Public Comments Being Accepted as FDA Considers AMX0035 Approval

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The ALS community has the opportunity to encourage The Food and Drug Administration (FDA) to approve Amylyx Pharmaceutical’s new drug application for AMX0035.

The agency will hold an FDA advisory committee meeting for AMX0035 on Wednesday, March 30, 2022 from 10 a.m. - 4:30 p.m. ET. The meeting will be virtual.

Public comments are being accepted in the run up to the meeting. Comments received by the agency by March 16 will be considered by the advisory committee, which is tasked with making a recommendation to the full FDA. Comments can still be submitted March 17 – 29 for consideration by the full agency.

The FDA has asked for input from the ALS community including people with ALS, care partners, family members, and clinicians.

We encourage you to submit a written comment by March 16 by following the steps below.

  1. Click here to view the comment form: https://www.regulations.gov/commenton/FDA-2018-N-0410-0058
  2. Write your comment. You can submit this in the “Comment” text box or upload your comment document in the “Attach Files” section. Some topics you may want to consider when submitting written comments include:
    • Your name, when you were diagnosed, and how ALS is impacting your life. Please also note if you were in the AMX0035 trial.
       
    • How you view the importance of a new therapy that offers the chance of slowing disease progression or providing six months of additional life.
       
    • How you weigh a new therapy that offers the chance of benefit in balance with:
      • the risk that the therapy ends up having no benefit at all; or
      • the therapy may have unanticipated side effects.
    • Additional points to add to your comment to share why AMX0035 must be approved by the FDA are below:
      • While there are a few approved ALS drugs, there is a significant need for better therapies.
      • The phase 2 clinical trial of AMX0035 has shown the treatment to be safe and effective at slowing down disease progression.
      • AMX0035 is understood to be generally safe and well-tolerated.
      • Given these important findings and the need for a therapy that slows progression, we believe the studies conducted to-date are enough for FDA approval.
    • Remember to:
      • Keep the focus on the therapy being reviewed. This will make the information most useful to FDA and industry. 
      • Avoid discussing cost of treatment. Although this is an important concern for many people, the FDA is not responsible for setting healthcare or therapy costs.
  3. Once your comment is added to the form, select “Individual” and add your name to the contact information section of the form.
  4. Click “submit comment.” NOTE: Comments submitted to the FDA will be posted on the agency's website.

While the Advisory Committee meeting for AMX0035 will be very technical and include a lot of discussion around data, they will take into consideration the viewpoints of people with ALS and other community stakeholders. Your voice and experiences matter. By participating in this session with the Advisory Committee and the FDA, you will have an opportunity to make sure key decisionmakers understand the priorities of people with ALS and take those into account when reviewing AMX0035.

Comments

Submitted by: Susan D. on Mon, 03/07/2022

Please include me.

Submitted by: Bruce K. on Mon, 03/07/2022

Please approve.

Submitted by: Bonita G. on Mon, 03/07/2022

I have lost my step-grandfather, uncle and father to ALS. ALS has taken many years, opportunities and memories from me and my family. My step father lost his battle with ALS just 5 years after his older brother.
There was a “new drug” available but not approved in Canada and I feel that my step father would have had a longer and better life had he had been able to access the drug.
There has to be more approvals and studies into preventative medicine for ALS. Without that, it’s devastating.

Submitted by: Barry G. on Mon, 03/07/2022

Diagnosed in June 2018. ALS is absolutely devastating to my family, friends, and business. It has completely altered my life and everyone close to me. I have not tried the treatment but would love the chance to.

Submitted by: Ramiz N. on Tue, 03/08/2022

I would love to see how soon this medical will be out. My mother is diagnosed with als since sep.2021.

Submitted by: John S. on Tue, 03/08/2022

With the terminal nature of this disease, it is worth any patients time to go for a trial if they do choose. I know results aren’t guaranteed, but hope can still be powerful.

Submitted by: Noryda L. on Wed, 03/09/2022

I was finally diagnosed 1/2019 but suffered signs of ALS for at least 3 years prior. I’m 50 years

Submitted by: Michael C. on Fri, 03/11/2022

I was recently Diagnosed and given little direction on current medications ans research as a 52 year old healthy man, I have quite a long list of things I would like to accomplish and see. ALS has progressed very quickly. Any medication that could stop the progression or reverse this disease would allow me to grow old with my 14 year old son and my wife.

