European Commission approves Livmarli for patients with Alagille syndrome
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Mirum Pharmaceuticals announced the European Commission has approved Livmarli for the treatment of cholestatic pruritis in patients with Alagille syndrome.
“Livmarli’s (maralixibat, Mirum) approval signifies the first and only approved treatment for Alagille syndrome (ALGS) in Europe; it is currently also approved in the U.S. and Israel,” Chris Peetz, president and CEO of Mirum Pharmaceuticals, told Healio. “ALGS is a rare and devastating liver disease impacting 1 out of every 30,000 people globally, with no treatments to address the severe and unrelenting itch that some patients describe as being ‘set on fire.’ The most burdensome symptom associated with ALGS, pruritus, can often lead to liver transplantation.”
Peetz noted this approval follows positive study results from ‘more than 6 years of data’ which showed livmarli induced and sustained ‘statistically significant and clinically meaningful’ reductions in pruritis compared with placebo among a cohort of patients with ALGS-associated cholestatic pruritis. Data also demonstrated improved growth and quality of life.
“We are thrilled to know that families dealing with debilitating itch associated with ALGS will now have a treatment option,” Roberta Smith, president of the Alagille Syndrome Alliance said in the press release. “Patients who have been prescribed LIVMARLI in the U.S. have experienced tremendous benefit, and we are thrilled that families in Europe will get to experience the same.”