Mirum’s LIVMARLI Now Approved for PFIC in Patients 12 Months and Older
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.
Mirum Pharmaceuticals (Nasdaq: MIRM) ha annunciato l'approvazione da parte della FDA per un'etichetta ampliata della LIVMARLI® (maralixibat) in forma di soluzione orale. La nuova etichetta include il trattamento per il prurito colestatico nei pazienti con colestasi intraepatica familiare progressiva (PFIC) di età pari o superiore a 12 mesi. Questa espansione, basata sullo studio MARCH di fase 3, include anche una formulazione a concentrazione superiore di LIVMARLI.
Chris Peetz, CEO di Mirum, ha espresso entusiasmo per il lancio di LIVMARLI per i pazienti affetti da PFIC, sottolineando l'importanza di iniziare il trattamento precocemente per ridurre la sofferenza causata dal prurito associato a questa rara malattia epatica. L'etichetta ampliata consente il trattamento in pazienti più giovani, in linea con la diagnosi precoce tipica della PFIC.
Mirum Pharmaceuticals (Nasdaq: MIRM) anunció la aprobación de la FDA para una etiqueta ampliada de LIVMARLI® (maralixibat) en solución oral. La nueva etiqueta incluye el tratamiento para prurito colestático en pacientes con colestasis intrahepática familiar progresiva (PFIC) de 12 meses o más. Esta ampliación, basada en el estudio MARCH de fase 3, también incluye una formulación de mayor concentración de LIVMARLI.
Chris Peetz, CEO de Mirum, expresó su entusiasmo por el lanzamiento de LIVMARLI para pacientes con PFIC, enfatizando la importancia de iniciar el tratamiento temprano para reducir el sufrimiento por el prurito asociado con esta rara enfermedad hepática. La etiqueta ampliada permite el tratamiento en pacientes más jóvenes, alineándose con el diagnóstico temprano típico de la PFIC.
Mirum Pharmaceuticals (Nasdaq: MIRM)는 LIVMARLI® (maralixibat) 경구용 용액의 확장된 라벨에 대해 FDA의 승인을 발표했습니다. 새로운 라벨에는 12개월 이상의 진행성 가족성 간내 담즙 정체 (PFIC) 환자의 담즙성 가려움증 치료가 포함됩니다. 이 확장은 MARCH 3상 연구를 기반으로 하며, LIVMARLI의 고농도 제형도 포함됩니다.
Mirum의 CEO인 Chris Peetz는 PFIC 환자를 위한 LIVMARLI의 출시 소식에 대해 열정을 표명하며, 이 희귀 간 질환과 관련된 가려움증으로 인한 고통을 줄이기 위해 조기 치료가 얼마나 중요한지를 강조했습니다. 확장된 라벨은 젊은 환자 치료를 허용하여 PFIC의 조기 진단과 일치합니다.
Mirum Pharmaceuticals (Nasdaq: MIRM) a annoncé l'approbation par la FDA d'une étiquette élargie pour la solution orale LIVMARLI® (maralixibat). La nouvelle étiquette inclut le traitement du prurit cholestatique chez les patients atteints de cholestase intra-hépatique familiale progressive (PFIC) âgés de 12 mois et plus. Cette expansion, basée sur l'étude MARCH de phase 3, inclut également une formulation à concentration plus élevée de LIVMARLI.
Chris Peetz, PDG de Mirum, a exprimé son enthousiasme pour le lancement de LIVMARLI pour les patients atteints de PFIC, en soulignant l'importance d'un début de traitement précoce pour réduire la souffrance causée par le prurit associé à cette maladie hépatique rare. L'étiquette élargie permet le traitement des patients plus jeunes, en adéquation avec le diagnostic précoce typique de la PFIC.
Mirum Pharmaceuticals (Nasdaq: MIRM) hat die Genehmigung der FDA für ein erweitertes Label der LIVMARLI® (maralixibat) als orale Lösung bekannt gegeben. Das neue Label umfasst die Behandlung von cholestatischem Juckreiz bei Patienten mit progressiver familiärer intrahepatischer Cholestase (PFIC), die 12 Monate oder älter sind. Diese Erweiterung, basierend auf der MARCH-Phase-3-Studie, umfasst auch eine höher konzentrierte Formulierung von LIVMARLI.
Chris Peetz, CEO von Mirum, äußerte seine Begeisterung über die Einführung von LIVMARLI für PFIC-Patienten und betonte die Bedeutung eines frühen Behandlungsbeginns, um das Leiden durch den mit dieser seltenen Lebererkrankung verbundenen Juckreiz zu verringern. Das erweiterte Label ermöglicht die Behandlung bei jüngeren Patienten und stimmt mit der typischen frühen Diagnose von PFIC überein.
- 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
- None.
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.
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.
“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
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
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
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
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?
What condition does LIVMARLI treat in PFIC patients?
What study supported the expanded label for LIVMARLI (MIRM)?