Submitted by: Michael C. on Fri, 03/11/2022

Recently diagnosed as a healthy 52 year old male. It has turned my life around and ruined my dreams. Any medication that will stop the progression or create a reversal would be a life-changer!

Submitted by: Richard Z. on Fri, 03/11/2022

I was diagnosed in March 2021 and very optimistic that new therapys can be approved before this progresses past the point of no return. Please approve AMX 0035 to buy us more quality of life time until a cure can be discovered,

Submitted by: Laren L. on Fri, 03/11/2022

I have ALS. I probably had it for about a year, before I was diagnosed in February 2021. I fall a lot and I keep getting weaker. I am on Riluzole, twice a day. I would love to get my hands on AMX0035. It is available in Canada! Why not here? I see commercials for all kinds of new drugs for serious medical diseases. I see none for ALS. Thank you for reading this!

Submitted by: maryellen R. on Fri, 03/11/2022

I was diagnosed with ALS in November 2018, but believe I had symptoms five years before being diagnosed. ALS has impacted my life in negative ways, even though my symptoms have progressed slowly. I have always been open to trying any medications that might slow ALS's symptoms. Certainly I would be willing to try a trial of AMX0035. It may not help me, but may help someone else.

Submitted by: Jeny S. on Sun, 03/13/2022

Please get an approval for this sweet family. Eric is a great guy, amazing husband and dad. Another amazing part of him, even Being sick he is still trying to work with the kids at Ogden high, he has done so much for their athletic program because of his care for the kids and for them to succeed. But most of all his wife, children, mom, dad, sisters, brothers, nieces, and nephews need him in their lives. Please approve this so he can enjoy life longer with his wonderful family. Thank you

Submitted by: Janie G. on Mon, 03/14/2022

As ALS patients we have very great need for medication available to us. Please approve this so we are given some hope in this horrific journey. I am keeping faith and positivity for a cure not only for myself but for my loved ones and the entire ALS community. We deserve to be given a chance with this medication. We are in dire need. Thank you

Submitted by: Shannon P. on Mon, 03/14/2022

My friend's husband was diagnosed with ALS last year. He was not in the trial but we have read about the promising reviews of this new drug. Patients with ALS have such a short life span we are willing to accept the risks that may come with this drug in the chance that it might prolong his life. Please approve and give us the chance to use it. If it is not approved it might be too late.

Submitted by: Jane B. on Mon, 03/14/2022

Hi. I was diagnosed JANUARY 2020, with symptoms 2 years prior. I've been unable to access trials due to my 2 years of my symptoms.
AMX0035 gives us more hope for a better quality of life.
None of us want to live with feeding tubed or breathing machines.
Although our numbers diagnosed due not compare to cancer, our lives are just as important. We didn't ask for or do anything to have this horrible disease.

Submitted by: Tara G. on Mon, 03/14/2022

My father was diagnosed May 3, 2021 and passed November 4, 2021. He lived only 6 months after being diagnosed. Please approve this drug. We can help defeat ALS together.

Submitted by: Brenda H. on Wed, 03/16/2022

Having a friend who is suffering from this awful disease, I urge the Right To Try to be implemented even before FDA approval .

Submitted by: Tara H. on Thu, 03/17/2022

Suffering patients AND families dealing with neuromuscular diseases, like ALS, need access to all available medical treatments at an affordable cost.

Submitted by: Carmelo R. on Wed, 03/23/2022

Fui diagnosticado con ela en diciembre 2021 espero aprueben este medicamento amx00035 es algo necesario para nuesta calidad de vida. Y la tranquilidad de nuestro cuidadora y familiares,, vivo en Puerto Rico y no nos permiten entrar a ninguna prueba

Submitted by: Evelyn C. on Mon, 03/28/2022

Living w ALS since November 2019 I am searching for all treatment that can help slow progression of this terrible disease

Submitted by: ken L. on Thu, 04/14/2022

My brother has ALS and I welcome any opportunity that would allow his life expectancy to be increased. Seems as though this has been proven safe and should be made available to as many people as possible - as quickly as possible.

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