Our Response to the FDA’s Latest Letter and What We’re Doing to Keep Up the Pressure

Our Response to the FDA’s Latest Letter and What We’re Doing to Keep Up the Pressure

Following our We Can’t Wait Action meeting with the FDA, we sent a letter to the agency asking it to reaffirm its commitment to the 2019 ALS Guidance and provide us with a detailed report showing how it has implemented the guidance. 

This week, the ALS Association received a response from Dr. Patrizia Cavazzoni, Director of the Center for Drug Evaluation and Research. You can read her letter here

Dr. Cavazzoni states:
We recognize the continued unmet need for treatments for patients living with ALS and are committed to engaging with companies and the patient community to facilitate the development of treatments for this disease. We can also assure you that we are exercising the regulatory flexibility described in FDA’s 2019 guidance for industry titled Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment (ALS Guidance) and we continue to commit to doing so.

We are encouraged that the FDA is committed to the guidance and we appreciate Dr. Cavazzoni’s acknowledgment that there is “unmet need for treatments” for people living with ALS. But the ALS community deserves to know exactly what the FDA is doing to help expedite approval of promising treatments. 

While Dr. Cavazzoni states that the FDA is “committed to exercising [the guidance] when appropriate,” that does not appear to be the case when it comes to AMX0035. The Phase 2 trial of AMX0035 met its primary and secondary endpoints and is generally safe, and yet the FDA appears to be requiring another clinical trial of the drug. While Amylyx is working with Health Canada to potentially bring the drug to market for Canadians living with ALS by the end of the year, it could be several years before Americans with ALS are able to access AMX0035, if at all. 

Just recently, the FDA used its regulatory flexibility to approve Aduhelm for the treatment of Alzheimer’s disease. This is good news for people living with Alzheimer’s and their loved ones. But we are left to wonder the FDA is not using similar flexibility for a promising ALS treatment with strong safety data that provides clinically meaningful benefits. People with ALS and their loved ones cannot wait. 

Our plan continues to be: 1) put public pressure on the FDA, 2) work behind the scenes to expedite change, and 3) seek greater public accountability for FDA.

First, our latest public pressure include comments in an Endpoints article and a guest opinion in Stat News

Second, we are working with the FDA, as well as behind the scenes with pharmaceutical companies to speed the drug development process. We cannot share details of conversations with pharmaceutical companies due to non-disclosure agreements with those companies. We will share what we can as soon as we are able.

Finally, we are continuing to push Congress for changes to the FDA to help expedite getting promising treatments to people with ALS. This includes supporting ACT for ALS, which will create a new research grant program at FDA, as well as a new framework for delivering experimental therapies to people with ALS, and supporting the Promising Pathways Act, which will create a conditional approval pathway similar to the one in Europe. 

We will keep you updated on any additional developments while we keep up the pressure.

This blog post has been revised to clarify that the ALS Association does not have any non-disclosure agreements with the FDA.


Submitted by: Jeff D. on Fri, 06/25/2021

Add my voice to the pressure to make available treatments such as AMX 0035!

Submitted by: John L. on Thu, 07/01/2021

Dr. Cavazzoni: The ALS community deserves the highest degree of attention and urgency given that there are no drugs of significance for this horrific condition. The FDA needs to act now to approve AMX0035 and any other drug that has shown safety and efficacy. I have been living with ALS for over two years and people like me have very little time. Thanks

Submitted by: becky H. on Thu, 07/01/2021

Please let us know what we can do personally to keep the pressure on!

Submitted by: Amy L. on Fri, 07/02/2021

Hi Becky, you can contact your members of Congress and ask them to support the ACT for ALS Act and the Promising Pathway Act. https://als.quorum.us/register/

Submitted by: Ora H. on Thu, 07/01/2021

Get on with it. People are dying.

Submitted by: George M. on Thu, 07/01/2021

Thank you’ll for your commitment to finding a cure for ALS.
I’m sorry but our representatives are paying to much attention to the border issues and other countries.
We are spending resources on foreign countries and there people.
Frankly I’m sick of it as my daughter and so many of our citizens suffer from ALS and other Sickness.
Those tax dollars should be used for our US citizens and not them so much needed research can be done enough is enough let’s worry about our own and let there governments take care of them.
GOD bless America

Submitted by: Susan L. on Fri, 07/02/2021

Dr. Cavazzoni: The ALS community deserves the highest degree of attention and urgency given that there are no drugs of significance for this horrific condition. The FDA needs to act now to approve AMX0035 and any other drug that has shown safety and efficacy. I have been living with ALS for over three years and people like me have very little time. Thanks

Submitted by: Laura W. on Fri, 07/02/2021


Submitted by: Karen L. on Sat, 07/03/2021

We deserve to have treatments now, our time is running out.

