Mirum’s Livmarli gets second FDA nod for severe itch associated with liver disease

14 Mar 2024
firstwordpharma.com
Clinical ResultDrug ApprovalPhase 2Phase 3
The FDA greenlit Mirum Pharmaceuticals’ Livmarli (maralixibat) for the treatment of cholestatic pruritus in patients aged five years and older with progressive familial intrahepatic cholestasis (PFIC), marking the oral ileal bile acid transporter inhibitor’s second US approval after its initial clearance for Alagille syndromeAlagille syndrome (ALGS) in 2021.
“Livmarli has the potential to have a transformational impact for patients with cholestatic pruritus associated with PFIC, and importantly, offers an option for those patients with the rarest of subtypes,” remarked Mirum CEO Chris Peetz. The company, however, noted that Livmarli is not intended for the treatment of patients with PFIC type 2 characterised by a severe impairment in the bile salt export pump (BSEP) protein.
The expanded label approval was based on findings from the Phase III MARCH trial, involving 93 patients with multiple PFIC genotypes, including those with unidentified mutations. In the study, Livmarli demonstrated significant improvements in pruritus, serum bile acid, bilirubin, and growth compared with placebo in a cohort comprising combined genetic subtypes. The application was also backed by favourable transplant-free survival outcomes seen with Livmarli in the Phase II INDIGO study.
Livmarli is rivalled by Ipsen’s Bylvay (odevixibat), the latter having secured US approvals for both ALGS and PFIC in 2021. Although Bylvay has an edge in PFIC owing to an earlier approval and a younger eligible population from three months old, Mirum hopes to tackle it by seeking approval of a higher-strength version for younger patients with PFIC, later this year.
Alongside the US, Livmarli has been approved for ALGS in the EU, Canada and other countries. The drugmaker has also filed with the European Medicines Agency for approval in patients with PFIC aged two months and older. Livmarli generated sales of $141.8 million last year.
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