Latest Hotspot

European Union Approval for Mirum Pharmaceuticals’ LIVMARLI to Treat PFIC Patients

10 July 2024
3 min read

Mirum Pharmaceuticals, Inc. revealed that the European Commission has approved the marketing authorization for LIVMARLI® (maralixibat) oral solution, intended for treating progressive familial intrahepatic cholestasis in patients aged three months and above.

👇Unlock in-depth information about this drug - its R&D Status, Core Patent, Clinical Trials, and Global Approval Status. Click on the image below and explore the latest data immediately.

The CHMP's positive assessment has led to the approval of LIVMARLI for PFIC, highlighting its significant clinical benefits, including enhanced efficacy and notable improvements in patient care compared to existing PFIC treatments. Furthermore, the COMP's evaluation upheld the Orphan Drug Designation for LIVMARLI in PFIC.

"We are delighted that the European Commission has authorized the marketing of LIVMARLI for PFIC, recognizing the robust data and the valuable treatment opportunity it offers for patients suffering from this rare liver condition," stated Chris Peetz, CEO of Mirum. "Our aim is for LIVMARLI to enhance critical liver parameters and bring better health prospects to young PFIC patients in Europe. We are deeply thankful to the researchers, patients, and families who contributed to this approval."

"The approval of LIVMARLI represents a treatment that has substantial clinical backing, data indicating reduced cholestatic itch, and improved liver health markers, such as decreased serum bile acids—key indicators of better long-term outcomes," said Professor Richard Thompson of King’s College London. "It's reassuring for European physicians to have a new treatment option that can potentially enhance liver health and the quality of life for patients and their families."

Emily Ventura, executive director of the PFIC Network, commented, "The European patient community will significantly benefit from LIVMARLI's approval, which is supported by extensive data showing improvements in the most challenging aspects of the disease. PFIC can drastically alter lives and severely impact patients. The approval provides hope for younger patients to experience relief from cholestasis and its burdens."

LIVMARLI has also received approval from the U.S. Food and Drug Administration for treating cholestatic pruritus in PFIC patients aged five and older. Mirum has submitted a supplemental new drug application to the FDA for a higher concentration formulation used in the MARCH study, aiming to expand the label for younger PFIC patients, with feedback expected within the year.

👇Explore the latest research progress on drug-related developments, indications, therapeutic organizations, clinical trials, results, and patents by clicking on the targeted picture link below. Unfold a world of comprehensive information on this target in just a click!

图形用户界面, 文本, 应用程序, 电子邮件

描述已自动生成

According to the data provided by the Synapse Database, As of July 10, 2024, there are 17 investigational drugs for the ISBT target, including 25 indications, 24 R&D institutions involved, with related clinical trials reaching 118, and as many as 2850 patents.

Maralixibat Chloride is a small molecule drug that targets the ileal bile acid transporter (ISBT). Maralixibat Chloride represents a significant advancement in the treatment of various cholestatic and liver-related conditions. Its approval in both the United States and China, as well as the regulatory designations it has received, underscore the potential impact of this small molecule drug in addressing the needs of patients with these challenging medical conditions. 

图形用户界面, 文本, 应用程序

描述已自动生成

Is Trilaciclib approved by the FDA?
Drug Insights
3 min read
Is Trilaciclib approved by the FDA?
10 July 2024
Trilaciclib was approved by the U.S. Food and Drug Administration (FDA) on February 12, 2021.
Read →
The European Union has approved Dupixent® (dupilumab) as the first targeted therapy for COPD patients
Latest Hotspot
4 min read
The European Union has approved Dupixent® (dupilumab) as the first targeted therapy for COPD patients
10 July 2024
Regeneron Pharmaceuticals, Inc. and Sanofi announced today that the European Commission has granted approval for Dupixent (dupilumab) as an additional maintenance therapy for adults with uncontrolled chronic obstructive pulmonary disease with elevated blood eosinophils.
Read →
Is Evinacumab approved by the FDA?
Drug Insights
3 min read
Is Evinacumab approved by the FDA?
10 July 2024
Evinacumab was approved by the U.S. Food and Drug Administration (FDA) on February 11, 2021.
Read →
Cartesian Therapeutics Administers First Dose in Phase 2 Study of Descartes-08 for Systemic Lupus Erythematosus
Latest Hotspot
3 min read
Cartesian Therapeutics Administers First Dose in Phase 2 Study of Descartes-08 for Systemic Lupus Erythematosus
10 July 2024
Cartesian Therapeutics, revealed that the initial patient has been treated in its Phase 2 open-label clinical study assessing Descartes-08 in individuals with systemic lupus erythematosus.
Read →
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.