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Alagille Syndrome
Progressive Familial Intrahepatic Cholestasis
Trending Topics

Maralixibat response in PFIC may be dependent on subtype

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Depending on progressive familial intrahepatic cholestasis subtype (PFIC), treatment with maralixibat may produce rapid and sustained reductions in serum bile acid (sBA)levels, according to a study.

In this open-label, Phase 2 long-term study, 33 patients with either FIC1 deficiency (n = 8) or BSEP deficiency (n = 25) received oral once daily maralixibat 266 μg/kg from baseline to Week 72; after Week 72, twice-daily dosing was permitted. Of those with BSEP, 6 had biallelic, protein truncating mutations (t)-BSEP, and 19 had ≥1 nontruncating mutation (nt)-BSEP.

A sBA response, defined as a reduction in sBAs of >75% from baseline or concentrations <102.0 μmol/L, was achieved in 6 patients with nt-BSEP while receiving once-daily dosing and 1 patient while receiving twice-daily dosing. Overall, sBA responders had marked reductions in sBAs and pruritus and increases in height, weight, and Quality of life; all were liver transplant-free after >5 years.

Patients with FIC1 deficiency or t-BSEP deficiency did not have an sBA response.

Overall, treatment was considered well-tolerated.

Reference
Loomes KM, Squires RH, Kelly D, et al. Maralixibat for the treatment of PFIC: Long-term, IBAT inhibition in an open-label, Phase 2 study. Hepatol Commun. 2022;doi: 10.1002/hep4.1980. Epub ahead of print. PMID: 35507739.

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