Business Wire

Mirum Pharmaceuticals’ LIVMARLI Approved in the European Union for Patients with PFIC

Share

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the European Commission has granted marketing authorization for LIVMARLI® (maralixibat) oral solution for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients three months of age and older. The approval follows a positive opinion by the CHMP which 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. Further, evaluation by COMP recommended maintenance of Orphan Drug Designation for LIVMARLI in PFIC.

The approval is 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. Data from MARCH showed statistically significant reduction in pruritus (p<0.0001) and serum bile acids (p<0.0001) between LIVMARLI versus placebo in the All-PFIC cohort (n=64). Significant improvements were also observed in total bilirubin and growth versus placebo. The most common treatment emergent adverse event was diarrhea, which was predominantly mild, with no severe cases, and transient.

“We are thrilled that the European Commission has granted marketing authorization for LIVMARLI in PFIC, acknowledging the strength of data collected and recognizing the important treatment opportunity for patients living with this rare liver disease,” said Chris Peetz, chief executive officer at Mirum. “Our hope is that LIVMARLI helps to improve key liver parameters and brings healthier days ahead to young patients diagnosed with PFIC in Europe. We are grateful to the researchers, patients, and families who made this approval possible.”

“LIVMARLI’s approval provides a treatment backed by years of clinical research and meaningful data that demonstrate a reduction in cholestatic itch and prognostic markers of improved liver health, including a reduction of serum bile acids, all important signals for positive long-term outcomes,” said Professor Richard Thompson, King’s College London. “It is encouraging to know that physicians in Europe will have a new option that has the potential to improve the liver health and quality of life for patients and their families.”

“The patient community in Europe will greatly benefit from LIVMARLI’s approval, supported by years of impressive data showing improvements in the most burdensome aspects of disease,” said Emily Ventura, executive director, PFIC Network. “PFIC can be life-altering and can have a devastating impact on patients. We are encouraged to see that young patients will have a new medication and hope for a life less burdened by cholestasis.”

LIVMARLI is also approved by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with PFIC five years of age and older. Mirum has submitted a supplemental new drug application to introduce a higher concentration formulation of LIVMARLI, used during the MARCH study, to enable label expansion for younger patients with PFIC, and expects to receive FDA feedback this year.

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, chenodiol, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results reported in 2023. Mirum has filed a new drug application with the FDA for the approval chenodiol to treat CTX in the U.S.

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 commercial success of LIVMARLI in Europe for PFIC, pricing reimbursement for LIVMARLI in any specific country, and the real world impact of LIVMARLI in PFIC. 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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240708474225/en/

Contacts

Media Contact:
Erin Murphy
media@mirumpharma.com

Investor Contact:
Andrew McKibben
investors@mirumpharma.com

(c) 2024 Business Wire, Inc., All rights reserved.

Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.

Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

New Esri Press Book by Jack Dangermond Explores Creating a Better Future through Modern GIS17.7.2024 20:46:00 CEST | Press release

Esri, the global leader in location intelligence, today announced the publication next month of The Power of Where: A Geographic Approach to the World's Greatest Challenges. Written by Esri president Jack Dangermond with a dedicated Esri team and numerous contributors, this important new book explores the geographic approach—a way of solving problems using spatial analysis to perceive and understand patterns ranging from wildlife migration and rising seas to urban planning and food production. According to Dangermond, sustainability begins with geography. "I believed this when we started Esri, and I'm even more convinced today," says Dangermond. "GIS [geographic information system technology] provides a platform for addressing problems, exploring alternatives, designing solutions, and sharing them in a visual way that inspires action." The Power of Where shows through maps and stories how the growing GIS community is facing humanity's big challenges, including climate change, hunger, w

Egle Therapeutics to share preclinical data for its first autoimmune disease program, EGL-003, with an oral presentation at the 2024 Promise of Interleukin-2 Therapy Meeting17.7.2024 18:00:00 CEST | Press release

