Mirum Pharmaceuticals’ LIVMARLI (maralixibat oral solution) Receives Positive Reimbursement Recommendation by Canada’s CADTH for Patients with Cholestatic Pruritus in Alagille Syndrome

Rhea-AI Summary

Mirum Pharmaceuticals receives positive HTA recommendation for reimbursement of LIVMARLI in Canada, marking a significant milestone in treating cholestatic pruritus in patients with Alagille syndrome. The recommendation by CADTH highlights the efficacy of LIVMARLI in reducing pruritus and bile acids, based on data from the ICONIC study. This approval expands access to the first approved therapy for this rare liver disorder in Canada, providing hope for patients and families.

Positive

• Positive HTA recommendation for reimbursement of LIVMARLI in Canada for treating cholestatic pruritus in patients with Alagille syndrome.
• Approval based on data from the pivotal ICONIC study showing significant reductions in pruritus and serum bile acids compared to placebo.
• LIVMARLI is the first and only approved medication for this indication in Canada, offering hope to patients suffering from the debilitating effects of Alagille syndrome.
• Recommendation signifies a significant advancement in the treatment of cholestatic pruritus, addressing the critical need for effective therapies in this patient population.
• Approval follows Health Canada's authorization of LIVMARLI in 2023, expanding access to a meaningful treatment option for patients with Alagille syndrome.

Negative

• None.

Pharmaceutical Industry Analyst

The recommendation by the Canadian Agency for Drugs and Technologies in Health (CADTH) for public reimbursement of LIVMARLI signifies a strategic advancement for Mirum Pharmaceuticals in the Canadian market. This not only expands the drug's accessibility but also potentially increases market penetration. The endorsement based on the ICONIC study's six-year data provides a robust foundation for the drug's efficacy and long-term benefits, which can be a strong selling point. It is essential to note the rarity of Alagille Syndrome (ALGS) and the limited patient population, which could mean that while the drug's approval is a significant win for Mirum, the direct financial impact may be moderate due to the small market size.

However, the precedent set for other rare disease treatments and the company's positioning in the niche market could attract investor interest. The long-term financial implications hinge on the uptake of the treatment and potential expansion into other indications. The approval in Canada complements the drug's presence in the U.S. and Europe, indicating a successful international regulatory strategy and reinforcing the company's global footprint in rare liver disorders.

Health Economics Expert

The positive reimbursement recommendation from CADTH is a pivotal event that can alter the cost dynamics for both Mirum Pharmaceuticals and the Canadian healthcare system. Reimbursement is a critical factor in the adoption of new therapies, especially in markets with public healthcare systems like Canada. By securing reimbursement, Mirum ensures that patients have reduced financial barriers to access LIVMARLI, which could lead to an increase in demand and treatment adherence.

The economic implications extend beyond immediate revenue generation. It sets a precedent for the valuation of treatments for rare diseases and may influence future pricing and reimbursement negotiations in other jurisdictions. The impact on the stock market could be seen as a positive signal of the company's ability to navigate complex reimbursement landscapes, which is often a significant hurdle for pharmaceutical companies. Investors may view this development as indicative of the company's potential for long-term growth and profitability in the rare disease market segment.

Medical Research Analyst

The clinical significance of LIVMARLI's efficacy in treating cholestatic pruritus in patients with ALGS cannot be overstated. The debilitating nature of pruritus, often leading to liver transplants, underscores the urgent need for effective treatments. The ICONIC study's findings of significant pruritus reduction and decreased serum bile acids provide a strong clinical argument for the drug's use.

From a research perspective, the long-term data is particularly valuable, demonstrating sustained efficacy over several years, which is important for chronic conditions like ALGS. This could lead to increased clinician confidence in prescribing LIVMARLI and may improve patient outcomes. The approval also encourages further research into similar therapies for other rare liver diseases, potentially opening new avenues for treatment. For stakeholders, this translates into the potential for improved quality of life for patients and reduced long-term healthcare costs associated with ALGS complications.

