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[{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum\u2019s Maralixibat Demonstrates Transplant-Free Survival for Pediatric Patients with Progressive Familial Intrahepatic Cholestasis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals Presents Maralixibat Five-Year Transplant-Free Survival Data for Patients With PFIC2 at Digital ILC 2020","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals Initiates Rolling Submission of an NDA for Maralixibat for Cholestatic Pruritus in Patients with Alagille Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Eversana","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Mirum Pharmaceuticals Announces Partnership With EVERSANA to Support Launch and Commercialization of Maralixibat for Alagille Syndrome in the United States","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals to Present Maralixibat Data and Host Symposium at the NASPGHAN Annual Meeting 2020","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals Announces New Data Being Presented in Late-Breaker Oral and Poster Presentations at the Annual Meeting of the AASLD","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals Announces Data Presented During AASLD Highlighting Durable Improvements in Pruritus and Quality of Life in Children with Alagille Syndrome Treated with Maralixibat","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Vivet Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Mirum Pharmaceuticals Ink License Agreement for Vivet\u2019s Gene Therapy Programs Targeting Progressive Familial Intrahepatic Cholestasis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Preclinical"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"CANbridge Pharmaceuticals","pharmaFlowCategory":"D","amount":"$120.0 million","upfrontCash":"$11.0 million","newsHeadline":"Mirum Pharmaceuticals and CANbridge Pharmaceuticals Ink Licensing Agreement for Maralixibat in Greater China for Rare Liver Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals Presents Analyses From Its Rare Liver Disease Programs at the EASL International Liver Congress 2021","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"GC Pharma","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$5.0 million","newsHeadline":"Mirum Pharmaceuticals and GC Pharma Ink Exclusive Licensing Agreement to Develop and Commercialize Maralixibat for Rare Liver Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals Submits European Marketing Authorization Application for Maralixibat in Alagille Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Mirum Pharmaceuticals and Takeda Enter into Exclusive Licensing Agreement to Develop and Commercialize Maralixibat for Rare Pediatric Liver Diseases in Japan","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"U.S. FDA Approves LIVMARLI (maralixibat) as the First and Only Approved Medication for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome One Year of Age and Older","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"September 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"New LIVMARLI\u2122 (Maralixibat) Data to be Showcased at AASLD The Liver Meeting\u00ae 2021","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"November 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"The Lancet Publishes Data From Mirum Pharmaceuticals' ICONIC Pivotal Study of Maralixibat (LIVMARLI) Treatment in Alagille Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"November 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Jefferies LLC","pharmaFlowCategory":"D","amount":"$110.0 million","upfrontCash":"Undisclosed","newsHeadline":"Mirum Pharmaceuticals Sells Rare Pediatric Disease Priority Review Voucher","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"November 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"CANbridge Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CANbridge Pharmaceuticals CAN108 New Drug Application\/Orphan Drug Registration (NDA\/ORD) for Alagille Syndrome Accepted by the Taiwan Food and Drug Administration","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Satiogen","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$2.8 million","newsHeadline":"Mirum Pharmaceuticals Acquires Satiogen Pharmaceuticals","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"May 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals to Showcase LIVMARLI\u00ae (maralixibat) Data at EASL and ESPGHAN Annual Congresses","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"June 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals Presents New Data at The International Liver Congress\u2122 of the European Association for the Study of the Liver (EASL)","therapeuticArea":"Hepatology (Liver, Pancreatic, Gall Bladder)","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Hepatology (Liver, Pancreatic, Gall Bladder)","graph2":"Phase II"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals Highlights Data Demonstrating Impact of LIVMARLI\u00ae (maralixibat) on Patients with Alagille Syndrome at European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"June 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"CANbridge Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"First Patient Dosed in CAN108 (Maralixibat) EMBARK Phase 2 Study in Biliary Atresia in China","therapeuticArea":"Hepatology (Liver, Pancreatic, Gall Bladder)","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Hepatology (Liver, Pancreatic, Gall Bladder)","graph2":"Phase II"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals to Present LIVMARLI\u00ae (maralixibat) Analyses and Host a Scientific Symposium at NASPGHAN 2022 Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals Presents Long-Term LIVMARLI\u00ae (maralixibat) Data and New Alagille Syndrome Caregiver Burden Analysis at NASPGHAN 2022 Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"NeoPharm Co., Ltd.","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals Announces Positive CHMP Opinion for LIVMARLI\u00ae (maralixibat) Oral Solution for Cholestatic Pruritus in Patients with Alagille Syndrome Two Months of Age and Older","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals\u2019 LIVMARLI Data Highlighting Quality of Life and Sleep Improvements in Patients with Alagille Syndrome Published in Journal of Pediatrics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Positive Topline Data Announced from Mirum\u2019s LIVMARLI Phase 3 MARCH Study in Progressive Familial Intrahepatic Cholestasis (PFIC)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"CANbridge Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CANbridge Announces Encouraging Development in Maralixibat\/LIVMARLI in Rare Liver Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals Provides Development Program Updates","therapeuticArea":"Hepatology (Liver, Pancreatic, Gall Bladder)","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Hepatology (Liver, Pancreatic, Gall Bladder)","graph2":"Phase II"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals\u2019 LIVMARLI (maralixibat) Approved by the European Commission for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome Two Months and Older","therapeuticArea":"Dermatology","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"December 2022","url1":"","url2":"","graph1":"Dermatology","graph2":"Approved"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals Submits Supplemental New Drug Application to FDA for LIVMARLI in Patients with Cholestatic Pruritus in Progressive Familial Intrahepatic Cholestasis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"February 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Announces Label Expansion for LIVMARLI in the United States to Include Infants Three Months of Age and Older","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Submits for European Approval of LIVMARLI in Progressive Familial Intrahepatic Cholestasis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals Announces Enrollment Completion in Phase 2b EMBARK Biliary Atresia Study","therapeuticArea":"Hepatology (Liver, Pancreatic, Gall Bladder)","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"May 2023","url1":"","url2":"","graph1":"Hepatology (Liver, Pancreatic, Gall Bladder)","graph2":"Phase II"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Travere Therapeutics","pharmaFlowCategory":"D","amount":"$445.0 million","upfrontCash":"$210.0 million","newsHeadline":"Travere Therapeutics Enters Agreement to Sell its Bile Acid Product Portfolio for the Treatment of Rare Liver Diseases to Mirum Pharmaceuticals","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals Announces Positive Phase 3 RESTORE Study Results Evaluating Chenodal in Patients with Cerebrotendinous Xanthomatosis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"October 2023","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals Announces New PDUFA Date for LIVMARLI for the Treatment of Cholestatic Pruritus in Progressive Familial Intrahepatic Cholestasis","therapeuticArea":"Hepatology (Liver, Pancreatic, Gall Bladder)","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"October 2023","url1":"","url2":"","graph1":"Hepatology (Liver, Pancreatic, Gall Bladder)","graph2":"Phase III"}]

