FOSTER CITY, Calif. - Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) has released data indicating a significant improvement in long-term outcomes for patients with Alagille syndrome (ALGS) treated with LIVMARLI® (maralixibat) oral solution.
The findings, published in the journal Hepatology, reveal a 70% reduction in the risk of clinical outcomes, including liver transplantation, for patients using LIVMARLI compared to a natural history control group.
The research evaluated a six-year period and is the first to show a transplant-free survival benefit in ALGS patients using a pharmacological therapy. The study compared 84 patients treated with LIVMARLI against a control cohort of 469 patients from the Global Alagille Alliance (GALA) database, which serves as the largest global ALGS natural history database.
According to Dr. Binita M. Kamath, senior author of the manuscript, the analysis is pivotal in demonstrating the potential of LIVMARLI to reduce the need for liver transplantation in ALGS, a rare genetic disorder that affects the bile ducts and can lead to progressive liver disease.
The pre-specified statistical analysis, conducted by Dr. Bettina Hansen from Erasmus MC, Netherlands, showed a notable improvement in six-year event-free survival with a p-value of <0.0001. This translates to a 70% overall reduction in clinical outcomes for patients treated with LIVMARLI. The study also included multiple sensitivity and subgroup analyses to ensure the robustness of the results.
LIVMARLI, an ileal bile acid transporter inhibitor, is currently the only medication approved by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in ALGS patients aged three months and older. It is also approved by the European Commission for the treatment of the same condition in patients aged two months and older.
Mirum Pharmaceuticals has a focus on rare diseases affecting both children and adults, with three approved medications including LIVMARLI. The company has submitted LIVMARLI for approval in the U.S. and Europe for cholestatic pruritus in PFIC patients, a related liver condition.
The study's findings are based on a press release statement and provide new hope for ALGS patients, potentially reducing the need for liver transplants and improving quality of life. The full publication with additional data is available on the Hepatology website.
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