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Madrigal files Resmetirom with FDA, possibly the first therapy for NASH

Madrigal Pharmaceuticals has released news that it has completed its filing for approval by FDA of its NASH drug candidate Resmetirom.

If you would like to read the official announcement, here is a link to it

Madrigal Pharmaceuticals Completes Submission of New Drug Application Seeking Accelerated Approval of Resmetirom for the Treatment of NASH with Liver Fibrosis | Madrigal Pharmaceuticals.

The Resmetirom phase 3 trials have produced strongly positive results across the board in patients with fibrotic NASH. Resmetirom achieved highly statistically significant results in two key primary endpoints with both daily oral doses, 80 mg and 100 mg, relative to placebo. Specifically:

  1. NASH resolution (ballooning of 0, inflammation of 0-1) and ≥2-point NAS reduction with no worsening of fibrosis
  2. Fibrosis improvement by at least one stage with no worsening of NAS at 80 and 100 mg, respectively)

These endpoints are the targets established by FDA for approval of drugs to treat NASH. 

It has been a long wait for FDA approval of a NASH therapy.  Our most recent hope, Ocaliva by Intercept, was rejected last month because of safety concerns.  This Resmetirom has a lower risk profile because it takes a unique approach as a modified thyroid hormone with fewer potential side effects.  Readers may recall that when we wrote about Ocaliva we commented that a better drug was in the pipeline and we thought we might see two drugs approved this year.  The isn't happening but Resmetirom is that better drug we wrote about. We thought the Intercept drug would be the first approved therapy but expected Resmetirom to quickly replace it.  We were wrong about Oclavia but remain very positive about Resmetirom.

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