Biliary atresia

Orphan designation: Autologous adipose-derived mesenchymal stem cells Treatment of oesophageal atresia, 21/04/2023 Positive

Retrieved on: 
Sunday, February 4, 2024
COMP, IRIS, European Commission, Committee, Biliary atresia, EMA, Pharmaceutical industry

EU/3/23/2772 - orphan designation for treatment of oesophageal atresia

Key Points: 
  • EU/3/23/2772 - orphan designation for treatment of oesophageal atresia
    Autologous adipose-derived mesenchymal stem cells
    OrphanHuman
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Mirum Pharmaceuticals Announces Data from EMBARK Phase 2b Study for Biliary Atresia

Retrieved on: 
Monday, December 18, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, Liver, Spiroplasma mirum, Patient, Biliary atresia, Medication, Bilirubin, Science, Medical imaging, Nursing

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), today announced top-line results of the Phase 2 EMBARK study evaluating LIVMARLI® (maralixibat) oral solution versus placebo given as an adjuvant therapy to Kasai surgery in patients with biliary atresia.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), today announced top-line results of the Phase 2 EMBARK study evaluating LIVMARLI® (maralixibat) oral solution versus placebo given as an adjuvant therapy to Kasai surgery in patients with biliary atresia.
  • The study did not meet its primary endpoint of mean change in bilirubin from baseline to Week 26, or the key secondary endpoints.
  • “We are disappointed in the outcome of the study in this post-surgery, high-need disease setting,” said Chris Peetz, president and chief executive officer at Mirum.
  • “We are grateful to the patients, families, and clinicians who participated in the study and advanced the science around this rare liver disease.”

Mirum Pharmaceuticals Reports Third Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Thursday, November 2, 2023
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, Xanthoma, FDA, CTX, BASD, PDUFA, Food, Event, Medication, VISTAS, Acquisition, Alagille syndrome, PFIC, Society, Cholic acid, Webcast, Zellweger spectrum disorders, Worldwide Angel, Biliary atresia, NDA, Investment, Patient, Shanda, Pharmaceutical industry, Nursing, Cryptocurrency, Gastroenterology, PBC, PSC, Nutrition

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

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for third quarter 2023 and provided a business update.
  • Third quarter 2023 net sales of $9 million recorded since acquisition close August 31st, 2023.
  • Total net revenue for the quarter ended September 30, 2023, was $47.7 million compared to $18.8 million for the quarter ended September 30, 2022.
  • Total operating expenses were $72.9 million for the quarter ended September 30, 2023, compared to $51.7 million for the quarter ended September 30, 2022.

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.

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 provides update on E.U. Marketing Authorization Application for Bylvay® for cholestatic pruritus in patients with Alagille syndrome  

Retrieved on: 
Friday, July 21, 2023
IPN, Maintenance, Ascending cholangitis, ADR, COMP, Skin, Quality of life, Biliary atresia, EMA, U.S. FDA, Committee, Incidence, Development, European Commission, Patient, Itch, Committee for Medicinal Products for Human Use, European Medicines Agency, Cholestasis, Liver, Ageing, Congress, European Society of Neurogastroenterology and Motility, Phase, PFIC, Cholestatic pruritus, CHMP, Pharmaceutical industry, ALGS, Nutrition, Ipsen, Alagille syndrome, Odevixibat, Euronext, Research

This negative COMP opinion prevents the retention of orphan-drug status in Bylvay’s marketing authorization in ALGS and might delay a final European Commission decision.

Key Points: 
  • This negative COMP opinion prevents the retention of orphan-drug status in Bylvay’s marketing authorization in ALGS and might delay a final European Commission decision.
  • for a second orphan indication for Bylvay, the treatment of pruritus in patients with ALGS.
  • More than 90% of patients were pruritus responders (≥ one point change at any time during 24 weeks).
  • In June 2023, Bylvay was also approved in the U.S. for the treatment of cholestatic pruritus in patients from 12 months of age with ALGS.

