The US Food and Drug Administration (FDA) has approved maralixibat (Livmarli®) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) aged 3 months or older, Mirum Pharmaceuticals announced.
The medication was previously approved for the treatment of patients aged 1 year or older. The reduction in age is based on positive safety and tolerability data obtained during the RISE study in infants under 1 year of age, according to a press release from Mirum.
“The vast majority of patients are diagnosed with ALGS before one year of age. The availability of Livmarli will offer an opportunity to introduce a treatment at the beginning of their ALGS journey with the goal of reducing serum bile acids and alleviating the unrelenting burden caused by pruritus,” said Chris Peetz, president and chief executive officer at Mirum.
The novel medication is currently being investigated in late-stage clinical studies of other rare cholestatic liver diseases such as biliary atresia.
Read more about ALGS therapies
Treatment with maralixibat might cause side effects, including fat-soluble vitamin deficiency, changes in liver tests indicative of a serious liver injury, and gastrointestinal problems such as diarrhea, stomach pain, and vomiting.
Maralixibat is an ileal bile acid transporter inhibitor and is the only FDA-approved medication for the treatment of cholestatic pruritus associated with ALGS. It is administered once per day as an oral solution.
Mirum announces label expansion for LIVMARLI in the United States to include infants three months of age and older. News release. Mirum Pharmaceuticals; March 14, 2023.