companies are not obliged to provide a drug under right to try. However Amylyx is patient friendly. If it goes phase 3 then I think they will have an expanded access program.
TUDCA ,one component , is available online The other component is prescription and very expensive. It almost certainly would require prior authorization from your insurance which is very difficult for an unapproved use
right to try and expanded access do not mean the drug is free under these programs
I hope we will soon know what the plan is. The company has been in active talks with the fda. Be aware if he has any interest in a clinical trial taking either or both components of this will be a trial exclusion until/ unless it is approved
if it goes to phase 3 there will likely be expanded access. I am not sure what would happen if he asked for expanded access if he was eligible for phase 3. Ask his neurologist if there is a route that will work. ( like the tudca plus pb prescription). They will know if right to try is possible. I think amylyx will be taking the expanded access and phase 3 route if not approved but of course I don’t know
if a trial isn't in question he could order the tudca part today if he isn’t already on it
was in an als phase 3 trial at emory a few years ago (8 or so years ago) and was told the meds would be provided cost free to trial participants if the drug proved effective. unfortunately, the drug proved ineffective. however, the scope of blood work taken during each visit told me more about myself than i knew was possible. overall, i would do it again if i were at that same stage in life.
One criterion for expanded access is that patients are not eligible for clinical trials with the agent in question, so as not to compromise trials that are required for approval/marketing. The RTT Act essentially overlapped with EA though with a different approval structure, and was a show pony. As Nikki says, none of these are free and AMX0035 would not be cheap.
On the other hand, TUDCA is still being studied on its own, bearing in mind that it targets one pathway and the AMX0035 compound targets two. Here is one protocol if you want the dosage.
will Amylyx Reverse ALS,, if my father has lost his ability to move hands or feet, and problem swallowing and he has lost 1 eyesight and have hard time speaking. i do not want to see him suffer , but i am not ready to loose him its only been 1 year since he was diagnosed with ALS. can i buy it from US ? if we live in Canada. please and thank you
I am brand new to all of this, with my husband diagnosed only a week ago with Bulbar ALS. It seems to me his progression is very fast, going from “perfectly normal” to having tremendous speech, swallow and respiratory problems in just 3 mos.
My question: Is AMX0035 available outside the US?