[{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orsini Specialty Pharmacy","sponsor":"Amicus Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2023","type":"Agreement","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Orsini Specialty Pharmacy","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orsini Specialty Pharmacy \/ Amicus Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Orsini Specialty Pharmacy \/ Amicus Therapeutics"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Beyond Batten Disease Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Miscellaneous","year":"2021","type":"Collaboration","leadProduct":"Miglustat","moa":"||Ceramide glucosyltransferase","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Beyond Batten Disease Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Beyond Batten Disease Foundation"},{"orgOrder":0,"company":"Theranexus","sponsor":"Beyond Batten Disease Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Miglustat","moa":"Ceramide glucosyltransferase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Theranexus","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Theranexus \/ Beyond Batten Disease Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Theranexus \/ Beyond Batten Disease Foundation"},{"orgOrder":0,"company":"Theranexus","sponsor":"Beyond Batten Disease Foundation","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Miglustat","moa":"Ceramide glucosyltransferase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Theranexus","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Theranexus \/ Beyond Batten Disease Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Theranexus \/ Beyond Batten Disease Foundation"},{"orgOrder":0,"company":"Beyond Batten Disease Foundation","sponsor":"Theranexus","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Miglustat","moa":"Ceramide glucosyltransferase","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Beyond Batten Disease Foundation","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Beyond Batten Disease Foundation \/ Theranexus","highestDevelopmentStatusID":"7","companyTruncated":"Beyond Batten Disease Foundation \/ Theranexus"},{"orgOrder":0,"company":"Breckenridge Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Miglustat","moa":"Ceramide glucosyltransferase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Breckenridge Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Breckenridge Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Breckenridge Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Arimoclomol Citrate","moa":"||HSF-1 pathway","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Undisclosed"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Arimoclomol Citrate","moa":"||HSF-1 pathway","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Undisclosed"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Arimoclomol Citrate","moa":"||HSF-1 pathway","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Zevra Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Zevra Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Agreement","leadProduct":"Arimoclomol Citrate","moa":"||HSF-1 pathway","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Zevra Therapeutics","amount2":0.14999999999999999,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0.14999999999999999,"dosageForm":"Capsule","sponsorNew":"Zevra Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Zevra Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Agreement","leadProduct":"Arimoclomol Citrate","moa":"||HSF-1 pathway","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Zevra Therapeutics","amount2":0.14999999999999999,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0.14999999999999999,"dosageForm":"Capsule","sponsorNew":"Zevra Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Zevra Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Arimoclomol Citrate","moa":"||HSF-1 pathway","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Zevra Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Zevra Therapeutics \/ Undisclosed"}]

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                          01

                          Details : Miglustat Capsules (generic for Zavesca), 100mg strength, is an inhibitor of the enzyme glucosylceramide synthase, which is a glucosyl transferase enzyme responsible for first step in synthesis of most glycosphingolipids.

                          Product Name : Miglustat-Generic

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          April 21, 2022

                          Lead Product(s) : Miglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Breckenridge Pharmaceutical

                          02

                          PackExpo
                          Not Confirmed
                          PackExpo
                          Not Confirmed

                          Details : Batten-1 is a novel and exclusive proprietary drug containing the active ingredient miglustat. The MOA of this substance blocks the accumulation of glycosphingolipids and neuroinflammation.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          May 13, 2025

                          Lead Product(s) : Miglustat

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Beyond Batten Disease Foundation

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          PackExpo
                          Not Confirmed
                          PackExpo
                          Not Confirmed

                          Details : Zevra sold it's Rare Pediatric Disease PRV for FDA approved of Miplyffa (arimoclomol). It is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC. C

                          Product Name : Miplyffa

                          Product Type : Miscellaneous

                          Upfront Cash : Undisclosed

                          April 07, 2025

                          Lead Product(s) : Arimoclomol Citrate,Miglustat

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : $150.0 million

                          Deal Type : Agreement

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                          04

                          PackExpo
                          Not Confirmed
                          PackExpo
                          Not Confirmed

                          Details : Zevra sold it's Rare Pediatric Disease PRV for FDA approved of Miplyffa (arimoclomol). It is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC. C

                          Product Name : Miplyffa

                          Product Type : Miscellaneous

                          Upfront Cash : Undisclosed

                          February 27, 2025

                          Lead Product(s) : Arimoclomol Citrate,Miglustat

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : $150.0 million

                          Deal Type : Agreement

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                          05

                          PackExpo
                          Not Confirmed
                          PackExpo
                          Not Confirmed

                          Details : Miplyffa (arimoclomol) is a first in class oral HSF1 activator approved for Niemann-Pick disease type C in combination with miglustat.

                          Product Name : Miplyffa

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          November 21, 2024

                          Lead Product(s) : Arimoclomol Citrate,Miglustat

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          PackExpo
                          Not Confirmed
                          PackExpo
                          Not Confirmed

                          Details : Miplyffa (Arimoclomol) is indicated for use in combination with miglustat for the treatment of Niemann-Pick disease type C in adult and pediatric patients 2 years of age and older.

                          Product Name : Miplyffa

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          September 20, 2024

                          Lead Product(s) : Arimoclomol Citrate,Miglustat

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          PackExpo
                          Not Confirmed
                          PackExpo
                          Not Confirmed

                          Details : Under the agreement, Orsini will dispense Pombiliti™ (cipaglucosidase alfa-atga), a hydrolytic lysosomal glycogen-specific enzyme, and Opfolda™ (miglustat), a two-component treatment approved for certain patients with late-onset Pompe disease.

                          Product Name : Pombiliti

                          Product Type : Enzyme

                          Upfront Cash : Undisclosed

                          September 28, 2023

                          Lead Product(s) : Cipaglucosidase Alpha,Miglustat

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Amicus Therapeutics

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          08

                          PackExpo
                          Not Confirmed
                          PackExpo
                          Not Confirmed

                          Details : The U.S. FDA has approved Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules. This two-component therapy is indicated for adults living with late-onset Pompe disease (LOPD) weighing ≥40 kg.

                          Product Name : AT-GAA

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          September 28, 2023

                          Lead Product(s) : Cipaglucosidase Alpha,Miglustat

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          PackExpo
                          Not Confirmed
                          PackExpo
                          Not Confirmed

                          Details : MHRA of the United Kingdom has granted marketing authorizations for Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) for adults living with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency).

                          Product Name : AT-GAA

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          August 15, 2023

                          Lead Product(s) : Cipaglucosidase Alpha,Miglustat

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          PackExpo
                          Not Confirmed
                          PackExpo
                          Not Confirmed

                          Details : Upon approval of enzyme replacement therapy (cipaglucosidase alfa) + opfolda (miglustat), the enzyme stabilizer component of AT-GAA, will be the first two-component therapy available in the EU for the treatment of adults living with late-onset pompe dise...

                          Product Name : AT-GAA

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          June 27, 2023

                          Lead Product(s) : Cipaglucosidase Alpha,Miglustat

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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