
Mirum’s Livmarli becomes first US approved Alagille syndrome treatment
pharmafile | September 30, 2021 | News story | |
Mirum Pharmaceutical’s oral drug has become the first approved treatment for treating itching in patients with a rare liver disorder called Alagille syndrome (ALGS).
ALGS is a rare genetic disorder in which bile ducts are abnormally narrow, malformed and fewer in number, which lead to bile accumulation in the liver.
Mirum Chief Executive Officer, Chris Peetz, told Reuters: “(Itching) is really devastating for children and families dealing with this disease…and ultimately that itching is a driving factor for liver transplant decisions”.
Livmarli works by blocking an important bile acid transport protein on the surface of the small intestine, which directs more bile acids out of the body through feces.
The drug’s approval includes information about serious side effects including changes in liver tests which often indicate liver injury.
Mirum has priced the drug at $1,550 per unit, and expects the per unit annual cost to be about $391,000 for a 17-kilogram child, the average weight of patients in its studies.
The dosing for the drug will be prescribed by physicians based on a patient’s weight.
Estimates have priced Livmarli’s peak sales at $400 million in ALGS indication by 2030, Mirum executives estimated a total market opportunity of about $500 million for the indication.
So far, the closest drug to Livmarli is Albireo Pharma’s oral therapy Bylvay, which is currently being tested in a late-stage study for ALGS.
In July, Bylvay received FDA approval for treatment of another liver disease, progressive familial intrahepatic cholestasis (PFIC), and is priced at $385,000 per year.
Kat Jenkins






