Mirum's Livmarli clinches second nod in EU for rare liver disease
09 Jul 2024
Mirum Pharmaceuticals on Monday said the European Commission approved Livmarli (maralixibat) oral solution to treat progressive familial intrahepatic cholestasis (PFIC) in patients aged three months and older. The decision marks the ileal bile acid transporter (IBAT) inhibitor’s second indication in the EU after its initial approval for Alagille syndromeAlagille syndrome in 2022.
The European Medicines Agency’s (EMA) orphan drug advisory body also adopted a favourable opinion about maintenance of the drug’s orphan drug designation in PFIC.
The expanded approval, which follows a positive opinion by the EMA’s drug advisory panel in May, was backed by findings from the Phase III MARCH trial of 93 patients with multiple PFIC genotypes, including those with unidentified mutations. Results showed significant reductions in pruritus and serum bile acid, alongside improvements in total bilirubin and growth with Livmarli versus placebo in a combined genotype cohort.
The IBAT inhibitorIBAT inhibitor was first approved in the US for Alagille syndrome in 2021 and recently for PFIC in patients aged five years and older. Meanwhile, the company is also eyeing FDA approval of a higher-strength version for younger patients with PFIC, which could be granted later this year.
The drug is likely to face competition from Ipsen’s Bylvay (odevixibat), which was cleared by the EU regulator to treat PFIC in 2021. Livmarli generated $141.8 million in global sales last year, amounting to nearly 90% growth over the previous year.
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