Mirum filed an application for the use of LIVMARLI in PFIC patients aged two months and above. Credit: Fé Ngô on Unsplash.

Mirum Pharmaceuticals has submitted a marketing authorisation variation application to the European Medicines Agency (EMA) for expanding the use of LIVMARLI (maralixibat) to treat progressive familial intrahepatic cholestasis (PFIC) patients aged two months and above.

EMA has already approved the oral solution for treating cholestatic pruritus in patients of this age group diagnosed with Alagille syndrome (ALGS).

Go deeper with GlobalData

Data Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

Mirum’s latest application to extend the label for PFIC is based on results from the MARCH Phase lll trial of LIVMARLI in PFIC patients.

The randomised trial enrolled 93 patients across a range of genetic PFIC types, including PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6, as well as those of unknown mutational status.

In the cohort that assessed combined genetic types, patients treated with LIVMARLI showed statistically significant improvements in pruritus, serum bile acids, bilirubin and growth as per weight z-score.

The variation also featured interim analysis results from an ongoing open-label extension trial, called MARCH-ON, which enrolled 85 of the 93 patients who took part in the pivotal trial.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

According to the findings, LIVMARLI showed a maintenance of treatment effect, with a sustained decrease in serum bile acid and bilirubin levels along with improved pruritus.

Throughout the follow-up, growth acceleration was further enhanced to show catch-up growth in PFIC children.

Mirum Pharmaceuticals president and CEO Chris Peetz said: “The expansive data we have collected from our PFIC studies give us great confidence that, if approved by the EMA, LIVMARLI will provide an impactful treatment option for patients across a broad range of genetic types, including those with phenotypic diagnosis but without a known variant.

“We have now submitted these groundbreaking data to regulatory agencies in the US and Europe, with the hope of making LIVMARLI available to PFIC patients across multiple geographies.”