Dear FDA Advisory Committee

Below are my comments submitted to the FDA Advisory Committee that will be meeting to address questions related to the Amylyx AMX0035 approval.

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Mom was diagnosed with bulbar-onset ALS 25 years ago at age 78 (after a long delay to diagnosis).  She had a fast case and died in 1997.

One might think that a therapy option that slows the speed of a difficult terminal disease might not be of much use to an elderly person in her situation.  I think that just the opposite is true.

One morning, for the first time ever, she asked me to help her button her blouse.  She had strength in her hands but was losing finger dexterity.  As I buttoned those few buttons, she said, "Do you know what the worst part is?"  I simply said that I did not.  She then said, "Once I lose the  ability to do something, I know I'll never be able to do it again."  

She had buttoned her last button. 

I think we all need to let that soak in.

She took riluzole without any pretense that she was going to beat the grim reaper.  She simply wanted to try to keep the independence and dignity of being able to button a button or take care of her personal hygiene as long as possible.

My understanding is that AMX0035 hit its primary endpoint with statistical significance and has a good safety profile.  It's a combination of substances that the FDA knows well.  We are realistic that not every therapy will work for every person with this wicked, heterogeneous disease. I think AMX0035 belongs in a physician's toolbox to try to help a person with ALS who isn't ready to throw in the towel on buttoning that last button.

Thank you very much for the opportunity to comment at an important regulatory moment.  This is the time to apply both the spirit and the letter of the FDA ALS Guidance.