FDA ap­proves Mirum Phar­ma's first treat­ment for rare pe­di­atric bile duct dis­ease

The FDA on Wednes­day ap­proved Mirum Phar­ma­ceu­ti­cals’ first drug, Liv­mar­li (mar­al­ix­i­bat), which is al­so the first treat­ment for life-threat­en­ing cholesta­t­ic pru­ri­tus in young pa­tients with the su­per rare Alag­ille syn­drome (AL­GS).

AL­GS is caused by ab­nor­mal­i­ties in bile ducts and can lead to pro­gres­sive liv­er dis­ease, trans­plants and po­ten­tial­ly death, ac­cord­ing to Mirum. An IBAT in­hibitor, Liv­mar­li works by block­ing bile acid ab­sorp­tion in the small in­testines, lead­ing to low­er lev­els of cir­cu­lat­ing bile acids oth­er­wise linked to se­vere symp­toms, in­clud­ing a se­vere and un­re­lent­ing itch.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER