Madrigal Pharmaceuticals' Rezdiffra (resmetirom, THR-β agonist) becomes the first US FDA approved drug for the treatment of NASH (MASH)
US FDA have granted accelerated approval for Rezdiffra in conjunction with diet and exercise for the treatment of adults with noncirrhotic NASH with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis).
Approval is based on Phase 3 MAESTRO-NASH trial that evaluated 80 or 100 mg of MGL-3196 vs. Placebo in pts. with NASH and Fibrosis
Primary endpoints:
- NASH resolution (including a reduction in the nonalcoholic fatty liver disease [NAFLD] activity score by ≥2 points) with no worsening of fibrosis An improvement in fibrosis by at least one stage with no worsening of the NAFLD activity score
Results published in NEJM showed that NASH resolution with no worsening of fibrosis was achieved in 25.9% of the pts. in the 80-mg group and 29.9% of those in the 100-mg group vs. 9.7% of those in the placebo group
Rezdiffra is expected to be available to patients in the U.S. in April and will be distributed through a limited specialty pharmacy network.
Focus on 315,000 U.S. Patients seen by ~14K target specialists
- Comes at an annual WAC price of $47,400.
- Co-pay support: $10 co-pays for Commercial patients
- Patient Assistance Program also available for pts. with no insurance or no coverage for Rezdiffra
MAESTRO-NASH remains ongoing as an outcomes study designed to generate confirmatory data that, if positive, will help verify clinical benefit and may support full approval
A second ongoing outcomes trial (MAESTRO-NASH OUTCOMES) is evaluating progression to liver decompensation events in patients with well-compensated NASH cirrhosis treated with Rezdiffra vs. placebo.
