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.