Submitted by: Mario S. on Fri, 06/25/2021

Dr. Patrizia Cavazzoni, I read your response to the ALS Association and you do not recognize the need for this these new drugs specifically AMX0035. These new drugs specifically this drug has helped slow the progression of ALS and people like me and it would further benefit us for additional drugs that can help the community our time is short and you need to act appropriately. Use your power to help Patience's with ALS and initiate regulatory flexibility for the ALS community 

Submitted by: Debbie B. on Thu, 07/01/2021

I can’t believe they aren’t doing more to find a drug that will slow down the progression of this disease or a cure for it. I bet if they or one of their loved ones had ALS they would being doing all they could to find a trial drug or cure. Why is Canada getting a trial drug but not the US! Doesn’t make sense! Please help!!!

Submitted by: Pat F. on Thu, 07/01/2021

Please continue to push Congress for changes to FDA. We need the new treatments badly.

Submitted by: Cindy Y. on Thu, 07/01/2021

Dr. Cabazzoni, I have read your response and believe ALS patients should receive attention immediately. Please approve the new drug AMX0035. Patients with ALS deserve as much attention and research as others.

Submitted by: Connie M. on Thu, 07/01/2021

I was diagnosed September 2020, ALS progressing slow but I would love to have any medication or trials. AMX00035 looks promising!

Submitted by: Patricia F. on Thu, 07/01/2021

Please continue to push Congress for changes to FDA to get treatment for us ALS patients, we need it.

Submitted by: Helki P. on Thu, 07/01/2021

Dr. Cavazzoni, it is with great urgency that you act on this valuable new treatment. In fact, I ask that you act with the same urgency that you would if your life or anyone in your family’s life depended on it. As we speak my father, Air Force Veteran and retired high school principal is in the next room fighting for every breath he takes, and my heart is broken.

Submitted by: Diane P. on Thu, 07/01/2021

Please, this is literally a matter of life and death. I want my youngest brother to have a chance to out live us. Please get AMX0035 to those suffering from ALS ASAP! If the Canadians can get it out by end of this year, why can't we?

Submitted by: Shavon N. on Thu, 07/01/2021

Add my voice to the pressure to make available treatments such as AMX 0035!

Submitted by: Mary E. S. on Thu, 07/01/2021

I live with ALS every day, how can you deny me a longer life. I want to see my child graduate from high school-college. Start a life, have kids (my grandchildren) for you to deny me and her of her mother is unthinkable. Do you have any idea what it cost to live with ALS, the medical bills, the cost of equipment, to get turned down every step of the way because you followed all the rules but you now have to give up everything you work for because you have to much according to the government and the state. But you don’t have enough to support your family. You can’t walk, talk, lift your hands and arms to even feed yourself. You can’t go to the bathroom, let alone wipe your own ass. You can’t cook, garden, talk to your child, your husband, take your daughter anywhere. How dare you deny me a little more time with my family, shame on you.

Submitted by: Lisa B. on Thu, 07/01/2021

My aunt has been diagnosed with ALS and her kids are in denial. So I took it as myself to dig deeper cause she’s slowly disappearing.

Submitted by: Pamela G. on Thu, 07/01/2021

I don't know anyone personally that has contracted COVID-19, (or died of it). I have lost my best friend to ALS, and now another one is getting close to death. ALS is not contagious, apparently, but it is on the rise, apparently.
I hope you accelerate the pace of research and development, as was done with COVID-19, which proves making a concerted effort with funding and encouragement works, before my oldest friend, the only one left from my high school days 55 years ago, feebly attempts to take her last breath.
We have found that when there is an outcry, pharmaceutical companies can work fast and get results. When people are close to death, after suffering for years, I think standard safety precautions should be reconsidered. This isn't headaches we are talking about.
The HIV cocktails treatments would probably not have been developed if not for pressure from the victims of AIDS taking things into their own hands and pushing pushing pushing for anything that would help. Now they have drugs that do help.
There aren't as many people with ALS or COVID-19, but their suffering is just as intense and frightening for them and their friends and family.
Please give AMX0035 a chance to potentially extend the life of my suffering friend, possibly long enough for a better drug to be developed. I am both glad and sorry that there isn't more of a market demand to spur on the pharmaceutical companies, for obvious reasons.
Please help my friend. I'm sure she wouldn't mind taking a risk. She is a fighter. She recently went birdwatching in a contraption she was strapped into that looked like something out of a Sci-fi movie, with triangular tread tracks reminding me of a tank, that could drive over walls. I don't know where she got that, or how, but it isn't prolonging her life, just her ability to live and enjoy her last hours.
You could help. Help her see a few more birds before she can't breathe any more, even with a ventilator.
Thank you

Submitted by: Jon B. on Thu, 07/01/2021

If we as a nation can solve Covid 19 using "Operation Warp Speed" urgency, then why in hell can't we do the same for ALS??? If the FDA had followed standard protocols for Covid vaccines, we would still be dying by the tens of thousands.
Get the lead out FDA. Make this the new "Operation Warp Speed " and save thousands of Americans.
My wife passed away from ALS after a courageous 3 year effort to fight it.

Submitted by: Robert D. on Thu, 07/01/2021

I was diagnosed with ALS in November of 2020 and I am guessing any one of us with this Horrible disease would be willing to take any drug or compound with a chance of prolonging life whether phase 1 2 3. We really have nothing to lose . I personally will sign any waiver if that is what it takes. We need help soon . We are dying out here.

