The Case for Granting Accelerated Approval to GM604

This past week I went to Washington DC to speak at a rally urging the FDA to grant Accelerated Approval for GM604. I saw old friends, made new friends, connected with many families in the ALS community, and met with members of Congress.

While we do not yet know how effective GM604 will end up being, Accelerated Approval was specifically designed to allow patients access to treatments, that have been shown to be safe, for serious conditions with limited therapeutic options.

Please note that Accelerated Approval does not come at the expense of good science. Data will continue to be collected and full approval will not be granted until results of a confirmatory trial prove efficacy. There are simple statistical techniques that can be used to control for placebo effects even in single-armed trials.

Here is the text of my speech in which I lay out why I believe Accelerated Approval should be granted:

March 25, 2015

Hello, my name is Stephen Finger.  It is an incredible honor to speak here today in front of so many families involved in this fight. I grew up across the river in Arlington, VA, but never anticipated coming to DC for this reason.

Two years ago I was diagnosed with ALS. Between my son James’s first birthday and his sister Mary Adair’s third, I was told, what I thought was a minor issue with my hands, was a disease that would rob me of my ability to play catch, to play tag, to walk, to speak, to eat, to breath.

My family’s lives where forever changed and if one thing was clear, it was that time was of the essence. And just as we recognized the need to live urgently; to hug today, to laugh today, to live today, to love today. We also recognized the need to act urgently, to act today. That is why we are here. Team Gleason says “Not quietly”. The ALS Association says “The time is now”. We are here telling the FDA that the status quo is not good enough and they need to do everything in their power speed the search for a cure. We are here urging our members in Congress to remind the FDA to work with the flexibility and urgency needed to fight this universally fatal disease.

I have now been a part of this community long enough to see patients who were diagnosed with this disease after me lose their fight. Last year, I had the pleasure of meeting Trickett Wendler in the halls of Congress. A young mother with three kids, a wife, a friend, a relentless advocate, she was diagnosed in the summer of 2013 and passed away in less than 2 years. Always one to speak her mind, she has asked her friends to continue to fight. So we are all here on her behalf.  On Tony Conway’s behalf. On so many other great people’s behalf, who have been lost to this disease that we know can be solved.

We are not just here acting in their memory, we are here inspired by the way they fought this disease. Everything we do must be done with the recognition that every 90 minutes another mother, another father, another daughter, another son, another friend will be lost.

It is with that urgency that we are here today asking that the FDA grant GM604 Accelerated Approval. Let’s not force it to go more of the traditional trials that will take years to complete. Based on the results of the Phase I and II trials, let’s make it available immediately to patients who desperately need it, while we gather confirmatory data required of full approval. We are not asking today to rewrite any laws. We are not asking for a special exception. We are asking that the FDA uses the tools at their disposal to make this treatment available to as many people as possible in the shortest amount of time.

Accelerated Approval is not a new program. It has been around for over 20 years, specifically designed for situations like this. Congress has provided them with the ability to act with the urgency this situation merits. The 2012 Food and Drug Administration Safety and Innovation Act, with near unanimous support in both the House and Senate, I quote: reiterated the “Agency’s longstanding commitment to regulatory flexibility regarding the evidence required to support product approval for the treatment of serious or life-threatening diseases with limited therapeutic options”.

That is us! Here is their chance to live up to their own directives. Today! We are simply asking that they use the tools at their disposal to act with the urgency Congress told them to act with. The urgency deserved by the families in this fight.   No ALS treatment has been granted Accelerated Approval before, so here is an opportunity to show a new way forward. The status quo is not good enough.

In GM604, we have a drug meets the FDA’s own criteria for Accelerated Approval. Its safety has been demonstrated according to traditional standards. Over 50 patients have received GM604 with no adverse events, consistent with the preclinical work.  Surrogate endpoints for efficacy have been reached. Even in a small trial, GM604 had a significant effect on Forced Vital Capacity, which we know is a predictor of survival. Because ALS is a complex disease and progressions vary, meeting traditional standards of efficacy will require additional large and lengthy trials. Well we don’t have time for that and Accelerated Approval was specially designed to address this. While we wait for confirmatory data, let’s give current patients a fighting chance.  That is the directive Congress has given.

Now sure after Phase I and Phase II trials there are still risks, but there will still be risks after Phase III and more importantly, we all know what the risk of inaction is. Accelerated Approval does not mean we enter the wild, wild west. Data will still be collected and approval could still be revoked if confirmatory trials do not go our way. But given the safety data, given the suggestion of efficacy, given the prognosis of the disease, given the urgency of the situation, isn’t that a risk we are willing to take?  Isn’t that a risk the FDA is supposed to take? Given the evidence from the competed trials, doesn’t that make more sense than telling current patients to sit tight for two years until another trial can be completed?  We won’t sit here quietly. Too many people have already been lost. Too many people have shown us the importance of acting today.

The FDA has recognized that different conditions require different acceptance of risk. They have granted Accelerated Approval status to cancer drugs, to HIV treatments. They have this tool at their disposal specifically for situations like this.  Just because ALS is not communicable does not mean that we can be complacent.

So we are telling the FDA to act urgently, to act today, to act for the families in this fight, to grant Accelerated Approval to GM604.

Thank you.

If you have not done so, please sign the White House petition requesting that the President urge the FDA to grant Accelerated Approval to GM604.

https://petitions.whitehouse.gov//petition/request-fda-grant-accelerated-approval-potentially-life-saving-treatments-als-now-starting-gm604