Egle Therapeutics, a biotechnology company focused on advancing the next generation of regulatory T cell-focused therapies for oncology and auto-immunity, will deliver an oral presentation at The Promise of Interleukin-2 Therapy meeting taking place in Paris from September 4th to 7th, 2024. The presentation, entitled “EGL-003, a novel IL-2 mutein to selectively expand and activate regulatory T cells and improve therapeutic efficacy in autoimmune disease”, will show evidence that EGL-003, a non-targeted Fc-fused IL-2 agonist mutein, selectively binds to regulatory T cells (Treg) and induces Treg IL-2 signaling. The resulting increase of Treg frequencies was observed in vitro and in different mouse models. Accordingly, EGL-003 significantly improved clinical, macroscopic, and histological parameters in a DSS-induced colitis mouse model with expansion of tissue-resident Tregs. EGL-003 is the first drug candidate being developed for treatment of autoimmune diseases in Egle’s pipeline and h

Mouser Electronics Unlocks Game-Changing Human-Machine Interfaces in Latest Content Series17.7.2024 17:11:00 CEST | Press release

Mouser Electronics, Inc., the authorized global distributor with the newest electronic components and industrial automation products, today released the latest installment of its Empowering Innovation Together (EIT) technology series, exploring the unique attributes of human-machine interfaces (HMI) for everyday devices and industrial applications. With the evolution of HMI, engineers are blending intuitive, user-centric designs with cutting-edge technologies to introduce a host of novel capabilities. In this series, Mouser takes a deeper look at the future technological and societal impact of HMIs across our devices, cars, houses, and each other. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20240717572923/en/ Enter the world of HMI and Human Factors, where engineers and researchers delve into the psychological, physiological, and ergonomic aspects of designing interfaces for human-machine interaction to optimize user experi

LambdaTest Integrates Allure Reports with HyperExecute for Enhanced Test Reporting17.7.2024 17:00:00 CEST | Press release

LambdaTest, a leading cloud-based unified testing platform, is thrilled to announce the integration of Allure Reports with HyperExecute. This new integration aims to transform the test reporting landscape, offering a more streamlined, detailed, and visually appealing way to manage and review test results. Allure Framework is a versatile, lightweight, multi-language test reporting tool that provides a concise representation of tested functionalities through visually appealing web reports. It facilitates the easy extraction of valuable information for all stakeholders involved in the development process, enhancing collaboration and efficiency. For teams already utilizing Allure reports, the integration with HyperExecute allows for the seamless generation of consolidated Allure reports for each job. These reports are accessible through the report section of the HyperExecute dashboard, centralizing test insights and simplifying management. Additionally, HyperExecute users can enhance their

LTIMindtree Delivers 2.6% QoQ USD Revenue Growth in CC17.7.2024 16:40:00 CEST | Press release

LTIMindtree [NSE: LTIM, BSE: 540005], a global technology consulting and digital solutions company, announced its consolidated results today for the first quarter ended June 30, 2024, as approved by its Board of Directors. “While the environment remains unchanged, fiscal 25 started on a positive note for us with Q1FY25 revenue of USD 1.1 billion, registering a 2.5% QoQ and 3.5% YoY revenue growth in USD terms. Our Q1FY25 EBIT expanded to 15% and order inflow remained stable at USD 1.4 billion. Our top 3 industry verticals and our largest geography have performed well sequentially. This is attributed to a measured uptick in IT spending for critical initiatives with clients balancing innovation and fiscal prudence.” -Debashis Chatterjee, Chief Executive Officer and Managing Director Key financial highlights: Quarter ended June 30, 2024 In USD: - Revenue at $1,096.2 million (+2.5% Q-o-Q / +3.5% Y-o-Y) - Net profit at $136.1 million (+2.8% Q-o-Q / -2.9% Y-o-Y) In INR: - Revenue at Rs 91,42

In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.

Visit our pressroom
HiddenA line styled icon from Orion Icon Library.Eye