- Positive HTA recommendation for reimbursement signifies an important milestone towards public funding to treat eligible patients with this rare liver disorder

- LIVMARLI is the first and only approved medication to treat cholestatic pruritus in patients with Alagille syndrome in Canada

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that the Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee (CDEC) has recommended public reimbursement for LIVMARLI® (maralixibat oral solution) for the treatment of cholestatic pruritus in patients with Alagille Syndrome (ALGS), with certain conditions. This announcement follows Health Canada’s authorization of LIVMARLI in this indication in 2023.

“The positive reimbursement recommendation for LIVMARLI by CADTH is an important step forward for patients suffering from the debilitating effects of cholestatic pruritus related to Alagille syndrome,” said Chris Peetz, chief executive officer at Mirum. “Accelerating access in all provinces to this meaningful treatment is critical as it is the first and only approved therapy available in this setting in Canada. These patients suffer greatly from the debilitating and disruptive effects of Alagille syndrome, which often starts in early childhood.”

“CADTH’s recommendation of reimbursement for LIVMARLI provides a meaningful advancement in the treatment of cholestatic pruritus for patients with Alagille syndrome. I am pleased that LIVMARLI will now become more accessible in Canada, where I have seen the critical need for a treatment to address the itch caused by cholestasis,” said Dr. Binita Kamath, Director, Hepatology Program and Senior Associate Scientist at The Hospital for Sick Children (SickKids), Toronto, Canada.

“This recommendation is incredibly meaningful as it signifies hope for patients and their families who have suffered from the disruptive and painful effects of pruritus for far too long,” said Roberta Smith, president, Alagille Syndrome Alliance and mother of a daughter with ALGS. “We are grateful to CADTH for ensuring support of this new and important medication.”

The CADTH recommendation 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, as well as significant reductions in serum bile acids, both of which were durably maintained over several years of treatment.

LIVMARLI is also approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. and Europe, and for Progressive Familial Intrahepatic Cholestasis (PFIC) in the U.S. and in Europe.

ALGS is a rare genetic disorder caused by abnormalities in bile ducts that can lead to progressive liver disease. Malformed or reduced bile ducts cause cholestasis, the accumulation of bile acids in the liver, which leads to inflammation and liver injury, and prevents the liver from working properly. Cholestasis in ALGS is associated with pruritus which is among the most common indications for liver transplant in ALGS.

About LIVMARLI® (maralixibat) oral solution

LIVMARLI® (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor and the only approved medication 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 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.

Mirum has also submitted LIVMARLI for approval in Europe in PFIC for patients two months of age and older.

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

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. Mirum has submitted for approval in Europe for the treatment of PFIC in patients two 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 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.

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 commercial success of LIVMARLI in Canada and other approved territories and the additional improvements in disease beyond pruritus. 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,” “intended,” “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 geopolitical and macroeconomic events , and the other risks described in Mirum’s filings with the Securities and Exchange Commission, including its most recent Annual Report on Form 10-K, as updated by any subsequent Quarter Reports on Form 10-Q. 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/20240402976390/en/

Media:

Erin Murphy

510-508-6521

media@mirumpharma.com

Investors:

Andrew McKibben

ir@mirumpharma.com

Source: Mirum Pharmaceuticals, Inc.

What is the significance of the positive HTA recommendation for Mirum Pharmaceuticals?

The positive HTA recommendation for reimbursement of LIVMARLI in Canada signifies a significant milestone in treating cholestatic pruritus in patients with Alagille syndrome, expanding access to a much-needed therapy.

What data supported the CADTH recommendation for LIVMARLI reimbursement?

The CADTH recommendation was based on data from the pivotal ICONIC study, demonstrating significant reductions in pruritus and serum bile acids in patients with Alagille syndrome.

Why is the approval of LIVMARLI important for patients with Alagille syndrome in Canada?

The approval of LIVMARLI in Canada is crucial as it is the first and only approved medication to treat cholestatic pruritus in patients with Alagille syndrome, providing hope and relief to patients and their families.

What is the impact of the CADTH recommendation on patients with Alagille syndrome?

The CADTH recommendation signifies a significant advancement in the treatment of cholestatic pruritus, addressing the critical need for effective therapies for patients with Alagille syndrome.

Which study provided data for the CADTH recommendation of LIVMARLI for Alagille syndrome?

The CADTH recommendation was supported by data from the ICONIC study, showing durable reductions in pruritus and serum bile acids in patients with Alagille syndrome.