FDA expands Livmarli approval for pruritus in PFIC to infants as young as 12 months
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The FDA has updated the label indication for Livmarli for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis, expanding the patient population to include infants aged 12 months and older.
This label expansion includes approval for a higher concentration formula of Livmarli (maralixibat, Mirum Pharmaceutical), a once-daily, ileal bile acid transporter, which was only approved earlier this year for patients aged 5 years or older with pruritus and PFIC.
“The launch of Livmarli in PFIC is going well and we are thrilled that it will now be available for patients 12 months and older,” Chris Peetz, CEO of Mirum Pharmaceuticals, said in a company press release. “PFIC is generally diagnosed when children are young, and initiating treatment quickly after diagnosis will help to ensure they have fewer days suffering from pruritus associated with this rare liver disease.”
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, according to a Mirum release.
This is the second rare liver disease indication for Livmarli following its approval for cholestatic pruritus associated with Alagille syndrome.
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