• First and only medication authorized in Canada for the treatment of cholestatic pruritus in patients with Alagille syndrome.
• Approval supported by more than six years of LIVMARLI data demonstrating improvements in pruritus and serum bile acids.
• Data from LIVMARLI clinical trials published in The Lancet and Hepatology

FOSTER CITY, Calif. / Jul 25, 2023 / Business Wire / Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that LIVMARLI® (maralixibat oral solution) has been authorized by Health Canada for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS).

ALGS is a rare genetic disorder caused by abnormalities in bile ducts that can lead to progressive liver disease. Malformed or reduced bile ducts cause cholestasis, the accumulation of bile acids in the liver, which leads to inflammation and liver injury, and prevents the liver from working properly. Cholestasis in ALGS is associated with pruritus which is among the most common indications for liver transplant in ALGS.

“Today’s approval in Canada furthers our commitment to ensuring patients globally have a meaningful and effective treatment option for the unrelenting burden of cholestatic pruritus impacting patients and their families living with ALGS,” said Chris Peetz, president and chief executive officer at Mirum. “We are grateful to the patients, families, clinicians and ALGS community who made this approval possible.”

“There have been meaningful advances in the treatment of Alagille syndrome with the approval of LIVMARLI and I am pleased that it is now available in Canada where I have seen, firsthand, the critical need for an approved therapy to address the itch caused by cholestasis. The years of data collected have shown that LIVMARLI can provide tremendous benefit, potentially giving many patients the opportunity to live transplant-free,” said Dr. Binita Kamath, Hospital for Sick Children (SickKids), Toronto, Canada. “The statistical robustness of results seen in the LIVMARLI studies give confidence that this treatment has the potential to have meaningful outcomes for patients with debilitating pruritus.”

“We are thrilled that patients in Canada will now have access to LIVMARLI as we have seen the powerful impact it can have on people living with life-altering pruritus,” said Roberta Smith, president of the Alagille Syndrome Alliance. “I know from my own experience how painful it is to watch your child suffer and how disruptive this disease can be for the whole family. It is wonderful to know that patients in Canada can have a chance at a life less burdened by cholestatic pruritus.”

The Health Canada authorization is based on data from the pivotal ICONIC study including six years of data across the LIVMARLI clinical program resulting in a robust body of evidence in patients with cholestatic pruritus in ALGS. Data from ICONIC demonstrated statistically significant and clinically meaningful reductions in pruritus compared to placebo, as well as significant reductions in serum bile acids, both of which were durably maintained over several years of treatment.

Results from these studies showing robust and meaningful improvements for patients with cholestatic pruritus associated with ALGS were featured in several publications, including The Lancet and Hepatology.

In addition to today’s authorization, LIVMARLI is approved in the United States for the treatment of cholestatic pruritus in patients with ALGS three months and older and in Europe in the same indication, two months of age and older. Mirum has submitted a supplemental new drug application to the FDA for approval of LIVMARLI in cholestatic pruritus associated with progressive familial intrahepatic cholestasis (PFIC) in patients three months and older, and to the EMA for the treatment of PFIC in patients two months of age and older. Both submissions are currently under regulatory review. Regulatory submissions outside of the U.S., Canada and Europe are being pursued by partners and distributors in key markets globally.

About LIVMARLI® (maralixibat) oral solution

LIVMARLI® (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor and the only approved medication by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) three months of age and older.

LIVMARLI is also the only approved IBAT inhibitor approved by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months and older, and by Health Canada for the treatment of cholestatic pruritus in ALGS. For more information for U.S. residents, please visit LIVMARLI.com.

Mirum has also submitted LIVMARLI for approval in the U.S. in cholestatic pruritus in PFIC patients three months of age and older, and in Europe, in PFIC for patients two months of age and older.

LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including biliary atresia. LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and biliary atresia. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website.

IMPORTANT SAFETY INFORMATION

LIVMARLI can cause side effects, including:

Changes in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome and can worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your healthcare provider should do blood tests 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) or loss of appetite.

Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea, stomach pain, and vomiting during treatment. 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. FSV deficiency is common in patients with Alagille syndrome but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment.

Other common side effects reported during treatment were gastrointestinal bleeding and bone fractures.

US Prescribing Information

EU SmPC

Canadian Product Monograph

About Mirum Pharmaceuticals, Inc.

Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare liver diseases. Mirum’s approved medication is LIVMARLI^® (maralixibat) oral solution which is approved in the U.S. for the treatment of cholestatic pruritus in patients with Alagille syndrome three months of age and older. LIVMARLI is also the only approved IBAT inhibitor approved by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months and older, and by Health Canada for the treatment of cholestatic pruritus in ALGS. For more information for U.S. residents, please visit LIVMARLI.com.

Mirum has also submitted LIVMARLI for approval in the U.S. in cholestatic pruritus in PFIC patients three months of age and older and in Europe in PFIC for patients two months of age and older.

Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver diseases affecting children and adults. LIVMARLI, an oral ileal bile acid transporter (IBAT) inhibitor, is currently being evaluated in clinical trials for pediatric liver diseases and includes the EMBARK Phase 2b clinical trial for patients with biliary atresia. In addition, Mirum has an expanded access program open across multiple countries for eligible patients with ALGS and PFIC.

Mirum’s second investigational treatment, volixibat, an oral IBAT inhibitor, is being evaluated in two potentially registrational studies including the VISTAS Phase 2b clinical trial for adults with primary sclerosing cholangitis and the VANTAGE Phase 2b clinical trial for adults with primary biliary cholangitis.

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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 commercialization plans and expectations for commercializing LIVMARLI in the United States, Canada, European Union and outside of the United States, estimates of the number of patients impacted by ALGS and who are appropriate for treatment with LIVMARLI, the potential benefits or competitive position of LIVMARLI, the timing of ongoing and planned clinical trials and the regulatory approval process of maralixibat in other indications and jurisdictions and of volixibat. 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,” “would,” “potential” 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 the COVID-19 pandemic, 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.