Mirum’s LIVMARLI Now Approved for PFIC in Patients 12 Months and Older

Rhea-AI Summary

Mirum Pharmaceuticals (Nasdaq: MIRM) announced FDA approval for an expanded label of LIVMARLI® (maralixibat) oral solution. The new label includes treatment for cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) aged 12 months and older. This expansion, based on the MARCH Phase 3 study, also includes a higher concentration formulation of LIVMARLI.

Chris Peetz, CEO of Mirum, expressed enthusiasm about the launch of LIVMARLI for PFIC patients, emphasizing the importance of early treatment initiation to reduce suffering from pruritus associated with this rare liver disease. The expanded label allows for treatment in younger patients, aligning with the typical early diagnosis of PFIC.

Positive

• FDA approval for expanded label of LIVMARLI to treat PFIC patients 12 months and older
• Inclusion of higher concentration formulation of LIVMARLI
• Potential for increased market reach due to expanded age range for treatment

Negative

• None.

Insights

Medical Research Analyst

The FDA's approval of LIVMARLI® (maralixibat) for treating cholestatic pruritus in PFIC patients as young as 12 months old is a significant development in pediatric hepatology. This expansion addresses a critical need in managing a rare and debilitating liver disease that often manifests in early childhood.

Key points to consider:

• PFIC typically presents in infancy or early childhood, making early intervention crucial. The expanded approval allows for earlier treatment, potentially improving long-term outcomes.
• Cholestatic pruritus, a severe itching caused by bile acid buildup, significantly impacts quality of life. LIVMARLI's mechanism of action in reducing bile acids could provide substantial relief for young patients.
• The approval includes a higher concentration formulation, which may improve adherence in pediatric patients by reducing the volume of medication needed.

While this approval is promising, it's important to monitor long-term safety and efficacy data in this younger population. The impact on disease progression and potential delay of liver transplantation will be key areas to watch in real-world use.

Financial Analyst

The label expansion for LIVMARLI® represents a significant market opportunity for Mirum Pharmaceuticals. Here's why this approval is financially impactful:

• Expanded patient pool: Lowering the age limit to 12 months substantially increases the addressable market, potentially driving revenue growth.
• First-mover advantage: As the first approved treatment for PFIC in this age group, LIVMARLI may establish strong market presence before potential competitors enter.
• Lifecycle management: The higher concentration formulation could extend patent protection and market exclusivity.
• Potential for increased adoption: Earlier treatment initiation may lead to better outcomes, potentially increasing physician confidence and prescription rates.

However, investors should consider:

• Market size: While expanded, PFIC remains a rare disease, limiting the overall revenue potential.
• Reimbursement challenges: Securing favorable pricing and coverage for an expanded indication in very young patients may require additional negotiations with payers.
• Long-term data: Continued positive real-world evidence will be important for maintaining market position and potentially expanding to other indications.

Overall, this approval strengthens Mirum's position in the rare liver disease market, but the company's ability to execute on commercialization will be key to realizing the full potential of this expanded indication.

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has approved a label expansion for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC). The expanded label includes use in PFIC patients 12 months and older as well as the higher concentration formulation of LIVMARLI evaluated in the MARCH Phase 3 study.

“The launch of LIVMARLI in PFIC is going well and we are thrilled that it will now be available for patients 12 months and older,” said Chris Peetz, chief executive officer at Mirum. “PFIC is generally diagnosed when children are young, and initiating treatment quickly after diagnosis will help to ensure they have fewer days suffering from pruritus associated with this rare 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 two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. 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 12 months of age and older; in Europe, it is approved for patients with PFIC three months 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 2 VISTAS study for primary sclerosing cholangitis (PSC) 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 submitted a new drug application with the FDA for the approval of 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 potential benefits of LIVMARLI in PFIC patients older than 12 years of age, the commercial success of LIVMARLI in PFIC generally, and the impact of the higher concentration formula of LIVMARLI. 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 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/20240725047219/en/

Media Contact:

Erin Murphy

media@mirumpharma.com

Investor Contact:

Andrew McKibben

investors@mirumphama.com

Source: Mirum Pharmaceuticals, Inc.

FAQ

What is the new FDA-approved age range for LIVMARLI in treating PFIC patients?

The FDA has approved LIVMARLI for treating cholestatic pruritus in PFIC patients 12 months and older.

What condition does LIVMARLI treat in PFIC patients?

LIVMARLI treats cholestatic pruritus, a symptom associated with progressive familial intrahepatic cholestasis (PFIC).

What study supported the expanded label for LIVMARLI (MIRM)?

The MARCH Phase 3 study supported the expanded label for LIVMARLI, including the higher concentration formulation.

Why is early treatment with LIVMARLI important for PFIC patients?

Early treatment with LIVMARLI is important because PFIC is generally diagnosed in young children, and quick initiation can reduce suffering from pruritus associated with this rare liver disease.