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LIVMARLI (maralixibat) Presentations Show Improvements in Quality of Life Measures in Alagille Syndrome and PFIC2 at NASPGHAN Annual Meeting 2021
- Pruritus response with LIVMARLI treatment is associated with improvements in sleep disturbance in analysis of ICONIC study in Alagille syndrome. - Serum
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":"\n- Pruritus response with LIVMARLI treatment is associated with improvements in sleep disturbance in analysis of ICONIC study in Alagille syndrome.\n\n- Serum bile acid response following LIVMARLI treatment in patients with PFIC2 is associated with clinically meaningful improvements in sleep, fatigue and other health-related quality of life symptoms.\n\n FOSTER CITY, Calif.--(BUSINESS WIRE)--\n\nMirum Pharmaceuticals, Inc. (NASDAQ: MIRM) presented new analyses from LIVMARLI™ (maralixibat) oral solution clinical studies during the North American Society for Pediatric Gastroenterology Hepatology and Nutrition Annual Meeting, taking place virtually December 12-18, 2021. These post-hoc analyses assessed the impact of maralixibat treatment response on changes in health-related quality of life (HRQoL) measures among children with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC) with BSEP deficiency (also known as PFIC2).\n\nResponse to treatment with maralixibat in Alagille syndrome is associated with improved health-related quality of life\n\nThe analysis evaluated 27 patients with ALGS who received 380 µg/kg per day of LIVMARLI and who remained in the ICONIC study1 through Week 48, comparing their HRQoL outcomes as reported at baseline and at Week 48. Treatment response to LIVMARLI was defined as a ≥1 point reduction in caregiver Itch-Reported Outcome (ItchRO) instrument score from baseline to Week 48. In addition, 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.\n\nAt Week 48, 20 patients (74%) met the definition of ItchRO response. Responders showed improved HRQoL measures compared with non-responders:\n\n\nMultidimensional Fatigue Total Scale Score from baseline to Week 48 (increase of 13.9 points) more than two times the minimal clinically important difference in responders.\n\n\nSix sleep-related items demonstrated significantly larger changes from baseline to Week 48 in responders.\n\n\nPedsQL Generic Core Total Scale Score increased on average by 8.8 points, almost two times the minimal clinically important difference in responders.\n\n\nThese data demonstrate that the significant improvements in pruritus seen with LIVMARLI...