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            Details:

            Livmarli (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor and NMPA has accepted the New Drug Application for the treatment of Cholestatic Pruritus in Progressive Familial Intrahepatic Cholestasis.

            Lead Product(s): Maralixibat

            Therapeutic Area: Hepatology (Liver, Pancreatic, Gall Bladder) Product Name: Livmarli

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 17, 2023

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            Chenodiol (chenodiol) tablets is a naturally occurring bile acid, which is investigated for the treatment of adult patients with cerebrotendinous xanthomatosis.

            Lead Product(s): Chenodeoxycholic Acid

            Therapeutic Area: Rare Diseases and Disorders Product Name: Chenodal

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 02, 2023

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            Under the agreement, Mirum will purchase Travere’s bile acid product portfolio including, Cholbam, indicated for the treatment of bile acid synthesis disorders and Chenodal, indicated for the treatment of radiolucent stones in the gallbladder.

            Lead Product(s): Cholic Acid

            Therapeutic Area: Genetic Disease Product Name: Cholbam

            Highest Development Status: Approved Product Type: Small molecule

            Recipient: Travere Therapeutics

            Deal Size: $445.0 million Upfront Cash: $210.0 million

            Deal Type: Divestment July 17, 2023

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            Livmarli® (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor being developed for the treatment of biliary atresia in infant who have previously undergone a hepatoportoenterostomy.

            Lead Product(s): Maralixibat

            Therapeutic Area: Hepatology (Liver, Pancreatic, Gall Bladder) Product Name: Livmarli

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 11, 2023

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            Livmarli® (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. FDA for the treatment of cholestatic pruritus in patients with alagille syndrome one year of age and older.

            Lead Product(s): Maralixibat

            Therapeutic Area: Genetic Disease Product Name: Livmarli

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 05, 2023

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            Details:

            Livmarli™ (maralixibat) is a reversible inhibitor of the ileal bile acid transporter (IBAT). It decreases the reabsorption of bile acids (primarily the salt forms) from the terminal ileum.

            Lead Product(s): Maralixibat

            Therapeutic Area: Genetic Disease Product Name: Livmarli

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 14, 2023

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            Details:

            Livmarli® (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. FDA for the treatment of cholestatic pruritus in patients with alagille syndrome one year of age and older.

            Lead Product(s): Maralixibat

            Therapeutic Area: Genetic Disease Product Name: Livmarli

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 14, 2023

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            Details:

            LIVMARLI® (maralixibat) oral solution which is approved in the U.S. for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older, and in Europe for the same indication in patients two months of age and older.

            Lead Product(s): Maralixibat

            Therapeutic Area: Dermatology Product Name: Livmarli

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 13, 2022

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            Details:

            SHP626 (Volixibat) is an oral, minimally absorbed agent designed to selectively inhibit IBAT for treatment of adult cholestatic diseases by blocking recycling of bile acids, through inhibition of IBAT, thereby reducing bile acids systemically and in the liver.

            Lead Product(s): Volixibat

            Therapeutic Area: Hepatology (Liver, Pancreatic, Gall Bladder) Product Name: SHP626

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 29, 2022

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            Details:

            Livmarli (maralixibat) is a minimally absorbed ileal bile acid transporter (IBAT) inhibitor that blocks the enterohepatic circulation of bile acids, reduces bile acid levels in the liver and serum, reduces the resultant liver injury and relieves pruritus.

            Lead Product(s): Maralixibat

            Therapeutic Area: Genetic Disease Product Name: Livmarli

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: CANbridge Pharmaceuticals

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 26, 2022

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