U.S. FDA approves Bylvay® for patients living with cholestatic pruritus due to Alagille syndrome

Retrieved on: 
Tuesday, June 13, 2023
IPN, ALGS, Cholestatic pruritus, Apache iBATIS, Ascending cholangitis, ADR, Ipsen, European Medicines Agency, European Directive on Traditional Herbal Medicinal Products, Biliary atresia, Trial of the century, EMA, Committee, Incidence, Development, MD, Patient, Physician, Hepatology, Child, Itch, Committee for Medicinal Products for Human Use, Division, NYU Langone Health, Cholestasis, Liver, Ageing, Hospital, Phase, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, PFIC, Food, Disease, Pharmaceutical industry, Alagille syndrome, Odevixibat, Euronext, Gastroenterology, Research

Bylvay was approved as the first drug treatment option for patients living with cholestatic pruritus due to progressive familial intrahepatic cholestasis (PFIC) in the U.S., and for the treatment of PFIC in Europe, in 2021.

Key Points: 
  • Bylvay was approved as the first drug treatment option for patients living with cholestatic pruritus due to progressive familial intrahepatic cholestasis (PFIC) in the U.S., and for the treatment of PFIC in Europe, in 2021.
  • More than 90% of patients were pruritus responders (≥ 1 point change at any time during 24 weeks).
  • No patients discontinued the study and 96% of patients rolled over into the open-label extension study.
  • In a third indication, the rare pediatric cholestatic liver disease, biliary atresia, Bylvay is in late-stage development with the Phase III BOLD trial.

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

Retrieved on: 
Friday, June 2, 2023
Health, General Health, Research, Pharmaceutical, Science, Biotechnology, ALGS, Marketing, Liver disease, PFIC, Biliary atresia, Maralixibat chloride, Patient, Cholestasis, Policy, Alagille syndrome, NMPA, Cholestatic pruritus, Pharmaceutical industry

LIVMARLI is approved in the United States for the treatment of cholestatic pruritus in patient with ALGS in patients three months of age and older and is authorized in Europe for patients two months and older.

Key Points: 
  • LIVMARLI is approved in the United States for the treatment of cholestatic pruritus in patient with ALGS in patients three months of age and older and is authorized in Europe for patients two months and older.
  • LIVMARLI was approved for cholestatic pruritus last year under the Early and Pilot Implementation Policy in the Boao Lecheng International Medical Tourism Pilot Zone in China.
  • LIVMARLI is the only approved medication in China to treat cholestatic pruritus associated with ALGS.
  • We look forward to bringing this promising new treatment to thousands of ALGS patients in China and to developing LIVMARLI for other indications.

CANbridge Announces Full Enrollment Reached in China Region of the Global LIVMARLI® (CAN108) EMBARK Phase 2 Study in Biliary Atresia

Retrieved on: 
Tuesday, May 30, 2023
Health, Science, Pharmaceutical, Research, Clinical Trials, ALGS, National Medical Products Administration, NDA, PFIC, Biliary atresia, Doctor of Philosophy, MD, Patient, Cholestasis, Policy, Alagille syndrome, NMPA, BA, Cholestatic pruritus, Safety, Disease, Pharmaceutical industry

This global, multi-center, randomized controlled Phase 2 study, evaluating the efficacy and safety of LIVMARLI in patients with BA after Kasai surgery, is sponsored by Mirum Pharmaceuticals, Inc. and has also completed enrollment across the other global locations where the trial is open.

Key Points: 
  • This global, multi-center, randomized controlled Phase 2 study, evaluating the efficacy and safety of LIVMARLI in patients with BA after Kasai surgery, is sponsored by Mirum Pharmaceuticals, Inc. and has also completed enrollment across the other global locations where the trial is open.
  • CANbridge has an exclusive license agreement for the development, commercialization and manufacturing, under certain conditions, of LIVMARLI in Greater China.
  • In this region, CANbridge has the right to develop and commercialize LIVMARLI for three indications: Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA).
  • CANbridge also expects to file an NDA for LIVMARLI for the treatment of PFIC with the NMPA soon.