Maralixibat

CANbridge Pharmaceuticals CAN108 New Drug Application (NDA) for Alagille Syndrome Accepted by China’s National Medical Products Administration

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星期二, 一月 18, 2022
Education, Health, Research, Science, Pharmaceutical, University, Biotechnology, MPS, New Drug Application, NDA, Research, Patient, Haemophilia, University of Massachusetts, Hunter syndrome, NMPA, Liver, Glioblastoma, Spiroplasma mirum, University of Massachusetts Medical School, Itch, Alagille syndrome, Liver injury, GBM, CEO, Biliary atresia, Jaundice, Hepatomegaly, Genetic distance, Xanthoma, 2-Bromo-LSD, WuXi Biologics, Review, University, University of Washington School of Medicine, Lists of diseases, Frost & Sullivan, IBAT, Primary biliary cholangitis, Excoriation disorder, Heart, Sleep, Disorder, MPS II, Serum, US Foods, Enterohepatic circulation, Glycogen storage disease, Cholestatic pruritus, FDA, Liver disease, PFIC, Pharmaceutical industry, Alagille syndrome (ALGS）, Maralixibat, ALAGILLE SYNDROME (ALGS）, MARALIXIBAT

CANbridge anticipates that the application will be granted priority review, upon the close of the publicity period of the priority review process, on January 17th.

Key Points: 
  • CANbridge anticipates that the application will be granted priority review, upon the close of the publicity period of the priority review process, on January 17th.
  • CANbridge and Mirum Pharmaceuticals signed an exclusive license agreement for the development and commercialization of maralixibat (CAN108) in Greater China last year.
  • Under the terms of the agreement, CANbridge has the right to develop and commercialize CAN108 for three indications: Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA) in Greater China.
  • Alagille syndrome (ALGS) is a rare autosomal dominant disorder that affects multiple organs, including the liver, heart, bones and eyes.

Mirum Pharmaceuticals and Takeda Enter into Exclusive Licensing Agreement to Develop and Commercialize Maralixibat for Rare Pediatric Liver Diseases in Japan

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星期二, 九月 21, 2021
Health, General Health, Research, Pharmaceutical, Science, Biotechnology, Cholestasis, Food, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Marketing, Liver disease, NDA, Primary sclerosing cholangitis, Biliary atresia, Hematology, Health, European Medicines Agency, TSE, Takeda Pharmaceutical Company, Private Securities Litigation Reform Act, U.S. Securities and Exchange Commission, FDA, Alagille syndrome, Multimedia, Patient, Cholestatic pruritus, Abdominal pain, Feces, Pregnancy, Primary biliary cholangitis, Medication, TAK, Itch, BA, BSEP, Security (finance), COVID-19, Vaccine, PDUFA, Genetics, R, Institute of Liver and Biliary Sciences, Investment, PFIC, Quality of life, Nasdaq, Database, Diarrhea, Pharmaceutical industry, Medical device, Maralixibat, Mirum Pharmaceuticals, Inc., Takeda Pharmaceutical Company Limited, MARALIXIBAT, MIRUM PHARMACEUTICALS, INC., TAKEDA PHARMACEUTICAL COMPANY LIMITED

View the full release here: https://www.businesswire.com/news/home/20210921005502/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20210921005502/en/
    Under the terms of the agreement, Takeda will be responsible for regulatory approval and commercialization of maralixibat in Japan.
  • We are excited for Takeda to engage in the development of maralixibat and collaborate in our effort to advance this potentially life-changing therapy.
  • This agreement reinforces Takedas commitment to developing highly differentiated medicines to improve the health and quality of life of patients.
  • Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases.

Mirum Pharmaceuticals Submits European Marketing Authorization Application for Maralixibat in Alagille Syndrome Supported by New Positive Results from Natural History Study Comparison

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星期一, 九月 13, 2021
Biotechnology, FDA, Health, Pharmaceutical, Clinical Trials, European Medicines Agency, Itch, Patient, Marketing, Alagille syndrome, Nasdaq, EMA, Primary sclerosing cholangitis, PFIC, MAA, Medication, Pharmaceutical industry, Maralixibat, Mirum Pharmaceuticals, Inc., MARALIXIBAT, MIRUM PHARMACEUTICALS, INC.

