Maralixibat treatment leads to a statistically significant improvement in 6-year event-free survival in patients with Alagille syndrome, compared to a natural history control cohort, according to a new analysis of data. The events were defined as biliary diversion surgery, liver transplant, hepatic decompensation, or death.

Mirum Pharmaceuticals, the developers of maralixibat, therefore submitted a marketing authorization application to the European Medicines Agency (EMA) for the treatment of cholestatic disease in Alagille syndrome, according to a new press release from the company.

The US Food and Drug Administration (FDA) has accepted the company’s New Drug Application for maralixibat for the treatment of cholestatic pruritus in patients with Alagille syndrome.

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“The six-year event-free survival data, coupled with the previously presented ICONIC data, provides a catalyst to accelerate our [Alagille syndrome] submission,” said Chris Peetz, president and chief executive officer at Mirum. He added “We feel a tremendous urgency to advance maralixibat for patients as quickly as possible as its availability may provide a significant shift in treatment options for patients living with this unrelenting rare liver disease.” 

The ICONIC study was a phase 2b study, which showed that patients with Alagille syndrome treated with maralixibat had significant reductions in bile acids and pruritus compared to those who were given a placebo. Results from the ICONIC study were compared to the natural history cohort in this new data analysis.

Maralixibat is a new experimental oral treatment that is minimally absorbed. It is being evaluated as a potential treatment for different rare cholestatic liver diseases including Alagille syndrome. Maralixibat works by inhibiting the apical sodium-dependent bile acid transporter (ASBT), with an outcome of causing more bile acid to be excreted in the feces. The systemic levels of bile acid are therefore reduced, potentially leading to less damage due to high bile acid levels. 

Maralixibat is currently available for patients with Alagille syndrome through Mirum’s expanded access program.


Mirum Pharmaceuticals submits European marketing authorization application for maralixibat in Alagille syndrome supported by new positive results from natural history study comparison. News release. Mirium Pharmaceuticals; September 13, 2021.