ALGS

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

Retrieved on: 
Wednesday, March 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.”

Hepatology Publishes Six-Year Data Demonstrating Improved Clinical Outcomes in Patients with Alagille Syndrome Treated with Mirum’s LIVMARLI

Retrieved on: 
Thursday, February 29, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, Medication, ALGS, Alagille syndrome, Patient

The analysis evaluated time to clinical outcome from the pooled LIVMARLI clinical studies versus a control cohort from the Global Alagille Alliance (GALA) clinical database, the largest global ALGS natural history database.

Key Points: 
  • The analysis evaluated time to clinical outcome from the pooled LIVMARLI clinical studies versus a control cohort from the Global Alagille Alliance (GALA) clinical database, the largest global ALGS natural history database.
  • The GALA control group was identified based on a pre-specified and blinded selection process to align with eligibility criteria from the LIVMARLI clinical studies.
  • Data from the analysis demonstrated a significant improvement in six-year event-free survival with a p-value of
  • The full publication including additional data from the analysis is available on the Hepatology website.

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

Retrieved on: 
Tuesday, January 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

Ipsen delivers solid sales growth in the first nine months of 2023 and confirms its full-year guidance

Retrieved on: 
Thursday, October 26, 2023
FDA, American Association for the Study of Liver Diseases, FY, Food, Progression-free survival, International, Heart, Hepatology, Committee, ADR, Rare disease, GMT, European Directive on Traditional Herbal Medicinal Products, CER1, Commerce, Arbitral tribunal, News, Symantec Endpoint Protection, European Medicines Agency, Alagille syndrome, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Research, ALGS, Cabozantinib, AB toxin, Webcast, Partnership, IPN, U.S. FDA, EMA, Primary biliary cholangitis, Committee for Medicinal Products for Human Use, Ipsen, Patient, YTD, AASLD, Fine chemical, Pharmaceutical industry, Cryptocurrency, Euronext, Neuroscience, Oncology, Odevixibat

Our portfolio has performed well across the three therapy areas, driven by strengthened commercial execution and the results of our external-innovation strategy.

Key Points: 
  • Our portfolio has performed well across the three therapy areas, driven by strengthened commercial execution and the results of our external-innovation strategy.
  • Based on the solid sales momentum, today we are confirming our guidance for the full year.
  • This was despite a positive opinion from the Committee for Medicinal Products for Human Use in July 2023.
  • The Company is planning to host a capital-markets event, starting at 12.30pm GMT on 7 December 2023 in London.

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

Retrieved on: 
Monday, October 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.

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

Retrieved on: 
Saturday, October 7, 2023
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, Hypercholesterolemia, Doctor of Philosophy, Food, ALGS, Bile, Safety, SBA, Hippeastrum mirum, Xanthoma, Cholestatic pruritus, FDA, Poster, Maintenance, Itch, Clinical trial, Publication, Alagille syndrome, Sleep, PFIC, Medication, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, RISE, Bilirubin, Patient, Quality of life, Pharmaceutical industry, Dietary supplement, Nursing

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)

Retrieved on: 
Monday, October 9, 2023
Cholestatic pruritus, IBAT, Cholestasis, Food, Bile, Death, Infant, Liver disease, Ageing, Interim, Tuba mirum, Disease, Patient, BA, System, Itch, Rare, Marketing, RISE, Genetic disorder, PFIC, Alagille syndrome, Biliary atresia, Research, Quality of life, Maralixibat chloride, Cirrhosis, Pharmaceutical industry, ALGS, BURLINGTON

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 Reports Second Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Thursday, August 3, 2023
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, Exercise, ALGS, Partnership, CTX, Spiroplasma mirum, Event, Hepatology, Travers Smith, PBC, VISTAS, Prescription Drug User Fee Act, Primary sclerosing cholangitis, Biliary atresia, CAMEO, Sale, Medication, Alagille syndrome, Cholic acid, Primary biliary cholangitis, Shanda, Acquisition, Patient, Investment, Itch, Bilirubin, PDUFA, Webcast, Therapy, PSC, Cholestatic pruritus, Volixibat, Pharmaceutical industry, Security (finance), Video game

