Recent data from Mirum's lead asset maralixibat in the pediatric liver diseases Alagille syndrome, or ALGS, and progressive familial intrahepatic cholestasis
, or PFIC, suggest the mixed results from prior studies were a result of underdosing, Seedhouse said in a Monday initiation note.
Albireo's lead product candidate, odevixibat (A4250), is being developed to treat rare pediatric cholestatic liver diseases and is in Phase 3 development in its initial target indication, progressive familial intrahepatic cholestasis
. Albireo's clinical pipeline also includes two Phase 2 product candidates.
7, 2019 (HealthDay News) -- For women with intrahepatic cholestasis
of pregnancy, treatment with ursodeoxycholic acid does not reduce adverse perinatal outcomes, according to a study published online Aug.
In a paper published in the journal 'The Lancet', it was found that 605 pregnant women with intrahepatic cholestasis
of pregnancy (ICP) were recruited with half receiving ursodeoxycholic acid (known as 'urso'), the current drug used to treat the condition, and half a placebo.
Biopharmaceutical company Mirum Pharmaceuticals stated on Tuesday that it has dosed the first paediatric patient with progressive familial intrahepatic cholestasis
(PFIC) under the Phase 3 MARCH-PFIC clinical trial of its lead drug candidate maralixibat, with topline results in fourth quarter of 2020.
The odevixibat PEDFIC program in progressive familial intrahepatic cholestasis
, PFIC, consists of two clinical trials.
She was delivered at 40 weeks gestation with a birth weight of 2020 g and presented at two weeks of life with significant neonatal cholestasis
. She had persistent jaundice and mild hepatomegaly with laboratory investigations showing evidence of conjugated hyperbilirubinemia [peak conjugated bilirubin 193 micromol/L, (normal range: 0-2 micromol/L)] and marked elevation of liver enzymes [peak aspartate aminotransferase (AST): 3633 U/L, (normal range: 0-110 U/L), peak alanine aminotransferase (ALT): 2120 U/L, (normal range: 0-60 U/L) and peak gamma-glutamyl transferase: 106, (normal range: 0-45 U/L)].
IFALD results in hyperbilirubemia, steatosis, cholestasis
, and hepatic fibrosis (Onishi et al., 2016).
Arthrogryposis, renal tubular dysfunction, cholestasis
(ARC) syndrome was originally described in 1973 by Lutz-Richner and Landolt (1) severe growth retardation, ichthyosis, recurrentfebriledisease, plateletabnormalities, sensorineural hearing loss, hypotonia and corpus callosum dysgenesis were later included as further features of the syndrome (1,2).
(NC) is a rare clinical condition characterized by decreased bile drainage and direct hyperbilirubinemia (1).
Persistent neonatal cholestasis
is a major clinicopathological concern affecting almost1 in 2500 infants and is an important diagnostic challenge1.
Vikki Meredith suffered from a liver condition called intrahepatic cholestasis
of pregnancy (ICP), and as a result had one of the worst cases of itching.