Accelerated Approval for GM604

I almost ignored this in my inbox, but then i thought…

What if I didn’t sign?

I watched this video.


via Change.org

Hello everyone, my name is Anthony Carbajal and my mom and I are both living with a devastating neurological disease called ALS. You may have seen my recent viral video that caught the worlds attention… even Ellen’s attention. In my video I described the urgency to find a viable treatment and how we desperately need support.

ALS is currently an incurable, progressive, degenerative neurological disorder and is designated as a terminal illness. For reasons that are still not completely understood, the nerve cells in my brain and spinal cord are gradually deteriorating. As a result, ALS patients like me slowly lose the ability to move, speak, swallow, and finally… to breathe. ALS leads to complete paralysis, and the average life expectancy from diagnosis is 2-5 years. 

Right now there is no viable treatment option for this terminal illness. The only FDA approved treatment is a drug called Riluzole, which extends life expectancy by a few months with no improvement to the quality of life. The really alarming part is that Riluzole was approved 20 years ago and nothing new has been made available since then!

There is a new drug called GM6, that is currently being evaluated by the FDA. According to Genervon, the company making the drug, “Preclinical, Phase 1, and Phase 2A data all demonstrate the safety and efficacy of GM6.” There were no negative side effects from the treatment, and most importantly, the differences in outcomes between the group of trial patients given the placebo and those given GM6 were “statistically significant” – even though the trials were small and not even set up to produce such dramatic results!

Genervon met with the FDA in early February 2015 and asked that GM6 be granted Accelerated Approval, which would mean that the drug would be made available to all ALS patients immediately. The FDA will make a decision by mid-April, which is why it is so important that we act now.

Thousands of ALS patients and myself want to be able to try this drug. I do not have any other options to fight.

 I don’t want to be just another statistic. Please sign our petition urging the FDA to grant accelerated approval for GM604. We can’t do this without YOUR participation and help! I honestly believe we’re stronger together. Thank you for your time and consideration.


The Letter:

Letter to
FDA Acting Director, Office of Drug Evaluation William Dunn, M.D.
FDA Deputy Commissioner Robert Califf, M.D.
FDA Chief Scientist Stephen Ostroff, M.D.
and 17 others
FDA Director, Office of Strategic Programs Theresa M. Mullin, Ph.D.
FDA, Director, Office of New Drugs John Jenkins, M.D.
FDA Deputy Center Director, Clinical Science Robert Temple, M.D.
FDA Director, Center for Drug Evaluation & Research Janet Woodcock, M.D.
Senator Sheldon Whitehouse
Senator Richard Burr
Senator Ron Wyden
Senator Mark Warner
Senator Tom Carper
Senator Lamar Alexander
Senator Patty Murray
Senator Elizabeth Warren
Senator Bernie Sanders
Senator Mark Kirk
Senator Rand Paul
Senator Tammy Baldwin
Senator Lisa Murkowski
Amyotrophic Lateral Sclerosis (ALS) is 100% fatal and results in a slow death, with suffocation resulting from a paralyzed diaphragm muscle being the most common cause of death. ALS leaves the brain in-tact, so patients are keenly aware of their inability to breath. Currently there is only one FDA treatment option for people with ALS, Riluzole, which extends life expectancy by a couple of months with no improvement to the quality of life. Riluzole received United States Food and Drug Administration (FDA) approval in 1995. In the past 20 years, there have been no new treatment options made available to patients with this horrific terminal illness.Early in February 2015 a biopharmaceutical comapny, Genervon, met with the FDA and asked that their treatment, GM6, be granted Accelerated Approval with post-marketing requirements. They presented the safety and efficacy data to back up their request and ALS fits all the criteria for this rapid access program.

The AS community has waited too long for a treatment option that proves to be promising, that offers real hope.

Grant Accelerated Approval for Genervon’s GM6.

4 thoughts on “Accelerated Approval for GM604”

  1. I think I am misunderstanding the whole FDA’s role, I thought they were meant to save lives? In the case of ALS patients, they are contradicting everything they stand for! Is the real reason they aren’t fast tracking it because they deem it to mostly affect people later in life who are thought to be a financial burden on society? Or is it because there is only a small percentage of people with ALS and it’s not financially beneficial? The FDA owes us an explanation as to why they are not doing everything possible to release this life-saving medication for already terminally ill.

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