Mirum Pharmaceuticals Announces Positive CHMP Opinion for LIVMARLI® (maralixibat) Oral Solution for the Treatment of PFIC in Patients Three Months of Age and Older

• Positive opinion from CHMP based on Phase 3 MARCH study with highly statistically significant (p<0.0001) reduction in pruritus severity between LIVMARLI vs. placebo across all PFIC types studied.
• CHMP assessment concluded that LIVMARLI in PFIC brings significant clinical benefit over the existing approved therapy.
• Additionally, LIVMARLI received favorable COMP opinion recommending maintenance of Orphan Drug Designation in PFIC.
• Following CHMP and COMP opinions, a decision by the European Commission is expected in the third quarter of 2024.

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion of LIVMARLI® (maralixibat) oral solution for the treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) in patients three months of age and older. The CHMP also concluded that LIVMARLI in PFIC brings significant clinical benefit based on improved efficacy and a major contribution to patient care compared to the existing approved treatment for PFIC. The CHMP positive opinion is the scientific recommendation to the European Commission for marketing authorization in Europe.

In addition, the Committee for Orphan Medicinal Products (COMP), has also recommended maintenance of Orphan Drug Designation for LIVMARLI in PFIC. The European Commission’s endorsement of the CHMP and COMP opinions is expected in the third quarter of 2024.

“The positive and favorable opinions by both CHMP and COMP recognizes the potential of LIVMARLI in treating patients with PFIC in Europe, underscoring the compelling long-term data collected in the largest PFIC study across a broad range of genetic types,” said Chris Peetz, chief executive officer at Mirum. “Further, we are thrilled that, if approved by the European Commission, the PFIC community will have a treatment option that has been shown to significantly improve key liver health parameters starting from three months of age.”

The CHMP and COMP opinions are based on data from the Phase 3 MARCH study, the largest randomized trial conducted in PFIC, with 93 patients across a range of genetic PFIC types, including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6, and unidentified mutational status.

LIVMARLI is currently approved in Europe for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) two months of age and older. In the United States, LIVMARLI is approved for the treatment of cholestatic pruritus in patients with ALGS three months of age and older and for cholestatic pruritus in patients with PFIC five years of age and older. LIVMARLI is also approved in other regions globally. Mirum has submitted a supplemental new drug application (sNDA) to introduce a higher concentration formulation of LIVMARLI, used during the MARCH study, to enable label expansion for younger patients with PFIC in the United States later this year.

About Progressive Familial Intrahepatic Cholestasis

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 the United States and Europe. More than six 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

LIVMARLI® (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor approved 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 and for progressive familial intrahepatic cholestasis (PFIC) five years 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.

LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS and PFIC. 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 Alagille syndrome 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.

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 Alagille syndrome 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.

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 diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution, CHOLBAM® (cholic acid) capsules, and CHENODAL® (chenodiol) tablets.

LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients five years of age and older. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms or liver disease. CHENODAL has received medical necessity recognition by the FDA to treat patients with cerebrotendinous xanthomatosis (CTX).

Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2b VISTAS study for primary sclerosing cholangitis and Phase 2b VANTAGE study for primary biliary cholangitis. Lastly, CHENODAL, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results reported in 2023.

To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (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 LIVMARLI and perceived superiority against other products, the marketing authorisation application pending with the European Commission and planned regulatory submissions in other countries. 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. A further description of risks and uncertainties can be found in Mirum’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors,” as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov.

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