Cholestatic pruritus

Mirum Pharmaceuticals Reports First Quarter 2024 Financial Results and Provides Business Update

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수요일, 5월 8, 2024
Pharmaceutical, Health, Clinical Trials, NDA, Depreciation, New Drug Application, CTX, CHMP, Cholestatic pruritus, Webcast, Cholestasis, VISTAS, Disease, Patient, Event, PFIC, Interim, Medication, Growth

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for first quarter 2024 and provided a business update.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for first quarter 2024 and provided a business update.
  • LIVMARLI first quarter net sales were $42.8 million, representing 47% growth compared to first quarter 2023.
  • Total revenue for the quarter ended March 31, 2024, was $69.2 million compared to $31.6 million for the quarter ended March 31, 2023.
  • Total operating expenses were $95.7 million for the quarter ended March 31, 2024, compared to $58.7 million for the quarter ended March 31, 2023.

Mirum Announces Publication of Phase 3 MARCH Data in The Lancet Demonstrating Benefits of LIVMARLI (maralixibat) in patients with PFIC

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화요일, 5월 7, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, IBAT, Liver disease, Hospital, Safety, The Lancet, TJP2, Cholestatic pruritus, Doctor of Philosophy, Research, Long-term care, Cholestasis, Bilirubin, Hepatology, Week, Data, Patient, Diarrhea, Itch, Serum, MDR3, Medication, Lancet, MYO5B, Growth, Pharmaceutical industry

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that data from the Phase 3 MARCH-PFIC study evaluating LIVMARLI® (maralixibat) oral solution in patients with PFIC was published in The Lancet Gastroenterology and Hepatology.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that data from the Phase 3 MARCH-PFIC study evaluating LIVMARLI® (maralixibat) oral solution in patients with PFIC was published in The Lancet Gastroenterology and Hepatology.
  • The MARCH-PFIC study was the largest, randomized, double-blind, placebo-controlled study in patients with PFIC, confirming the efficacy and safety of LIVMARLI, an IBAT inhibitor, across a broad genetic range of PFIC types.
  • “These data advance our understanding of LIVMARLI’s potential to provide meaningful improvements in pruritus and a number of parameters impacting patients with various PFIC types.
  • These data demonstrate the significant impact LIVMARLI can have in this severe cholestatic setting.”
    To read the full article, please visit the publication on The Lancet Gastroenterology and Hepatology’s website .

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

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화요일, 4월 2, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, Itch, Medication, Alagille syndrome, Liver disease, Inflammation, Patient, Cholestasis, Bile, Cholestatic pruritus, CADTH, Liver injury, The Hospital, Liver, CDEC, Genetic disorder, PFIC, Pharmaceutical industry

“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.

Key Points: 
  • “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.
  • 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.
  • 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.
  • Cholestasis in ALGS is associated with pruritus which is among the most common indications for liver transplant in ALGS.

Mirum Pharmaceuticals’ LIVMARLI Receives FDA Approval for Treatment of Cholestatic Pruritus in Patients with Progressive Familial Intrahepatic Cholestasis

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수요일, 3월 13, 2024
Health, FDA, Infectious Diseases, General Health, Clinical Trials, Pharmaceutical, Biotechnology, Growth, Medication, Alagille syndrome, Disease, Patient, Shanda, Sleep, ALGS, Cholestatic pruritus, Quality of life, Life, Cholestasis, Multimedia, Itch, Collection, Family, Food, Pharmaceutical industry

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has approved LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients five years of age and older 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 approved LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients five years of age and older with progressive familial intrahepatic cholestasis (PFIC).
  • View the full release here: https://www.businesswire.com/news/home/20240313135184/en/
    LIVMARLI® (maralixibat) oral solution is now available for prescribing in the US for cholestatic pruritus in patients with PFIC.
  • “LIVMARLI has the potential to have a transformational impact for patients with cholestatic pruritus associated with PFIC, and importantly, offers an option for those patients with the rarest of subtypes,” said Chris Peetz, chief executive officer at Mirum.
  • We are hopeful that LIVMARLI will have a measurable impact for patients, potentially offering a new normal and relief from the sleepless nights and disruption to their lives caused by 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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화요일, 1월 2, 2024
Medical Subject Headings, Liver, Diarrhea, Marketing, Bile, INN, Chronic pain, ATC, International, Hepatology, Disease, Ageing, Blood, Language, Mouth, Abdominal pain, Medicine, European Medicines Agency, ALGS, Cholestatic pruritus, Cholestasis, Select, Patient, Safety, Anatomy, IBAT, IIA, Medical device

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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월요일, 11월 13, 2023
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Infectious Diseases, Clinical Trials, Sleep, Hepatology, MDR3, Self-harm, Safety, Hepatocellular carcinoma, Bilirubin, Cholestasis, AASLD, Liver, Disease, Risk, Cholestatic pruritus, Hematology, Quality of life (healthcare), MYO5B, PFIC, Patient, Publication, Pregnancy, Nature, TJP2, Ascending cholangitis, GI, Hippeastrum mirum, Liver support system, Volixibat, ICP, IBAT, Progressive familial intrahepatic cholestasis, FIC, Augmented fifth, Growth, Doctor of Philosophy, Quality of life, AND, Itch, Medication, MCID, HIV disease progression rates, SBA, Chloride, Pharmaceutical industry, Nursing, Medical imaging, Dietary supplement, Boston, BSEP

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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월요일, 10월 30, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, Cholestasis, TJP2, Publication, Itch, Hepatology, Medication, Quality of life, American Association for the Study of Liver Diseases, Pregnancy, MYO5B, Cholestatic pruritus, Safety, Patient, Liver cancer, AASLD, Pharmaceutical industry

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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월요일, 10월 23, 2023
Ethnographic Museum of the University of Zurich, Patient, American College, IL-31, Itch, UDCA, The Duke of Edinburgh's International Award - Canada, Hope, Pathology, Conditional sentence, NRS, Liver, ALP, American College of Gastroenterology, HIV disease progression rates, PBC, Ursodeoxycholic acid, Risk, European Association for the Study of the Liver, Research and development, Cholestatic pruritus, Alkaline phosphatase, Primary biliary cholangitis, University, EASL, Cholestasis, Atopic dermatitis, Therapy, Doctor of Philosophy, Pharmaceutical industry, Research, MD

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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월요일, 10월 23, 2023
FDA, Food, Itch, Cirrhosis, Breakthrough therapy, Conditional sentence, Liver, ALP, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, PBC, Child, Obeticholic acid, Cholestasis, Cholestatic pruritus, Alkaline phosphatase, Primary biliary cholangitis, NDA, Therapy, Patient, Pharmaceutical industry, Agency

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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월요일, 10월 23, 2023
Ageing, Incidence, CHMP, Itch, Committee, ADR, European Directive on Traditional Herbal Medicinal Products, European Medicines Agency, PFIC, Development, COMP, Phase, Medicine, IPN, EMA, Cholestatic pruritus, Nutrition, Biliary atresia, Committee for Medicinal Products for Human Use, Safety, European Society for Medical Oncology, Ipsen, Odevixibat, Patient, Diagnosis, Pharmaceutical industry, Euronext, Research, Alagille syndrome, ALGS

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.