Mirum Pharmaceuticals Stumbles on Health Canada Approval

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) shares dipped Thursday. The company, treating rare diseases company, announced that Health Canada has authorized LIVMARLI® (maralixibat) oral solution and tablet formulation for the treatment of cholestatic pruritus in patients aged 12 months or older with progressive familial intrahepatic cholestasis (PFIC).
PFIC is a rare, inherited pediatric liver disease caused by impaired bile flow, leading to the accumulation of bile acids in the liver and bloodstream.
Cholestasis in PFIC is associated with severe pruritus that can significantly impede quality of life for patients and caregivers. LIVMARLI is available in Canada for the treatment of PFIC as a 19 mg/mL oral solution and as a tablet formulation for patients weighing 22 kg or more who can swallow tablets.
“Health Canada’s authorization of LIVMARLI for the treatment of cholestatic pruritus in patients with PFIC aged 12 months and older has the potential to be transformational for patients living with the severe burden of symptoms associated with this disease,” said General Manager Jamie Twiselton.
“The authorization of both the 19 mg/mL oral solution and the tablet formulation of LIVMARLI offers meaningful flexibility and convenience to support treatment as patients with PFIC age.”
MIRM shares slipped $2.33, or 2.2%, to $101.63.

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