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

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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 : Other Small Molecule

                          Upfront Cash : Inapplicable

                          April 21, 2022

                          Lead Product(s) : Miglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Breckenridge Pharmaceutical

                          02

                          BePharma
                          Not Confirmed
                          BePharma
                          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 : Other Small Molecule

                          Upfront Cash : Undisclosed

                          February 27, 2025

                          Lead Product(s) : Arimoclomol,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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                          03

                          BePharma
                          Not Confirmed
                          BePharma
                          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 : Other Small Molecule

                          Upfront Cash : Inapplicable

                          November 21, 2024

                          Lead Product(s) : Arimoclomol,Miglustat

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          BePharma
                          Not Confirmed
                          BePharma
                          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 : Other Small Molecule

                          Upfront Cash : Inapplicable

                          September 20, 2024

                          Lead Product(s) : Arimoclomol,Miglustat

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          BePharma
                          Not Confirmed
                          BePharma
                          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 : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Amicus Therapeutics

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          06

                          BePharma
                          Not Confirmed
                          BePharma
                          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 : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          BePharma
                          Not Confirmed
                          BePharma
                          Not Confirmed

                          Details : Batten-1 is a novel and exclusive proprietary drug containing the active ingredient miglustat. The mechanism of action of this substance blocks the accumulation of glycosphingolipids and neuroinflammation. It is being investigated for CLN3 batten disease...

                          Product Name : Batten-1

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          September 05, 2023

                          Lead Product(s) : Miglustat

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Beyond Batten Disease Foundation

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          BePharma
                          Not Confirmed
                          BePharma
                          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 : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          BePharma
                          Not Confirmed
                          BePharma
                          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 : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          BePharma
                          Not Confirmed
                          BePharma
                          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

                          April 26, 2023

                          Lead Product(s) : Cipaglucosidase Alpha,Miglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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