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It's Charlie Brown time again with FDA, will they or won't they approve a NAFLD/NASH drug

People who are lost can walk in a circle and get back to where they started. It is a bit like that for the patient community.  In 2020 we were getting ready for the FDA to hold an adcom meeting on the Intercept drug Ocaliva. Adcom is where they take public testimony and as patients we wanted to speak. This was to be the first drug that had met the targets set by FDA for a NAFLD/NASH therapy. We all knew it wasn't a perfect solution but when there is no therapy at all it was a place to start and we were very hopeful.

The FDA punted, decided not to hear from patients and told the company to get more safety data or go suck rocks. That was a crushing blow to Intercept as they had already spent hundreds of millions of dollars doing studies to meet the FDA targets and had succeeded.  It is important to understand that the drug was already being used to treat PBC, another liver disease, so this really wasn't unknown territory.

To their credit, Intercept did go forward with two phase 3 trials and has built what, I think, is the largest body of patients in any NAFLD/NASH trial. One of their trials was for early NASH and the other was stage 4 cirrhosis patients. Once all the results were compiled it was clear that they met the goals for early NASH but not quite for stage 4. Helpful for some late stage patients perhaps but more challenging. Very few companies are trying to deal with stage 4 as it is so difficult.

So the stage is set. Intercept has submitted again to FDA and they have a 6 month window for action so we anticipate a June decision.  We are starting to think about what to say to FDA if they take public comment this time. We have gone round the circle and are once again where we started in 2020.

Just to make this more confusing, Madrigal Pharmaceuticals has completed extensive phase 3 trials and is expected to make its own submission to FDA for its drug Resmetirom this year. This becomes complicated because the trial results suggest that the Madrigal drug will help more patients. It works on a different chemistry and we are very optimistic that this drug will also get FDA approval. The challenge is that even if approved the earliest it could be available is 2024 sometime.

Great we say as patients, more options for our therapy, but we can't help but wonder what FDA does with two drugs now when they stumbled with one last time. There are a lot of patients and treating us is going to be expensive. Might they punt again with improving therapy possibly coming to market? How do the lobbyists for the payers weigh in on this? As patients we know that there isn't a "cure". No single bullet, like a Hep C treatment can save us and we have different versions of this disease so we need alternatives. We wonder if our view matters in this process?

I've been very enthused in the past and felt cast aside by FDA when we went through this before, but this is a very hopeful time for patients.  We have Intercept in the batter's box with Madrigal on deck and it is important to understand that even better drugs are coming through the pipeline. There is some exciting research coming to fruition and it will still take some years for those to mature but NAFLD/NASH will become a treatable condition with multiple useful options for patients.

Like Charlie Brown, we remain ever hopeful that this time they won't take the ball away. Stay tuned.

As a reminder, we would like you to be sure to check this out, we gave a demo of The Wellness League which will be the main patient outreach platform for us in the future. It is a short, 30 minute, description of our strategy for delivering patient support presented at the recent conference of the AASLD.  Click the link below to watch it.



Patient care and therapies are changing fast and keeping you informed is an important part of our job.