Data From Mirum Pharmaceuticals' ICONIC Study Evaluating Livmarli in Patients with Alagille Syndrome Published in Journal of Pediatrics
19 October 2022 - - Data highlighting quality of life and sleep improvements from US-based Mirum Pharmaceuticals, Inc's (NASDAQ: MIRM) pivotal ICONIC study evaluating Livmarli (maralixibat) oral solution in patients with Alagille syndrome, were published in the Journal of Pediatrics, the company said.

ALGS is a rare genetic disease caused by abnormalities in bile ducts that can lead to progressive liver disease.

Malformed or reduced bile ducts cause retention of bile acids in the liver leading to cholestasis and ultimately inflammation and liver injury which prevents the liver from working properly.

Cholestasis in ALGS leads to a debilitating pruritus impacting the quality of life so severely that pruritus is a leading indication for liver transplantation.

The analysis evaluated 27 patients with ALGS who received 380 µg/kg per day of maralixibat and who remained in the pivotal ICONIC study through week 48.

The analysis compared health-related quality of life (HRQoL) outcomes from baseline and week 48 based on treatment response to maralixibat, defined as a ≥1-point reduction in pruritus (measured by Itch-Reported Outcome Observer (ItchRO[Obs]) where a 1-point reduction is clinically meaningful).

The Pediatric Quality of Life (PedsQL) questionnaires (Generic Core PedsQL, Family Impact Scale, and Multidimensional Fatigue Scale) were prospectively collected via a caregiver proxy report and analyzed retrospectively.

Pruritus treatment response at week 48 was associated with clinically meaningful improvement in HRQoL from baseline to week 48.

Across all domains evaluated, the average increase in responders' HRQoL scores from baseline to week 48 exceeded the minimal clinically important difference, compared to non-responders.

Responders experienced a significantly greater change in Family Total Impact Scale Score and Multidimensional Fatigue Total Scale Score, and a numerically higher increase in PedsQL Generic Core Total Scale Score compared with non-responders (where higher scores indicates a better HRQoL).

Importantly, six sleep-related items demonstrated significantly larger changes from baseline to week 48 in responders (p