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the submission of a Marketing Authorization Application (MAA) for the treatment of cholestatic liver disease in patients with Alagille syndrome (ALGS) to the European Medicines Agency (EMA).

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the submission of a Marketing Authorization Application (MAA) for the treatment of cholestatic liver disease in patients with Alagille syndrome (ALGS) to the European Medicines Agency (EMA).
  • Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases.
  • Additionally, Mirums marketing authorization application for the treatment of cholestatic liver disease in patients with ALGS has been submitted to the European Medicines Agency.
  • Such forward-looking statements include statements regarding, among other things, the regulatory approval pathway for maralixibat and the safety and efficacy of maralixibat.

Mirum Pharmaceuticals Reports Second Quarter 2021 Financial Results and Provides Business Update

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星期四, 八月 5, 2021
Biotechnology, Pharmaceutical, Health, Clinical Trials, Investor, Primary sclerosing cholangitis, Webcast, Low-density lipoprotein, Abdominal pain, U.S. Securities and Exchange Commission, Prescription Drug User Fee Act, Cholestasis, Primary biliary cholangitis, Liver disease, Biliary atresia, Cholestatic pruritus, Feces, Security (finance), Private Securities Litigation Reform Act, IND, Review, Vistas, European Medicines Agency, Patient, PFIC, ABCB11, COVID-19, Growth, Database, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Volixibat, Spiroplasma mirum, Diarrhea, Food and Drug Administration, Itch, Alagille syndrome, Adult, Pregnancy, NDA, Liver, Recycling, Therapy, Food, Xanthoma, Pharmaceutical industry, Maralixibat, Volixibat, Mirum Pharmaceuticals, MARALIXIBAT, VOLIXIBAT, MIRUM PHARMACEUTICALS

Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases.

Key Points: 
  • Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases.
  • The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021.
  • Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis.
  • Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

Mirum Pharmaceuticals Presents Analyses From Its Rare Liver Disease Programs at the EASL International Liver Congress 2021

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星期三, 六月 23, 2021
Health, Consumer, Women, Clinical trials, Pharmaceutical, Biotechnology, European Association for the Study of the Liver, Hepatology, Maralixibat, Volixibat, Mirum Pharmaceuticals, Inc., MARALIXIBAT, VOLIXIBAT, MIRUM PHARMACEUTICALS, INC.

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the presentation of analyses from its rare liver disease programs during the EASL International Liver Congress.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the presentation of analyses from its rare liver disease programs during the EASL International Liver Congress.
  • In placebo-controlled studies, GI events occurred at similar rates between maralixibat and placebo in the background of ALGS.
  • We hope this in turn will lead to improved outcomes for women with this uncommon and unrelenting liver disease.
  • Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases.

Mirum Pharmaceuticals to Showcase Analyses From Its Rare Liver Disease Programs at The International Liver Congress 2021

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星期三, 六月 16, 2021
Biotechnology, Pharmaceutical, Health, Clinical trials, Medical specialties, Health care, Priority review, Food and Drug Administration, Primary biliary cholangitis, Maralixibat, Volixibat, Mirum Pharmaceuticals, Inc., MARALIXIBAT, VOLIXIBAT, MIRUM PHARMACEUTICALS, INC.

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leader in rare liver disease, today announced its participation in The International Liver Congress taking place virtually June 23-26, 2021.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leader in rare liver disease, today announced its participation in The International Liver Congress taking place virtually June 23-26, 2021.
  • Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases.
  • Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases.
  • The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021.

Mirum Pharmaceuticals Showcases Maralixibat Transplant-Free Survival Data for Progressive Familial Intrahepatic Cholestasis and Long-Term Safety Analyses for Alagille Syndrome during WCPGHAN Annual Meeting

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星期四, 六月 3, 2021
Biotechnology, Health, Science, Pharmaceutical, Research, Medical specialties, Hepatology, Digestive diseases, Clinical medicine, Progressive familial intrahepatic cholestasis, Alagille syndrome, Cholestasis, PFIC, Biliary atresia, Liver, Bile acid, Bile duct, Mirum Pharmaceuticals, Inc., Maralixibat, MIRUM PHARMACEUTICALS, INC., MARALIXIBAT

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) presented analyses from its clinical studies evaluating maralixibat in patients with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis type 2 (PFIC2), two rare liver diseases affecting children.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) presented analyses from its clinical studies evaluating maralixibat in patients with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis type 2 (PFIC2), two rare liver diseases affecting children.
  • These data demonstrated that patients with long-term maralixibat treatment who achieved serum bile acid (sBA) response had five-year transplant-free survival.
  • Mirums lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia.
  • More than 1,600 individuals have received maralixibat, including more than 120 children who have received maralixibat as an investigational treatment for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).

