom - (R - NC) (202) 224-6342 http://www.tillis.senate.gov/content/contact-thom North Dakota Heitkamp, Heidi - (D - ND) (202) 224-2043 http://www.heitkamp.senate.gov/public/index.cfm/email-heidi Hoeven, John - (R - ND) (202) 224-2551 http://www.hoeven.senate.gov/public/index.cfm/email-the-senator Ohio Brown, Sherrod - (D - OH) (202) 224-2315 http://www.brown.senate.gov/contact Portman, Rob - (R - OH) (202) 224-3353 https://www.portman.senate.gov/public/index.cfm/contact-form Oklahoma Inhofe, James M. - (R - OK) (202) 224-4721 http://www.inhofe.senate.gov/contact Lankford, James - (R - OK) (202) 224-5754 http://www.lankford.senate.gov/content/contact-james Oregon Merkley, Jeff - (D - OR) (202) 224-3753 http://www.merkley.senate.gov/contact Wyden, Ron - (D - OR) (202) 224-5244 http://www.wyden.senate.gov/contact Pennsylvania Casey, Robert P., Jr. - (D - PA) (202) 224-6324 http://www.casey.senate.gov/contact Toomey, Patrick J. - (R - PA) (202) 224-4254 http://www.toomey.senate.gov/?p=contact Rhode Island Reed, Jack - (D - RI) (202) 224-4642 http://www.reed.senate.gov/contact/email Whitehouse, Sheldon - (D - RI) (202) 224-2921 http://www.whitehouse.senate.gov/contact/ South Carolina Graham, Lindsey - (R - SC) (202) 224-5972 http://www.lgraham.senate.gov/public/index.cfm?FuseAction=Contact.EmailSenatorGraham Scott, Tim - (R - SC) (202) 224-6121 http://www.scott.senate.gov/contact/email-me South Dakota Rounds, Mike - (R - SD) (202) 224-5842 http://www.rounds.senate.gov/content/contact-mike Thune, John - (R - SD) (202) 224-2321 http://www.thune.senate.gov/public/index.cfm/contact Tennessee Alexander, Lamar - (R - TN) (202) 224-4944 http://www.alexander.senate.gov/public/index.cfm/email Corker, Bob - (R - TN) (202) 224-3344 http://www.corker.senate.gov/public/index.cfm/emailme Texas Cornyn, John - (R - TX) (202) 224-2934 http://www.cornyn.senate.gov/public/index.cfm?p=ContactForm Cruz, Ted - (R - TX) (202) 224-5922 http://www.cruz.senate.gov/?p=email_senator Utah Hatch, Orrin G. - (R - UT) (202) 224-5251 http://www.hatch.senate.gov/public/index.cfm/email-orrin Lee, Mike - (R - UT) (202) 224-5444 http://www.lee.senate.gov/public/index.cfm/contact Vermont Leahy, Patrick J. - (D - VT) (202) 224-4242 https://www.leahy.senate.gov/contact/ Sanders, Bernard - (I - VT) (202) 224-5141 http://www.sanders.senate.gov/contact/comment Virgina Kaine, Tim - (D - VA) (202) 224-4024 https://www.kaine.senate.gov/contact Warner, Mark R. - (D - VA) (202) 224-2023 http://www.warner.senate.gov/public/index.cfm?p=ContactPage Washington Cantwell, Maria - (D - WA) (202) 224-3441 http://www.cantwell.senate.gov/public/index.cfm/email-maria Murray, Patty - (D - WA) (202) 224-2621 http://www.murray.senate.gov/public/index.cfm/contactme West Virginia Capito, Shelley Moore - (R - WV) (202) 224-6472 http://www.capito.senate.gov/content/contact-shelley Manchin, Joe, III - (D - WV) (202) 224-3954 http://www.manchin.senate.gov/public/index.cfm/contact-form Wisconsin Baldwin, Tammy - (D - WI) (202) 224-5653 http://www.baldwin.senate.gov/contact Johnson, Ron - (R - WI) (202) 224-5323 http://www.ronjohnson.senate.gov/public/index.cfm/email-the-senator Wyoming Barrasso, John - (R - WY) (202) 224-6441 http://www.barrasso.senate.gov/public/index.cfm/contact-form Enzi, Michael B. - (R - WY) (202) 224-3424 http://www.enzi.senate.gov/public/index.cfm/e-mail-senator-enzi