|
 "(Itching) is really devastating for children and families dealing
with this disease...and ultimately that itching is a driving factor
for liver transplant decisions," Chief Executive Officer Chris Peetz
told Reuters.
Mirum priced the drug, to be made available for distribution
immediately under brand name Livmarli, at $1,550 per unit. It
expects the per unit annual cost at 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.
SVB Leerink analyst Mani Faroohar told Reuters that Livmarli could
easily reach peak sales of $400 million in ALGS indication by 2030.
Mirum executives estimated a total market opportunity of about $500
million for the indication.
Faroohar said that Albireo Pharma's oral therapy Bylvay is the
closest competitor to Livmarli, but currently being tested in a
late-stage study for ALGS.
[to top of second column] |
 In July, Bylvay received U.S.
Food and Drug Administration's approval for
treatment of another liver disease called
Progressive familial intrahepatic cholestasis (PFIC)
and is priced at $385,000 per year.
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.
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.
(Reporting by Mrinalika Roy and Bhanvi Satija in Bengaluru; Editing
by Shinjini Ganguli)
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