FDA approves Mirum’s LIVMARLI for cholestatic pruritus

14 Mar 2024
www.pharmaceutical-technology.com
Drug ApprovalOrphan DrugClinical ResultPhase 3Acquisition
FDA approves Mirum’s LIVMARLI for cholestatic pruritus
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Source: Pharmaceutical Technology
Mirum Pharmaceuticals’s LIVMARLI (maralixibat) oral solution is now available in the US for cholestatic pruritus in patients with PFIC. Credit: Mirum Pharmaceuticals / Business Wire.
The medication is intended for patients aged five years and above with progressive familial intrahepatic cholestasis (PFIC).
A once-daily, orally administered ileal bile acid transporter inhibitor, LIVMARLI is the sole FDA-approved treatment for PFIC.
The approval is based on findings from the Phase III MARCH clinical trial in 93 PFIC patients.
The randomised trial enrolled people with PFIC genetic types PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6, along with people with unidentified mutational status.
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FDA approves Mirum’s LIVMARLI for cholestatic pruritus
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FDA approves Mirum’s LIVMARLI for cholestatic pruritus
Preview
Source: Pharmaceutical Technology
The FDA, the European Commission and Health Canada have also granted approval for LIVMARLI for the treatment of cholestatic pruritus in patients with Alagille syndromeAlagille syndrome in their respective regions.
In addition to its current approvals, Mirum has filed a supplemental new drug application to the FDA. The aim is to introduce a higher concentration formulation of LIVMARLI, which was utilised in the Phase III MARCH study.
This could expand the treatment’s label for younger PFIC patients later in 2024.
A rare genetic disorder, PFIC results in progressive liver disease that can go on to cause liver failure.
Mirum Pharmaceuticals CEO Chris Peetz stated: “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.
“Thank you to the patients, families and clinicians whose study participation made this approval possible.”
In September 2023, the company concluded the acquisition of the bile acid product portfolio of Travere Therapeutics.
Mirum gained the rights and assets related to Cholbam (cholic acid) and Chenodal (chenodiol).
Travere secured $210m from Mirum, with the potential to receive $235m in sales-based milestone payments.
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