Just last week the FDA reversed its decision on Amylyx's ALS drug AMX0035. In April, the FDA requested additional phase 3 placebo-controlled trials. Why the recent change of heart by the FDA? Did they just wake up one morning last week and realize how absurd their request for an additional placebo-controlled trial in this patient population is? No, patients, caregivers, families, advocates, organizational support, etc. all stepped up. Letters, emails, phone calls, meeting requests etc. applied enough pressure for the FDA to reconsider.
An individual diagnosed with ALS has a prognosis for survival of 2 to 5 years. It is a death sentence and every day is precious. These people are fathers, mothers, spouses, children, grandparents. They are simply human beings who want the ability to fight for more time with their loved ones. It took 5 long months before the FDA has a change of heart and now decides it is satisfied with the strength of the earlier phase 2 data. 5 months is an eternity for anyone with ALS. Ask yourself, how outraged you would be watching a loved one suffer this debilitating disease while watching an FDA with no sense of urgency.
A placebo-controlled trial? How ethical is that? Imagine the sense of hope, an ALS sufferer and their families have finding out that they are eligible for a clinical trial to then be placed in the placebo group. You find out that the drug showed efficacy only to later also find out that you were in a placebo group. If you are fortunate to be in the treatment group that showed response then you no longer have access to the treatment once the trial is over. Instead, you now wait for the FDA's lengthy process of approval which could take anywhere from months to years. Imagine the outrage that you would feel, this is all so wrong and sickening!
The FDA should expand the right to try and emergency use authorization. These people are staring death in the face on a daily basis! I think any of us in their unfortunate situation would drastically change our risk tolerance. Yet, they are not allowed to at the very least try and fight. I understand the need to ensure public safety and health but what options do these people have at this point.
The FDA's erratic decision-making clearly shows an agency with no direction and a lack of leadership. There is a severe disconnect between the FDA and the public, medical, and scientific communities. In July, I watched as Dr. Cavazzoni testified during a congressional hearing, I remember thinking to myself that she could use someone whispering in her ear to show some occasional compassion. These people within the FDA are really cut from a different cloth. They may have brains but definitely, no heart. They can learn something from the countless caregivers who interact with patients on a daily basis. It's not at all about the patients, it's about checking off boxes, red tape, data collection, and upholding their so-called "gold standard" with no real sense of urgency in helping those suffering these horrendous diseases.
The FDA loves to remind people of the great clinical advancements made in the treatment of HIV and AIDS. What it fails to mention is its lack of urgency as it waited while thousands of people died. Patients, caregivers, families, and advocacy groups had to beg, plea, and essentially kick the door down before the FDA realized the severity of the situation. This is all nothing new with them. Same old story