FDA Approves First Treatment For Alagille Syndrome-Associated Cholestatic Pruritus From Mirum Pharma

The FDA has approved Mirum Pharmaceuticals Inc MIRM Livmarli (maralixibat) oral solution for cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older.
Livmarli, a minimally absorbed ileal bile acid transporter (IBAT) inhibitor, is the first and only FDA-approved medication in this rare liver disease.
Cholestatic pruritus is the sensation of itch due to nearly any liver disease.
ALGS is an inherited condition in which bile builds up in the liver, resulting in liver damage.
In conjunction with the approval, Mirum received a rare pediatric disease priority review voucher.
Related: Mirum, Takeda Ink Maralixibat Licensing Pact In Japan.
The approval is based on the pivotal ICONIC study and five years of data from supportive studies from 86 patients with ALGS.
Data from ICONIC demonstrated statistically significant reductions in pruritus, one of the most common and arduous symptoms associated with the disease, which was maintained through four years.
Mirum will host an investor call today at 4:30 p.m. ET.
Related content: Benzinga's Full FDA Calendar.
Price Action: MIRM stock is down 4.98% at $17.74 in the after-hour session on the last check Wednesday.