FOSTER CITY - Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM), a biopharmaceutical company developing treatments for rare diseases, today announced that Livmarli is indicated for the treatment of cholestatic pruritus in patients affected by Alagille syndrome (ALGS) aged 2 months and older.

LIVMARLI is the first and only drug admitted for reimbursement by the Italian Medicines Agency (AIFA) for this indication. LIVMARLI is the first and only orphan drug to have obtained orphan drug status and marketing authorization in the European market for cholestatic pruritus in ALGS.

'We are pleased that the Italian Medicines Agency has recognized the significant potential benefit that LIVMARLI offers to patients with cholestatic pruritus in ALGS and has approved the drug for reimbursement for patients with this rare and severe liver disease,' said Chris Peetz , president and CEO of Mirum. 'Years of collected data show that many patients improve after treatment with LIVMARLI and we hope that this drug will also offer Italian patients the possibility of a life less compromised by the disease'.

'Patients with Alagille syndrome not only have significantly impaired quality of life due to pruritus, but are also at risk of developing severe long-term hepatic impairment due to the accumulation of bile acids in the liver,' he said Dr. Lorenzo D'Antiga, head of pediatrics at the Papa Giovanni XXIII Hospital in Bergamo.

LIVMARLI, an ileal bile acid transporter (IBAT) inhibitor, is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the United States (three months and beyond), Europe (two months and beyond) and in Canada.

Alagille syndrome (ALGS) is a rare genetic disorder in which the bile ducts are narrowed, malformed and reduced in number, resulting in accumulation of bile in the liver and progressive liver disease. The estimated incidence of ALGS is one in 30,000 births. In patients with ALGS, the mutation can affect multiple organs and systems, including the liver, heart, kidneys and central nervous system. The accumulation of bile acids prevents the liver from functioning properly to eliminate waste substances from the bloodstream, and as reported in recent statistics, 60-75% of patients with ALGS undergo liver transplantation before reaching age adult. Signs and symptoms resulting from liver damage in ALGS may include jaundice (yellowing of the skin), xanthomas (disfiguring cholesterol deposits under the skin), and itching. The pruritus experienced by patients with ALGS is among the most severe in any chronic liver disease and appears in the majority of affected children by the third year of life.

Information about LIVMARLI (maralixibat) oral solution

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

LIVMARLI is also the only IBAT inhibitor approved by the European Commission for the treatment of cholestatic pruritus in patients with ALGS aged two months and older and by Health Canada for the treatment of cholestatic pruritus in ALGS.

Mirum has also requested approval of LIVMARLI for cholestatic pruritus in patients with Progressive Familial Intrahepatic Cholestasis (PFIC) aged three months and older in the United States and for PFIC in patients aged three months and older in Europe. or more than two months.

LIVMARLI is currently being evaluated in late-phase clinical trials for other rare cholestatic liver diseases, including biliary atresia. LIVMARLI has received FDA Breakthrough Therapy Designation for ALGS and PFIC Type 2 and Orphan Drug Designation for ALGS, PFIC and Biliary Atresia.

About Mirum Pharmaceuticals, Inc.

Mirum Pharmaceuticals, Inc. is a biopharmaceutical company committed to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved drugs: LIVMARLI (maralixibat) in oral solution, Cholbam (cholic acid) in capsules and Chenodal (chenodiol) in tablets.

LIVMARLI, an IBAT inhibitor, is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the United States (three months of age and older), Europe (two months of age and older), and Canada. Mirum has also requested approval of LIVMARLI for cholestatic pruritus in patients with Progressive Familial Intrahepatic Cholestasis (PFIC) aged three months and older in the United States and in Europe for PFIC for patients aged three months or older. more than two months. Cholbam is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme defects and for the adjunctive treatment of peroxisomal disorders in patients exhibiting signs or symptoms or liver disease. Chenodal has received medical necessity approval from the FDA for the treatment of patients with cerebrotendinous xanthomatosis.

Mirum's late-stage pipeline includes three investigational treatments for severe liver disease. LIVMARLI's development program includes the Phase 2b EMBARK study for biliary atresia. Mirum's second investigational IBAT inhibitor is volixibat, which is being evaluated in two potentially pivotal studies, including the Phase 2b VISTAS study for adults with primary sclerosing cholangitis and the Phase 2b VANTAGE clinical trial for adults with cholangitis primary biliary. Finally, Chenodal was evaluated in a phase 3 clinical trial, RESTORE, for the treatment of patients with CTX.

Forward-Looking Statements

Statements 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, commercialization plans and expectations for the commercialization of LIVMARLI in the United States, Italy and outside the United States, 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 as well as other Mirum product candidates. Because such statements are subject to risks and uncertainties, actual results may differ significantly from those expressed or implied by such forward-looking statements. Words such as 'will/will,' 'may,' 'should,' 'potential' and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based on Mirum's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results may differ significantly from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including, but not limited to, risks and uncertainties associated with Mirum's business generally, the impact of the COVID pandemic -19 and 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 assumptions and estimates of management as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that arise after the date they are made, except as required by law.

Contact:

Erin Murphy

Tel: 510-508-6521

Email: media@mirumpharma.com

Andrew McKibben

Email: ir@mirumpharma.com

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