Mirum's Livmarli secures second EU approval for rare liver disease

Mirum Pharmaceuticals announced on Monday that the European Commission has approved Livmarli (maralixibat) oral solution for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged three months and older. This marks the second indication for the ileal bile acid transporter (IBAT) inhibitor in the European Union, following its initial approval for Alagille syndrome in 2022. The European Medicines Agency’s (EMA) orphan drug advisory committee also issued a favorable opinion on maintaining the orphan drug designation for Livmarli in PFIC.

Chris Peetz, CEO of Mirum, expressed optimism about the drug's potential to improve liver parameters and enhance the quality of life for young PFIC patients in Europe. The expanded approval was based on positive recommendations from the EMA’s drug advisory panel in May and supported by data from the Phase III MARCH trial. This trial involved 93 patients with various PFIC genotypes, including those with unidentified mutations. Results indicated significant reductions in pruritus and serum bile acid levels, along with improvements in total bilirubin and growth, in patients treated with Livmarli compared to those receiving a placebo.

Livmarli was initially approved in the United States for Alagille syndrome in 2021 and recently received FDA approval for treating PFIC in patients aged five years and older. Additionally, Mirum Pharmaceuticals is seeking FDA approval for a higher-strength version of Livmarli for younger PFIC patients, which could be authorized later this year.

Despite its promising outlook, Livmarli will face competition from Ipsen’s Bylvay (odevixibat), which the EU approved for treating PFIC in 2021. Last year, Livmarli generated $141.8 million in global sales, representing a nearly 90% increase over the previous year.