VIDEO: Livmarli reduces serum bile acids and pruritus in PFIC, Alagille syndrome
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In a Healio video exclusive, Pam Vig, PhD, highlights new data on Livmarli for the treatment of patients with pruritus due to progressive familial intrahepatic cholestasis and Alagille syndrome, which were presented at EASL Congress.
Vig, head of research and development for Mirum Pharmaceuticals Inc., noted Livmarli (maralixibat, Mirum Pharmaceuticals) is currently approved by the FDA for the treatment of cholestatic pruritus due to Alagille syndrome (ALGS) in patients aged 3 months and older and by the European Commission in those aged 2 months and older.
“What is very important in this setting, as we’ve said many times before, is the symptom burden is real,” Vig said. “These patients go to transplant for the poor quality of life and the pruritus that is such a disturbance to their sleep and in the overall impact of the health and the families.”
According to Vig, patients treated with maralixibat have significant reductions in pruritus and serum bile acids, and improvements in event-free and transplant-free survival. Researchers have also studied the drug’s effect in patients aged 16 years and older, and report maintenance of efficacy through adulthood.
Vig also discussed maralixibat for PFIC and results from the randomized, phase 3 MARCH-PFIC trial, which evaluated its safety and efficacy in children. Key takeaways include:
- Among 64 children with PFIC, maralixibat decreased total/direct bilirubin compared with placebo across all PFIC types. Further, 40% of patients with abnormal baseline bilirubin treated with maralixibat achieved normalization vs. none in the placebo group.
- Maralixibat was associated with complete or near-complete resolution of pruritus in most patients with PFIC, which correlated with improved sleep and quality of life.
- Among a subset of 47 children with PFIC, safety analysis showed maralixibat was well-tolerated with no new safety signals.
Researchers assessed the safety and efficacy of maralixibat in patients aged 16 years and older with ALGS who either transitioned to adult care (n = 11) or initiated treatment (n = 3). Highlights include:
- Improvements in pruritus and serum bile acid during childhood were maintained into early adulthood.
- Patients receiving maralixibat in early adulthood had improvements in pruritus and serum bile acid, which persisted throughout therapy.
- Maralixibat was well-tolerated and demonstrated a consistent safety and tolerability profile.
“With Livmarli, by reducing serum bile acids and improving pruritus, we’re improving sleep quality and thus the quality of life for these patients,” Vig said. “I think the picture that’s emerging from all of this is the overall health of the patient from quality of life to pruritus control and also to the burden of the central problem of serum bile acids and cholestasis and how that relates to liver health.”
References:
- D’Antiga L, et al. Abstract: OS-072: Maralixibat leads to significant reductions in bilirubin for patients with progressive familial intrahepatic cholestasis: Data from MARCH-PFIC. Presented at: EASL Congress; June 21-24, 2023; Vienna (hybrid meeting).
- Kamath B, et al. Abstract: WED-257: Impact of maralixibat on cholestatic pruritus in adults aged 26 years and older with Alagille syndrome. Presented at: EASL Congress; June 21-23, 2023; Vienna (hybrid meeting).
- Miethke A, et al. Abstract: WED-282: Analysis and safety in maralixibat-treated participants with progressive familial intrahepatic cholestasis: Data from MARCH-PFIC. Presented at: EASL Congress; June 21-23, 2023; Vienna (hybrid meeting).
- Mirum Pharmaceuticals to present new Livmarli data at the 2023 EASL Congress. https://ir.mirumpharma.com/news-events/News/news-details/2023/Mirum-Pharmaceuticals-to-Present-New-LIVMARLI-Data-at-the-2023-EASL-Congress/default.aspx. Accessed June 22, 2023.
- Thomas RJ, et al. Abstract: LBP-035: Long-term maintenance of response and improved liver health with maralixibat in patients with progressive familial intrahepatic cholestasis (PFIC): Data from the MARCH-ON study; WED-252: Maralixibat leads to significant reductions in pruritus and Improvements in sleep for children with progressive familial intrahepatic cholestasis: Data from MARCH-PFIC. Presented at: EASL Congress; June 21-23, 2023; Vienna (hybrid meeting).
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