Latest data on Resmetirom is published, FDA approval is expected in March

[nash2]

Today marks a significant milestone for Madrigal's NASH/MASH program and a major step forward towards securing the first-ever FDA approved treatment for NASH (MASH).

Madrigal's Phase 3 trial of resmetirom highlighted (MAESTRO-NASH): The New England Journal of Medicine (NEJM) today featured Madrigal's Phase 3 trial results of resmetirom.

Approximately 50% of patients treated with resmetirom 100 mg with biopsies at Week 52 showed either NASH resolution or fibrosis improvement. More than 80% of patients with biopsies at Week 52 had either fibrosis reversal or no progression of fibrosis.

Press release issued: Madrigal announced the publication news through a press release this morning: (HERE).

We are on the brink of FDA approval for the first drug treatment for NASH. The decision date for the FDA decision is called the PDUFA date.

The Prescription Drug User Fee Act (PDUFA) date for resmetirom is March 14, 2024. This date is the target by which the FDA intends to complete its review of the drug. Resmetirom has been granted Priority Review status by the FDA, which is reflected in the PDUFA date assignment. Industry believes that the FDA will approve the Madrigal application paving the way for the first drug approved for the treatment of NASH fibrosis.

This is an important event as it will change the dynamics of treatment for advanced liver disease. However, as patients it is important to understand the complexity we will face in this new environment.

A few of the issues we will face are:

The drug is expected to be expensive and payors are reluctant to authorize payment for drugs that may be taken for an extended period.

The drug will not be approved for F4 patients. It was studied for the F2/F3 population.

It is not a magic bullet as documented benefits, greater than placebo, of one stage of fibrosis improvement occurred in about 20% of patients although many patients showed some benefit of no increase in fibrosis during the study. How to determine who is likely to benefit and when to discontinue the drug are complex.

Despite the issues it is important to remember that this is the first drug for this disease. It is proof that we can find life saving treatments. It will likely stimulate more investor interest in the condition and accelerate the development of better treatments. It will also help focus the PCP community on early detection of NASH. Resmetirom isn't perfect but it is a very important step forward for the patient community and our hats are off to Madrigal.

fattyliverfoundation.org/re...

[Cats2018]

Hi Wayne,

I may have told you previously that I participated in the Madrigal trials of Resmetirom. The 1st year I was in the trial I was not told if I was on the drug or placebo. The final year I participated I was definitely on the Resmetirom and saw improvement in my FibroScan at the end of the trial year. I definitely want to be able to start Resmetirom again as soon as FDA approves it, but I’m afraid that my insurance will probably not cover it & it will be too expensive. I’ve been off the drug since November 15, 2022. I hated to have to go off the drug at the end of the trial.

Char

[nash2 Partner in reply to Cats2018]

Remind me after approval. We will have to see if they will be willing to help prior trial participants.

[ThyroidDeb in reply to Cats2018]

I remember you saying you were in the trial, but i don't remember any details. How did it go for you, obviously it seemed to help but did you have any side affects? I hope Wayne can help if approved, I think if you risked a trial you should be rewarded and get free meds.

[Cats2018 in reply to ThyroidDeb]

It went well for me and I did not have any symptoms that were noticeable. I hated to see have to stop the Resmetirom!

[Alterity in reply to Cats2018]

Usually when in a trial and it is FDA approved the trial people get it free for life or at least a huge discount, let's hope you get it. The trials I have been on have failed.

[Cats2018 in reply to Alterity]

I used to think that was the case too, but apparently it is not guaranteed. I am hoping that it does end up being at least discounted for trial participants. I’m sure insurance isn’t going to cover a newly approved drug. Fingers crossed! 😊

[Livmar]

This is wonderful news! I thought Madrigal was also involved in an Open Label Arm Study for “MAESTRO-NAFLD-1 open label arm” for the assessment of Resmetirom on more advanced cirrhotic patients. Is this still ongoing for another couple of years?

[Cats2018 in reply to Livmar]

When my trial ended, I’d had 2 years with Madrigal. I was hoping to be offered a 3rd year, but you are correct that Madrigal extended the trial for participants who already had cirrhosis, They decided not to extend for NASH participants, only cirrhotic participants.

[Livmar in reply to Cats2018]

Hi Cats2018, thanks for confirming. Yes I believe it was for well compensated cirrhosis patients. I would think that has 1 or 2 more years to go in the trial.

[Robboref in reply to Cats2018]

Hi Cats, I’m into the third year of the trial and I haven’t been told that I have cirrhosis. I also feel really good with no side effects. It’s still unknown if I’m on drug or placebo. Regards Wyn..

[Cats2018 in reply to Robboref]

Hi! I was in Madrigal14 and Madrigal18. Did you start in Madrigal11? I was not accepted into Madrigal11 because I had ballooning indicated in my liver biopsy. But they did accept me in Madrigal14 and 18 fortunately. Good luck Wyn!

[Robboref in reply to Cats2018]

Thanks for your reply. Yes I did start in madrigal 11 on 19th January 2021. Hope your well and I will keep up with any changes. Regards Wyn.

[Cats2018 in reply to Robboref]

Hi Wyn,

I remember how disappointed I was when I was not accepted for Madrigal 11! So then they told me I could apply to Madrigal 14 and I was accepted to that trial for 1 year not knowing if I was on Placebo or Resmetirom. Then I rolled over to Madrigal 18 and in that trial I was definitely on the drug. I took the low dose for 3 months and the highest dose for 9 months. My FibroScan definitely improved during that year. I hope you are on the Resmetirom and continue to do well! Char

[Robboref in reply to Cats2018]

Hi Char, to be honest I currently feel the best I have for a long time, even going back to referee football games (at my age). Strangely though my fibroscan results are going up every time. Wyn..

[ThyroidDeb]

Hi Wayne, wonderful news but I was wondering..and this might be a stupid question, if it might help in stopping advancement of fibrosis why they left out F1 category but let's say it gets approved and insurance pays, would that category get left out. .am I making sense???

[nash2 Partner in reply to ThyroidDeb]

In the study they only used patients with a subset of the F1 bracket, it is called F1B and is for patients in the upper part of the range, so selection is difficult. There is discussion about including F1 but for the early stages simplifying patient selection looks to be the preferred strategy. I imagine F1 will be included at some point as there will be pressure to do that.

[Golendoodle]

Wayne will they go by fibroScans to see who should take the medication? Also, is this drug for life or once your fibroscan shows no more fibrosis you can stop taking the medication? I also wonder once you stop taking the dedication does the fibrosis come back? I wonder if we will always be at high risk and will have to continue to watch everything we eat. Sorry for so many questions but I can’t find any of these answers about the medication online.

[Ilovejoe]

Thank you so much for this great news!

[LaurieRose]

That's great news. Not available in UK because of profit margins or something. But I'm happy for those who can receive this new treatment.