Mirum Pharmaceuticals to Present Maralixibat Data and Host Symposium at the 6th World Congress of Pediatric Gastroenterology, Hepatology and Nutrition (WCPGHAN) Annual Meeting 2021

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星期三, 五月 26, 2021
Health, General Health, Research, Pharmaceutical, Science, Biotechnology, Medical specialties, Digestive diseases, Clinical medicine, Hepatology, Primary biliary cholangitis, Alagille syndrome, Alex Mowat, Maralixibat, Mirum Pharmaceuticals, Inc., MARALIXIBAT, MIRUM PHARMACEUTICALS, INC.

This abstract has been nominated for the prestigious Annual Alex Mowat Prize for best oral presentation in hepatology.

Key Points: 
  • This abstract has been nominated for the prestigious Annual Alex Mowat Prize for best oral presentation in hepatology.
  • Results to be announced during the congress closing ceremony on June 5, 2021 at 1:30 p.m. CEST.
  • Abstract number: H-ePwP-030: Gastrointestinal tolerability of maralixibat in patients with Alagille syndrome: An integrated analysis of short- and long-term treatment.
  • Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases.

Mirum Pharmaceuticals Reports First Quarter 2021 Financial Results and Provides Business Update

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星期四, 五月 6, 2021
Biotechnology, Pharmaceutical, Genetics, Health, Medical specialties, Hepatology, Primary sclerosing cholangitis, Cholestasis, PFIC, Primary biliary cholangitis, Ascending cholangitis, ABCB11, Progressive familial intrahepatic cholestasis, Maralixibat, Volixibat, Mirum Pharmaceuticals, MARALIXIBAT, VOLIXIBAT, MIRUM PHARMACEUTICALS

In a Phase 2 PFIC study , a genetically defined subset of BSEP deficient (PFIC2), patients responded to maralixibat with an increase in transplant-free survival.

Key Points: 
  • In a Phase 2 PFIC study , a genetically defined subset of BSEP deficient (PFIC2), patients responded to maralixibat with an increase in transplant-free survival.
  • The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021.
  • Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis.
  • Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.\n'

Mirum Pharmaceuticals and CANbridge Pharmaceuticals Enter into Exclusive Licensing Agreement to Develop and Commercialize Maralixibat in Greater China for Rare Liver Diseases

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星期四, 四月 29, 2021
Biotechnology, Health, Genetics, Pharmaceutical, Oncology, WuXi Biologics, Health care, Pharmacology, Mirum Pharmaceuticals, Inc., CANbridge Pharmaceuticals Inc., Maralixibat, MIRUM PHARMACEUTICALS, INC., CANBRIDGE PHARMACEUTICALS INC., MARALIXIBAT

b'Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) and CANbridge Pharmaceuticals, Inc., announced today that they have entered into a licensing agreement, pursuant to which CANbridge has agreed to develop and commercialize maralixibat in Greater China (China, Hong Kong, Macau and Taiwan).

Key Points: 
  • b'Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) and CANbridge Pharmaceuticals, Inc., announced today that they have entered into a licensing agreement, pursuant to which CANbridge has agreed to develop and commercialize maralixibat in Greater China (China, Hong Kong, Macau and Taiwan).
  • Mirum has commenced a global Phase 2b maralixibat study (EMBARK) for the treatment of BA.\nUnder the terms of the licensing agreement, CANbridge has obtained the exclusive right to develop and commercialize maralixibat within the Greater China regions for ALGS, PFIC, and BA.
  • CANbridge has a global partnership with WuXi Biologics to develop and commercialize proprietary therapeutics for the treatment of rare genetic diseases.
  • CANbridge also has entered into a strategic collaboration and licensing agreement with LogicBio Therapeutics to develop, manufacture and commercialize gene therapy candidates for treatments for Fabry and Pompe diseases.