VIDEO: Treatment with Livmarli yields ‘broad base effects’ across range of PFIC types

BOSTON — In a Healio video exclusive, Pam Vig, PhD, reports late-breaking data that show Livmarli improved liver health and maintained symptom response over 2 years among pediatric patients with progressive familial cholestasis.

“MARCH was the largest phase 3 study ever to be conducted in this rare disease of PFIC, which encompasses groups of different genetic mutations that really affect bile acid composition and transport,” Vig, head of research and development for Mirum Pharmaceuticals Inc., said. “As a result, these children have significantly elevated bile acids which affect liver health.”

She continued: “In fact, some of these patients will be listed for liver transplant and receive a liver transplant, even in the absence of disease progression, but disease does progress as a result of these toxic bile acids that can accumulate in the liver.”

According to results presented at The Liver Meeting, patients receiving Livmarli (maralixibat, Mirum Pharmaceuticals), an ileal bile acid transporter inhibitor, achieved pruritus control with “clinically significant improvements” in quality of life.

In addition, most individuals on maralixibat had large reductions in serum bile acid, and one participant with MYO5B deficiency experienced normalization of bilirubin, demonstrating maralixibat’s efficacy across a range of genetic subtypes, including those with TJP2 and MYO5B deficiencies.

Further, patients who continued maralixibat treatment for up to 2 years in the open-label, long-term MARCH-ON study had “significant and sustained” responses in pruritus severity, serum bile acid levels, total bilirubin and growth.

“We’re really pleased with the data set, because the broad base effects that we see across all these genetic types is really critical as we move forward in the treatment paradigm of this disease,” Vig said.

References:

• 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; Nov. 10-14, 2023; Boston (hybrid meeting).
• Horslen SP, et al. Maralixibat leads to significant improvement in cholestatic pruritis for children with progressive familial intrahepatic cholestasis without a genetic diagnosis: Data from the MARCH-PFIC trial. Presented at: The Liver Meeting; Nov. 10-14, 2023; Boston (hybrid meeting).
• Miethke AG, et al. Maralixibat leads to significant improvement in cholestatic pruritis for children with progressive familial intrahepatic cholestasis due to TJP2 or MYO5B deficiency: Data from the MARCH-PFIC trial. Presented at: The Liver Meeting; Nov. 10-14, 2023; Boston (hybrid meeting).
• Thompson RJ, et al. 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; Nov. 10-14, 2023; Boston (hybrid meeting).