FDA approves first treatment for patients with NASH with fibrosis

The US Food and Drug Administration (FDA) yesterday approved Rezdiffra (resmetirom) for the treatment of adults with non-cirrhotic non-alcoholic steatohepatitis (NASH) with moderate to advanced liver scarring (fibrosis), to be used along with diet and exercise.

The FDA granted the approval to Madrigal Pharmaceuticals (Nasdaq: MDGL), whose shares shot up 28% to $311.75 pre-market, and marked the first time the agency has authorized a new treatment for NASH.

The FDA authorized Rezdiffra under the accelerated approval pathway, which allows for earlier approval of drugs that treat serious conditions and address an unmet medical need, based on a surrogate or intermediate clinical endpoint that is reasonably likely to predict clinical benefit.  Continued approval for this indication may be contingent upon verification and description of clinical benefit in ongoing confirmatory trials, which \madrigal says are ongoing.