Submitted by: Angela B. on Thu, 07/01/2021

Please, PLEASE approve this drug. I have been diagnosed with ALS two years ago. Is progressing slowly. I don’t know how long I have left. Why you won’t approve it??? We, ALS people, have only two options, this new drug or death. Let’s us try the first option before it’s too late!!! Please, approve this drug!!!

Submitted by: Mary G. on Thu, 07/01/2021

My husband was diagnosed with ALS in May 2020, but he started with symptoms in June of 2019. It took months to get approval for tests and to see ALS doctors. (Insurance approval and then trying to get appointments took months) Today he can no longer walk, has no use of his arms or hands. He still talks a little but it’s very difficult to make out what he is saying. He is only 66 years old and has the possibility of a long life ahead of him if not for this terrible disease. We need a cure! We need more attention and importance given to finding a cure for this terrible disease! Our loved ones are dying way before their time. Why is Canada approving this drug and the US wants more trials? We need action now! Please help our family and friends live and approve AMXOO35 now!

Submitted by: Bill W. on Thu, 07/01/2021

The FDA needs to move quickly on NU-9 (Northwestern). I see nothing else that is very promising (my son was diagnosed 2 months ago at Mayo). I am a retired hospital and health care finance CFO and I intend to get involved!

Submitted by: Kimberly T. on Thu, 07/01/2021

My mother passed away in 2018 from ALS. I watched her lose every ability she had . When she passed she could not walk talk eat or move any part of her body except for her thumb. I hope that if there is something out there that can help people it should be made available as soon as possible. I pray for anyone that is suffering from this. May God bless each and every one of you and I pray they will find treatment for you soon ❤️

Submitted by: David L. on Fri, 07/02/2021

Dr. Cavazzone

The FDA must act with more forethought and expediency to approve drugs for ALS. Your actions are not consistent with the words you speak. People with ALS are suffering and dying while being denied access to potentially helpful drugs that have been proven safe.
The US should lead the world with its compassion care and understanding of complex diseases and expedite potential treatments.
FDA lead from the frontlines of the battle.
David L. MD

Submitted by: Jeffrey A. on Fri, 07/02/2021

I was diagnosed with ALS in February 2020 and since then my symptoms have of course deteriorated. I have been astonished by the absolute lack of progress that the medical community have made in finding a means to arrest this terrible disease. To be blunt, it almost seems there has been a stoppage of physical development in the effort to find a cure. On behalf of all ALS sufferers I implore the FDA to accelerate ASL development as time is running out. Approval of AMX0035 would be a meaningful first step.

Submitted by: Debra H. on Fri, 07/02/2021

The FDA needs to understand that these people with ALS right now have no chance of survival. My father died of ALS in May of this year. I watched him die a horrible death. ALS med is needed now.

Submitted by: Suzanne S. on Fri, 07/02/2021

Dear Dr Catacombs, your message does not acknowledge that for those of us with ALS, AMZ0035 could mean the difference be tween walking and being in a wheelchair; feeding yourself or a feeding tube; being able to speak or not; being able to breath without a respirator. Please realise the day to day struggle s we have and how the slowing of our symptoms is so critical

Submitted by: David D. on Fri, 07/09/2021

Dr. Cavazzoni, I was diagnosed with ALS in December of 2018, I am a slow progressor but no longer eligible to participate in in the phase III. I am not advocating for myself as much as I am for those similar to me that could benefit from AMX0035 and are not eligible when this may be the drug that allows them the life to continue the fight and "live with ALS" I urge you to today make AMX0035 available as you have done for Alzheimer patients with Aduhelm.

Submitted by: Christopher P. on Fri, 07/09/2021

Simple questions....what is hanging up the approval? Where can folks suffering with ALS go to follow what is being done to help?

Submitted by: Dell A. on Mon, 07/12/2021

A very dear 66 year-old woman who brightened any room she ever entered recently passed just short of her 2 yr diagnosis of ALS .Her family and friends deserved to have more time with her. Get off your collective derrieres and do something to help American citizens afflicted with this diagnosis.

Submitted by: Mike W. on Tue, 07/13/2021

When your brother has been diagnosed with ALS, it makes you reflect on his lively, energetic, engaged life. It puts you in mind of his being bucked off a horse on his first visit to the West; his cycling and biking thru the Rockies and the West; his ranch, and his horses that accompanied him on mountain treks; his becoming recognized as a leading expert in appraising land for conservation easements; his wife, whose support and love shine brightly; his service to community and professional organizations; his grin under one or another of his hats and caps.

In contrast to such reflections, one hears the measured tone of the FDA: patience patience patience. One has little sense of urgency, of recognition that ALS usually runs a terribly short course. To each FDA staffer involved in any way in developing and approving drugs for ALS: Imagine a loved one having been diagnosed with ALS. Imagine how much patience you would have. Imagine how much patience your loved one would have. Act accordingly. Please.

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