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Alagille Syndrome
Conference Roundup
Progressive Familial Intrahepatic Cholestasis

Long-term benefits of maralixibat in PFIC demonstrated in 2-year study

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Maralixibat (MRX) demonstrates significant and sustained improvements in pruritus, serum bile acids (sBA), total bilirubin (TB), and growth in patients with progressive familial intrahepatic cholestasis (PFIC) over a 2-year period, according to a study presented at The Liver Meeting 2023. The study suggests that MRX treatment contributes to overall improved liver health across a broad range of genetic PFIC types and can be effectively maintained long-term.

Maralixibat has shown promising results in the 26-week MARCH Phase 3 study.

In the MARCH-ON study, an open-label, long-term extension of MARCH, researchers evaluated the long-term effects of MRX in 2 groups: those initially randomized to MRX in MARCH (MRX-MRX group) and those who received a placebo in MARCH and switched to MRX in MARCH-ON (PBO-MRX group).

Results from the MRX-MRX group demonstrated sustained improvements in pruritus, sBA, tb, and growth over the 2-year period. Notably, significant improvements observed in the first 26 weeks of the MARCH study were maintained through Week 104 in MARCH-ON. The PBO-MRX group also showed substantial improvements, aligning with observations from the initial MARCH MRX group.

Key findings include a reduction in pruritus, sBA, and TB, as well as positive growth outcomes in both groups. No new safety concerns were identified, with gastrointestinal-related adverse events being the most common, primarily mild and transient diarrhea.

Reference
Arikan C and Ovchinsky N. Long-term maintenance of response and improved liver health with maralixibat in patients with progressive familial intrahepatic cholestasis (PFIC): 2-year data from the MARCH-ON study. Presented at: The Liver Meeting 2023.

Rare Disease 360® is the Official Media Partner and Official Publication of The Alagille Syndrome Alliance (Alagille.org).

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