Mirum Pharmaceuticals Announces Health Canada Authorization of LIVMARLI® for the Treatment of Cholestatic Pruritus in Patients with Progressive Familial Intrahepatic Cholestasis (PFIC)

Key Terms

cholestatic pruritus medical

Severe itching caused by impaired bile flow from the liver into the gut, where substances that normally leave the body build up and irritate nerve endings in the skin. Investors should care because this symptom can drive demand for treatments, shape clinical trial measures and regulatory decisions, and affect a drug’s safety profile and market potential—much like a visible fault that influences customer trust and product value.

progressive familial intrahepatic cholestasis medical

A rare, inherited liver disorder that prevents the normal flow of bile from liver cells, leading over time to persistent jaundice, liver damage and often liver failure in children and young adults. Investors track it because the condition represents a clear unmet medical need with limited treatment options, making successful therapies or regulatory approvals potentially valuable—like fixing a clogged drain that restores an essential function and can command premium pricing and regulatory incentives.

phase 3 medical

Phase 3 is the late-stage clinical testing step for a new drug or medical treatment, where the product is given to large groups of patients to confirm effectiveness, monitor side effects, and compare it to standard care. Successful Phase 3 results are often the final scientific hurdle before regulators decide on approval and market launch—like passing a final exam before graduation—and can sharply change a company's valuation and future revenue prospects.

randomized trial medical

A randomized trial is a study where participants are assigned by chance—like flipping a coin—to different treatments or to a comparison group so the results reflect the effect of the treatment rather than other factors. Investors care because these trials provide the most reliable evidence about whether a drug, device, or strategy works, and strong, clear results can change a company’s approval prospects, market size and risk profile quickly.

p<0.0001 technical

p<0.0001 indicates a statistical result whose probability of appearing by random chance is less than 0.01%, so the observed effect is extremely unlikely to be accidental. For investors this raises confidence that a study, test or claim cited in an announcement is reliable, but it does not say how large or economically important the effect is — like finding a tiny but very consistent difference versus a big change.

- Authorization includes oral solution and tablet formulation to support flexible dosing

TORONTO--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today 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 Jamie Twiselton, General Manager, Mirum Pharmaceuticals Canada. “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.”

Health Canada’s authorization of LIVMARLI for the treatment of cholestatic pruritus in PFIC is supported by data from the Phase 3 MARCH study, the largest randomized trial conducted in PFIC, which enrolled 93 patients, including patients from Canada. The MARCH study included patients across a range of genetic PFIC types, including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6 and unidentified mutational status. In the MARCH study, patients treated with LIVMARLI demonstrated a highly statistically significant (p<0.0001) reduction in pruritus severity versus placebo.

“PFIC is a complex condition to treat, and pruritus is often among the most burdensome symptoms for children and families,” said Susan M. Gilmour, MD, MSc, FRCPC, Professor Pediatric Gastroenterology/Nutrition, Department of Pediatrics, Faculty of Medicine and Dentistry, University of Alberta, Stollery Children’s Hospital. “An authorized option in Canada that addresses cholestatic pruritus and is indicated across multiple PFIC types, while offering flexibility in formulation, represents an important advancement for clinical care.”

“Patients and families affected by PFIC know how all-consuming cholestatic pruritus can be,” said Emily Ventura, Co-Founder and Executive Director of the PFIC Network. “For many patients, the itch is so severe it impacts sleep, nutrition and child development. Health Canada’s authorization of LIVMARLI offers hope for meaningful relief from pruritus, and the availability of both liquid and tablet formulations offers treatment flexibility for patients.”

LIVMARLI^® is also authorized in Canada for the treatment of cholestatic pruritus in patients 12 months of age or older with Alagille syndrome (ALGS).

About PFIC

Progressive familial intrahepatic cholestasis (PFIC) is a rare genetic disorder that causes progressive liver disease, typically leading to liver failure. In people with PFIC, liver cells are less able to secrete bile. The resulting buildup of bile causes liver disease in affected individuals. Signs and symptoms of PFIC typically begin in infancy. Patients experience severe itching, jaundice, failure to grow at the expected rate (failure to thrive) and an increasing inability of the liver to function (liver failure). The disease is estimated to affect one in every 50,000 to 100,000 births in Canada. Many types of PFIC have been genetically identified, all of which are similarly characterized by impaired bile flow and progressive liver disease.

About LIVMARLI^® (maralixibat) oral solution and LIVMARLI^® (maralixibat) tablets

LIVMARLI^® (maralixibat) is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. LIVMARLI is authorized in Canada for the treatment of cholestatic pruritus in patients aged 12 months and older with ALGS.

LIVMARLI is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. In Canada, LIVMARLI is authorized for the treatment of cholestatic pruritus in patients aged 12 months or older with progressive familial intrahepatic cholestasis (PFIC).

LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website.

IMPORTANT SAFETY INFORMATION

Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein.

LIVMARLI can cause side effects, including:

Liver injury. Changes in certain liver tests are common in patients with ALGS and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal. LIVMARLI is contraindicated in patients with prior or active hepatic decompensation events.

Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you.

A condition called Fat Soluble Vitamin (FSV). Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat is common in patients with ALGS and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects.

Canadian Product Monograph
US Prescribing Information
EU SmPC

About Mirum Pharmaceuticals

Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum combines deep rare disease expertise with strong connections to patient communities. The company’s commercial portfolio includes LIVMARLI^® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), CHOLBAM^® (cholic acid) for bile-acid synthesis disorders, and CTEXLI^® (chenodiol) for cerebrotendinous xanthomatosis (CTX). Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV), and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS). Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. Learn more at www.mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and X.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the potential benefits of a tablet formulation of LIVMARLI versus a liquid formulation, ability of patients to remain on therapy as they age, the availability of a tablet formulation for all patients taking LIVMARLI and the real life usage patterns of patients on LIVMARLI. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “will,” “could,” “can,” “would,” “potential,” “hope,” “opportunity,” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum’s business in general, the impact of macroeconomic and geopolitical developments, and the other risks described in Mirum’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. Mirum’s Quarterly Report on Form 10-K for the quarter ended December 31, 2024 and subsequent filings with the U.S. Securities and Exchange Commission and available at www.sec.gov.

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Investor Contact:

Andrew McKibben

ir@mirumpharma.com

Media Contact:

Meredith Kiernan

Media@mirumpharma.com

Canadian Media Contact (English):

Olivia Simmonds

energi PR

Olivia.simmonds@energipr.com

724-524-8108

Source: Mirum Pharmaceuticals, Inc.