Mirum Pharmaceuticals’ LIVMARLI (maralixibat) Approved by the European Commission for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome Two Months and Older

Rhea-AI Summary

Mirum Pharmaceuticals announced that the European Commission has granted marketing authorization for LIVMARLI® (maralixibat) to treat cholestatic pruritus in patients with Alagille syndrome aged two months and older. This marks the first approval in both the U.S. and Europe for this condition. The approval is supported by over six years of clinical data showing improved survival rates and quality of life for patients. LIVMARLI is expected to launch in Germany in early 2023, with additional regulatory reviews ongoing in China, South Korea, and elsewhere.

Positive

• First approved medication for cholestatic pruritus in both U.S. and Europe.
• Approval supported by over six years of data from clinical studies.
• Statistically significant reduction in pruritus and serum bile acids demonstrated in the ICONIC study.

Negative

• None.

- First and only medication approved in both the U.S. and Europe to treat cholestatic pruritus in patients with Alagille syndrome.

- Approval supported by more than six years of LIVMARLI data demonstrating improvements in transplant-free survival, serum bile acids, growth, xanthomas, and quality of life.

- Expected to launch first in Germany in early 2023.

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the European Commission has granted marketing authorization for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) two months of age and older. The approval follows a positive opinion granted by the European Committee for Medicinal Products for Human Use (CHMP) in September 2022. LIVMARLI is the first and only approved treatment in both the European Union and the United States to treat this rare liver disease affecting one out of 30,000 people globally.

LIVMARLI is also approved by the U.S. Food and Drug Administration and the Israeli Ministry of Health for the treatment of cholestatic pruritus in patients with ALGS one year of age and older. Regulatory reviews are also underway in China, Taiwan, South Korea, and Great Britain.

The European Commission’s approval 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 with a mean difference of -1.4 points between groups, as well as significant reductions in serum bile acids, both of which were durably maintained over several years of treatment. The label also includes reductions in xanthoma severity and cholesterol, and the submission was supported by data showing an improvement in event-free survival compared to a natural history cohort.

“We are delighted that the European Commission has approved LIVMARLI and that patients and physicians in Europe will have a new standard of care for this challenging disease,” said Chris Peetz, president and chief executive officer at Mirum. “With more than six years of data and real-world evidence showing LIVMARLI’s impact, we believe patients and their families will have a chance at experiencing a new normal with relief from the burden caused by cholestasis. We look forward to advancing LIVMARLI’s availability throughout Europe and getting this important treatment option into the hands of patients.”

“Patients with Alagille syndrome have long been challenged by the lack of effective medical treatment options available to address cholestatic pruritus. It is encouraging to know that physicians will now have a medication to reduce cholestatic pruritus and to possibly decrease the need for liver transplantation when pruritus is the indication for liver transplantation,” said Professor Emmanuel Jacquemin, MD, PhD, Head of Pediatric Hepatology and Liver Transplantation Unit, Bicêtre Hospital, AP-HP, Paris-Saclay University, Le Kremlin-Bicêtre, France. “The data collected in the clinical studies demonstrate significant potential for LIVMARLI to reduce serum bile acids and pruritus, which could significantly improve the quality of life for patients and their families.”

"The approval of LIVMARLI in Europe is very exciting and provides hope for a better future for those living and dealing with Alagille Syndrome,” said Roberta Smith, president, Alagille Syndrome Alliance. “We are thrilled to know that families dealing with debilitating itch associated with ALGS will now have a treatment option. Patients who have been prescribed LIVMARLI in the U.S. have experienced tremendous benefit, and we are thrilled that families in Europe will get to experience the same."

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

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) one year of age and older and is the only FDA-approved medication to treat cholestatic pruritus associated with Alagille syndrome. For more information, please visit LIVMARLI.com.

LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including progressive familial intrahepatic cholestasis (PFIC) and 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 bone fractures and gastrointestinal bleeding.

US Prescribing Information

EU SmPC

About Mirum Pharmaceuticals

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 one year of age and older, and in Europe for the same indication in 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 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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20221213005467/en/

Media Contact:

Erin Murphy

media@mirumpharma.com

Investor Contacts:

Ian Clements, Ph.D.

ir@mirumpharma.com

Sam Martin

Argot Partners

ir@mirumpharma.com

Source: Mirum Pharmaceuticals, Inc.

FAQ

What is LIVMARLI and what condition does it treat?

LIVMARLI (maralixibat) is approved for treating cholestatic pruritus in patients with Alagille syndrome, a rare liver disease.

When is LIVMARLI expected to launch in Germany?

LIVMARLI is expected to launch in Germany in early 2023.

What data supported the approval of LIVMARLI in Europe?

The approval was supported by over six years of data from the ICONIC study, demonstrating significant improvements in multiple health metrics.

Is LIVMARLI approved in other regions besides Europe?

Yes, LIVMARLI is also approved by the U.S. FDA and the Israeli Ministry of Health, with ongoing reviews in other countries.

What significant outcomes did the ICONIC study present?

The ICONIC study showed significant reductions in pruritus and serum bile acids, with durable effects over several years.