- Commercial-stage biotech Mirum Pharmaceuticals ( NASDAQ: MIRM ) announced Monday that its FDA-approved therapy Livmarli reached the main goal in a pivotal Phase 3 trial for rare liver disease progressive familial intrahepatic cholestasis (PFIC).
- Livmarli is an oral therapy indicated in the U.S. to treat cholestatic pruritus in patients with rare and genetically driven liver disease Alagille syndrome (ALGS).
- According to the topline data from the 93-patient study named MARCH, Livmarli, also known as maralixibat, met the primary endpoint of improvement in pruritus severity with statistical significance in PFIC2, which accounts for nearly 60% of PFIC cases.
- The most common treatment-emergent adverse event diarrhea was found to be mild with no severe cases and lasted a median of 5.5 days.
- The company plans to share these results with regulators and expects to submit more data from the MARCH study at a future scientific conference.
For further details see:
Mirum says liver disease therapy met main goal in late-stage study