Cholestatic pruritus

Human medicines European public assessment report (EPAR): Livmarli, Maralixibat chloride, Date of authorisation: 09/12/2022, Revision: 4, Status: Authorised

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Tuesday, January 2, 2024

Human medicines European public assessment report (EPAR): Livmarli, Maralixibat chloride, Date of authorisation: 09/12/2022, Revision: 4, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Livmarli, Maralixibat chloride, Date of authorisation: 09/12/2022, Revision: 4, Status: Authorised

Mirum Pharmaceuticals Presents New Data at The Liver Meeting®

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Monday, November 13, 2023

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced new data from its LIVMARLI and volixibat programs presented at The Liver Meeting®, the American Association for the Study of Liver Diseases (AASLD) annual meeting in Boston, Massachusetts.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced new data from its LIVMARLI and volixibat programs presented at The Liver Meeting®, the American Association for the Study of Liver Diseases (AASLD) annual meeting in Boston, Massachusetts.
  • “PFIC is a chronic, debilitating cholestatic liver disease characterized by elevated bile acids and pruritus so severe that patients can be listed for liver transplant even in the absence of disease progression.
  • These data suggest overall improved liver health with maralixibat treatment in patients with PFIC that can be maintained long-term.
  • No clinically meaningful changes in liver enzyme levels or hematology parameters were observed after volixibat treatment.

Mirum Pharmaceuticals to Present New Data at The Liver Meeting®

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Monday, October 30, 2023

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that data from its LIVMARLI and volixibat programs will be presented at The Liver Meeting®, the American Association for the Study of Liver Diseases (AASLD) annual meeting, taking place November 10-14, 2023, in Boston, Massachusetts.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that data from its LIVMARLI and volixibat programs will be presented at The Liver Meeting®, the American Association for the Study of Liver Diseases (AASLD) annual meeting, taking place November 10-14, 2023, in Boston, Massachusetts.
  • Poster 4602-C: Improvements in pruritus with maralixibat are associated with improved quality of life for patients with progressive familial intrahepatic cholestasis: Data from the MARCH-PFIC trial
    Poster 4604-C: Maralixibat leads to significant improvement in cholestatic pruritus for children with progressive familial intrahepatic cholestasis due to TJP2 or MYO5B deficiency: Data from the MARCH-PFIC trial
    Poster 4605-C: Maralixibat leads to significant improvements in cholestatic pruritus for children with progressive familial intrahepatic cholestasis without a genetic diagnosis: Data from the MARCH-PFIC trial
    Poster 4547-C: Efficacy, safety and tolerability of volixibat in patients with intrahepatic cholestasis of pregnancy: A case series of 4 patients
    Full data will be available within the Publications and Presentations section on Mirum’s website on November 13.

CymaBay Presents Results on the Potential of Seladelpar in Treatment of Patients with Primary Biliary Cholangitis at ACG 2023

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Monday, October 23, 2023

VANCOUVER, British Columbia, Oct. 23, 2023 (GLOBE NEWSWIRE) -- CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a biopharmaceutical company focused on innovative therapies for patients with liver and other chronic diseases, announced today the presentation of findings from its post-hoc analysis of the Phase 3 ENHANCE study of seladelpar for the treatment of primary biliary cholangitis (PBC), showing baseline intensity of patient-reported pruritus was associated with higher levels of serum IL-31. The presentation, named the recipient of this year’s International Award by the American College of Gastroenterology, will be presented by Professor Andreas E. Kremer, MD, Ph.D., MHBA, a leading authority in cholestatic pruritus from the University of Zurich. Featured results included novel aspects of the anti-pruritic and anti-cholestatic mechanisms of seladelpar, CymaBay’s first-in-class oral, selective PPARδ agonist, or "delpar," being investigated for the treatment of patients with PBC.

Key Points: 
  • Featured results included novel aspects of the anti-pruritic and anti-cholestatic mechanisms of seladelpar, CymaBay’s first-in-class oral, selective PPARδ agonist, or "delpar," being investigated for the treatment of patients with PBC.
  • The data were previously presented at the European Association for the Study of the Liver (EASL)’s The International Liver Congress™ 2023 in Vienna, Austria.
  • “These results offer a glimmer of hope in that they link IL-31 levels in patients with PBC to itch.
  • Elevated risk due to ELF was identified in 43.2% of patients who currently meet guidelines for second-line treatment vs. 27.2% within patient groups not recommended for second-line treatment.

Seladelpar Granted Revised Breakthrough Therapy Designation for the Treatment of Primary Biliary Cholangitis Including Pruritus in Patients Without Cirrhosis or With Compensated Cirrhosis

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Monday, October 23, 2023

Seladelpar is the only potent, selective, orally active PPARδ agonist, or delpar, with phase 3 results demonstrating a statistically significant improvement in PBC-related cholestatic pruritus.

