Mirum's oral drug becomes first U.S. approved Alagille syndrome therapy

Reuters

Published Sep 29, 2021 04:45PM ET

Updated Sep 29, 2021 06:21PM ET

By Mrinalika Roy and Bhanvi Satija

(Reuters) -Mirum Pharmaceuticals Inc said on Wednesday its drug became the first U.S. approved therapy for treating itching in patients with a rare liver disorder called Alagille syndrome (ALGS).

"(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 (NASDAQ:ALBO)'s oral therapy Bylvay is the closest competitor to Livmarli, but currently being tested in a late-stage study for ALGS.

In July, Bylvay received U.S. Food and Drug Administration's approval for treatment of another liver disease called Progressive (NYSE:PGR) 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.