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Progressive Familial Intrahepatic Cholestasis

Maralixibat demonstrates significant improvements beyond pruritus control in PFIC study”

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The Phase 3 clinical trial, MARCH-PFIC, demonstrates the significant positive impact of maralixibat (MRX) on the quality of life (QoL) for patients with progressive familial intrahepatic cholestasis (PFIC). The study focused on the reduction of pruritus, a challenging symptom of PFIC, and found that patients receiving MRX experienced clinically significant improvements in various domains of QoL, including social and physical functioning, as well as positive effects on their families.

Participants in the MARCH trial, were randomly assigned to receive either MRX (570 µg/kg BID) or a placebo over a 26-week period. The study utilized various instruments all measured on a 0-100 scale, with higher scores indicating better QoL.

The results found that MRX recipients experienced a significant change from baseline (CFB) in Pediatric Quality of Life Inventory (PedsQL) for pruritus responders, with a score increase of 22 (P < 0.0001). Conversely, non-responders showed minimal change (-2; P = 0.7062), and the relative change between the groups was statistically significant (P = 0.0014). Similar patterns were observed in PedsQL-Social Functioning, PedsQL- Physical Functioning, and Family Impact Total Scale, with MRX demonstrating significant CFB for social and physical functioning and family impact among pruritus responders.

Reference
Aqul AA, et al. Improvements in pruritus with maralixibat are associated with improved quality of life for patients with Progressive Familial Intrahepatic Cholestasis: Data from the MARCH-PFIC trial. Presented at: The Liver Meeting 2023.

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