regulatory
confidence high
sentiment positive
materiality 0.65
Mirum's LIVMARLI gets positive CHMP opinion for PFIC; EC decision in Q3 2024
Mirum Pharmaceuticals, Inc.
- CHMP adopted positive opinion for LIVMARLI (maralixibat) to treat PFIC in patients aged 3 months and older.
- CHMP concluded LIVMARLI provides significant clinical benefit vs already-approved PFIC treatment.
- COMP recommended maintaining Orphan Drug Designation for LIVMARLI in PFIC.
- European Commission decision on marketing authorization expected in Q3 2024.