Key Points: 
  • Seladelpar is the only potent, selective, orally active PPARδ agonist, or delpar, with phase 3 results demonstrating a statistically significant improvement in PBC-related cholestatic pruritus.
  • This program was designed by the FDA to help ensure patients gain access to important new therapies through FDA approval as soon as possible.
  • The updated Breakthrough Therapy Designation of seladelpar was granted based on additional evidence provided to the Agency, which supports that seladelpar may provide substantial improvement over existing therapy based on a reduction in alkaline phosphatase (ALP) and pruritus in patients without cirrhosis or with compensated cirrhosis.
  • “A Breakthrough Therapy designation is accompanied by the benefit to submit the NDA on a rolling basis.

Ipsen updates on E.U. Marketing Authorisation Application for odevixibat in Alagille syndrome

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Monday, October 23, 2023

This is despite a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) in July 2023.

Key Points: 
  • This is despite a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) in July 2023.
  • Orphan designation has a strong influence on the reimbursement mechanisms and access for patients to medicines in some countries in the E.U.
  • “We are disappointed with the opinion of the COMP as the Orphan Medicinal Product Regulation aims to stimulate research and development for rare diseases.
  • In the trial, there were no patient discontinuations and 96% of patients rolled over into the open-label extension trial.

Mirum Pharmaceuticals Announces New PDUFA Date for LIVMARLI for the Treatment of Cholestatic Pruritus in Progressive Familial Intrahepatic Cholestasis

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Tuesday, October 17, 2023

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has extended the review of the Supplemental New Drug Application (sNDA) for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC).

Key Points: 
  • Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has extended the review of the Supplemental New Drug Application (sNDA) for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC).
  • The new Prescription Drug User Fee Act (PDUFA) date is March 13, 2024.
  • The FDA extended the PDUFA date to allow time for a full review of a submission provided in response to an FDA information request, which the FDA deemed a major amendment.
  • The submission comprised further analysis of certain clinical information that was previously provided to the FDA by the company.

Mirum Pharmaceuticals Presents LIVMARLI Clinical Data and Real-World Evidence at NASPGHAN

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Saturday, October 7, 2023

Data presented during NASPGHAN demonstrated continued evidence of the impact of LIVMARLI for patients with PFIC as well as both clinical data and real-world use of LIVMARLI in patients with ALGS.

Key Points: 
  • Data presented during NASPGHAN demonstrated continued evidence of the impact of LIVMARLI for patients with PFIC as well as both clinical data and real-world use of LIVMARLI in patients with ALGS.
  • Presentations included:
    Data from the MARCH PFIC Phase 3 Study was awarded the prestigious Gerard Odell prize for Excellence in Pediatric Hepatology Research.
  • The MARCH PFIC clinical trial was conducted across the broadest range of PFIC types ever studied.
  • Real-world data in patients with ALGS was presented on adherence to LIVMARLI as well as reduction of other medications and vitamin supplementation while on LIVMARLI.

CANbridge Announces Marketing Approval of CAN108 (/LIVMARLI®) in Taiwan for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome (ALGS)

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Monday, October 9, 2023

LIVMARLI is a minimally absorbed ileal bile acid transporter (IBAT) inhibitor, and the first and only treatment approved medication in Taiwan for the treatment of cholestatic pruritus (itching caused by slowed or stalled bile flow) in patients with Alagille syndrome (ALGS) aged one year or older.

Key Points: 
  • LIVMARLI is a minimally absorbed ileal bile acid transporter (IBAT) inhibitor, and the first and only treatment approved medication in Taiwan for the treatment of cholestatic pruritus (itching caused by slowed or stalled bile flow) in patients with Alagille syndrome (ALGS) aged one year or older.
  • ALGS is a rare genetic disorder that can lead to end-stage liver disease and death.
  • Cholestatic pruritus is the most burdensome symptom in ALGS, greatly reducing quality of life.
  • The availability of LIVMARLI in Taiwan represents a significant turning point in addressing the unique challenges faced by individuals affected by ALGS.

Mirum Pharmaceuticals to Highlight LIVMARLI Data from its ALGS and PFIC Programs at the NASPGHAN Annual Meeting

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Tuesday, September 26, 2023

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that data from its LIVMARLI® (maralixibat) oral solution studies as well as data from real- world experience will be presented at the upcoming North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) annual meeting taking place October 4-7, 2023, in San Diego, California.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that data from its LIVMARLI® (maralixibat) oral solution studies as well as data from real- world experience will be presented at the upcoming North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) annual meeting taking place October 4-7, 2023, in San Diego, California.
  • Highlighted below are the titles that have been accepted for presentation during the meeting.
  • The abstracts are available via the NASPGHAN website .
  • Full analyses will be available following their presentation within the Publications & Presentations section on Mirum’s website .