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for second quarter 2023 and provided a business update.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for second quarter 2023 and provided a business update.
  • Mirum to pay $210 million upfront and up to $235 million in potential tiered annual net sales-based milestones.
  • Second quarter 2023 global net sales grew 86% as compared to second quarter 2022, on track for 50% US net product sales growth in 2023 as compared to 2022.
  • Total operating expenses were $61.8 million for the quarter ended June 30, 2023 in comparison to $48.9 million for the quarter ended June 30, 2022.

Ipsen delivers solid H1 2023 results and upgrades its full-year guidance

Retrieved on: 
Thursday, July 27, 2023
IPN, Heart, Cholestatic pruritus, Maintenance, CHC, FY, Ascending cholangitis, Civil service commission, ADR, Prescription Drug User Fee Act, Fibrodysplasia ossificans progressiva, Ursodeoxycholic acid, Account, Ipsen, Galderma, Bank statement, Oxaliplatin, COMP, Rare disease, New Drug Application, European Directive on Traditional Herbal Medicinal Products, Research, Committee, EMA, ICC, Primary biliary cholangitis, Report, FOP, Patient, Committee for Medicinal Products for Human Use, PDUFA, Webcast, Consumer, Growth, Ageing, Alagille syndrome, Indoor Living, FDA, Regulation of tobacco by the U.S. Food and Drug Administration, Food, European Commission, Safety, CHMP, Pharmaceutical industry, Medical device, Cryptocurrency, ALGS, Oncology, Neuroscience, Odevixibat, Euronext

The performance in the first half of the year is reflected in today’s upgrade to our full-year guidance.

Key Points: 
  • The performance in the first half of the year is reflected in today’s upgrade to our full-year guidance.
  • The Company’s auditors performed a limited review of the H1 2023 condensed consolidated financial statements.
  • Ipsen intends to publish its year-to-date and third-quarter sales update on 26 October 2023.
  • The performance shown in this announcement covers the six-month period to 30 June 2023 (H1 2023) and the three-month period to 30 June 2023 (Q2 2023), compared to six-month period to 30 June 2022 (H1 2022) and the three-month period to 30 June 2022 (Q2 2022), respectively, unless stated otherwise.

Mirum’s LIVMARLI® Now Authorized in Canada for Cholestatic Pruritus in Patients with Alagille Syndrome

Retrieved on: 
Tuesday, July 25, 2023
Biotechnology, Pharmaceutical, Health, Hospital for Sick Children (Toronto), Bile, Tuba Mirum (Paus), Life, Liver disease, Cholestasis, Liver injury, Multimedia, Inflammation, Hospital for Sick Children, Alagille syndrome, EMA, Publication, Genetic disorder, Patient, Child, Itch, Liver, Hospital, FDA, Lancet, Health Canada, PFIC, Cholestatic pruritus, Disease, Pharmaceutical industry, Nursing, ALGS, Hepatology

Cholestasis in ALGS is associated with pruritus which is among the most common indications for liver transplant in ALGS.

Key Points: 
  • Cholestasis in ALGS is associated with pruritus which is among the most common indications for liver transplant in ALGS.
  • “Today’s approval in Canada furthers our commitment to ensuring patients globally have a meaningful and effective treatment option for the unrelenting burden of cholestatic pruritus impacting patients and their families living with ALGS,” said Chris Peetz, president and chief executive officer at Mirum.
  • Results from these studies showing robust and meaningful improvements for patients with cholestatic pruritus associated with ALGS were featured in several publications, including The Lancet and Hepatology .
  • Regulatory submissions outside of the U.S., Canada and Europe are being pursued by partners and distributors in key markets globally.