Mirum Pharmaceuticals Inc. has announced it has submitted a variation application to the Marketing Authorization for Livmarli (maralixibat) oral solution to the European Medicines Agency (EMA).
The application is to extend the label for progressive familial intrahepatic cholestasis (PFIC) in patients two months of age and older. Livmarli is currently approved by the EMA for the treatment of cholestatic pruritis in patients with Alagille syndrome – another chronic liver condition affecting the bile ducts – in patients two months and older.
Data has come from a phase 3 study of Livmarli called March in patients with PFIC. MARCH is the largest randomized trial conducted in patients with the disease with 93 patients across a broad range of genetic types and unidentified mutational status.
In the cohort evaluating combined genetic types, Livmarli-treated patients had statistically significant improvements in pruritus, serum bile acids, bilirubin, and growth as measured by weight z-score.
The variation also included interim analysis data from the ongoing open-label extension study called MARCH-ON which included 85 of the 93 patients that were enrolled in the pivotal trial. Overall, Livmarli demonstrated maintenance of treatment effect with sustained reductions in serum bile acid and bilirubin levels as well as continuous pruritus improvement.
Growth acceleration was further improved throughout the follow-up indicating catch-up growth in children with PFIC.
“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,” said Chris Peetz, president and chief executive officer at Mirum.
“We have now submitted these ground-breaking data to regulatory agencies in the U.S. and Europe with the hope of making Livmarli available to PFIC patients across multiple geographies. We are grateful to the clinicians and families who participated in the research that has meaningfully advanced treatment for PFIC.”
Livmarli is currently approved in the US and Europe for the treatment of cholestatic pruritus in patients with Alagille syndrome three months of age and older, and two months of age and older, respectively. In addition to the Marketing Authorization variation announced today, Mirum has also submitted a supplemental new drug application to the US Food and Drug Administrarion (FDA) for the treatment of cholestatic pruritus in patients three months of age and older with PFIC. Regulatory submissions outside of the US, Canada and Europe are being pursued by partners and distributors in key markets globally.