Mirum Pharmaceuticals Announces Health Canada Authorization of LIVMARLI® Tablet Formulation for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome

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.

alagille syndrome medical

A rare genetic disorder that causes problems in the liver, heart, eyes and other organs by disrupting the formation of small bile ducts and other tissues; think of it as a congenital wiring fault that affects how the body drains bile and develops certain structures. It matters to investors because the condition drives demand for specialized medicines, diagnostics and potentially costly treatments, so clinical trial results, regulatory decisions or new therapies can significantly affect companies working in rare-disease drugs and related healthcare markets.

health canada regulatory

Health Canada is the government department responsible for overseeing the safety and regulation of health-related products and services in Canada, including medications, medical devices, and vaccines. Its approval and guidelines help ensure that these products are safe and effective for public use. For investors, Health Canada's decisions can influence the success of healthcare companies and affect the availability of new medical treatments in the Canadian market.

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.

- Tablet authorization builds upon established use of LIVMARLI oral solution in Canada

TORONTO--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that Health Canada has authorized the tablet formulation of LIVMARLI® (maralixibat) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS).

LIVMARLI had previously been authorized in Canada as an oral solution for the treatment of cholestatic pruritus in patients with ALGS aged 12 months or older. The tablet formulation complements the existing 9.5 mg/ml oral solution, offering an additional dosing option for patients with ALGS weighing 22 kg or more who are able to swallow tablets.

“LIVMARLI’s tablet formulation provides an important new option for patients with ALGS,” said Jamie Twiselton, General Manager, Mirum Pharmaceuticals Canada. “With a liquid formulation for younger patients and a convenient one tablet per dose option available for older patients, LIVMARLI now provides greater dosing flexibility, supporting continuity of care as patients with ALGS age.”

ALGS is a rare genetic disorder that affects multiple organ systems, including the liver, where bile duct abnormalities can lead to cholestasis and pruritus. Chronic itching associated with ALGS can significantly disrupt sleep, growth and daily life for patients and their families.

“As children with Alagille syndrome grow, treatment needs can change,” said Dr. Or Steg Saban, Division of Gastroenterology, Hepatology and Nutrition, The Hospital for Sick Children, University of Toronto. “The availability of a tablet formulation of LIVMARLI in Canada provides an additional option for appropriate patients, which may support treatment adherence and continuity of care while continuing to address cholestatic pruritus.”

Earlier today, Mirum announced that Health Canada has authorized LIVMARLI® for the treatment of cholestatic pruritus in patients aged 12 months and older with progressive familial intrahepatic cholestasis (PFIC), including both the 19 mg/mL oral solution and tablet formulations to support flexible dosing. Link

About Alagille syndrome

Alagille syndrome (ALGS) is a rare genetic disorder in which bile ducts are abnormally narrow, malformed and reduced in number, which leads to bile accumulation in the liver and ultimately progressive liver disease. The estimated incidence of ALGS is one in every 30,000 people. In patients with ALGS, multiple organ systems may be affected by the mutation, including the liver, heart, kidneys and central nervous system. The accumulation of bile acids prevents the liver from working properly to eliminate waste from the bloodstream and, according to recent reports, 60% to 75% of patients with ALGS have a liver transplant before reaching adulthood. Signs and symptoms arising from liver damage in ALGS may include jaundice (yellowing of the skin), xanthomas (disfiguring cholesterol deposits under the skin) and pruritus (itch). The pruritus experienced by patients with ALGS is among the most severe in any chronic liver disease and is present in most affected children by the third year of life.

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 12 months of age 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.