FDA approves Livmarli for pruritus in progressive familial intrahepatic cholestasis
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The FDA has approved Mirum Pharmaceuticals’ Livmarli oral solution for treatment of cholestatic pruritus in patients aged 5 years and older with progressive familial intrahepatic cholestasis, according to a company release.
Livmarli (maralixibat), a once-daily, ileal bile acid transporter, is also approved for the treatment of cholestatic pruritus associated with Alagille syndrome in infants aged 3 months and older. The drug was originally approved by the FDA in 2021 for those 1 year and older, and then the prescribing age was reduced to include patients as young as 3 months old in 2023.
“Livmarli’s approval in cholestatic pruritus for patients with PFIC is a result of years of investigation and a collection of a strong body of clinical evidence showing meaningful improvements across a number of important parameters, including pruritus, affecting children with PFIC,” Richard J. Thompson, BM, BCh, MRCP, MRCPCH, professor of molecular hepatology at King’s College London, said in the release. “I am pleased that we will have a well-studied and efficacious option to offer patients whose life has been disrupted by itch.”
The agency based its decision on data from the phase 3 MARCH study, a randomized trial of 93 patients with a range of genetic PFIC types including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6 and unidentified mutational status. Results showed that Livmarli achieved a “highly statistically significant” reduction in pruritus severity compared with placebo, the release 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,” Chris Peetz, CEO at Mirum, said in the release. “Thank you to the patients, families, and clinicians whose study participation made this approval possible.”
According to the release, adverse events associated with Livmarli may include liver injury, gastrointestinal issues and fat-soluble vitamin deficiency.
The company noted it has also submitted an additional supplemental new drug application to introduce a higher concentration Livmarli formulation than was used during the MARCH trial to expand the label to include younger patients with PFIC.
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