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    [{"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":"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":"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"},{"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 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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 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Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ 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":"Actelion Pharmaceuticals Ltd","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":"Actelion Pharmaceuticals Ltd","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Actelion Pharmaceuticals Ltd \/ Beyond Batten Disease Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Actelion Pharmaceuticals Ltd \/ Beyond Batten Disease Foundation"},{"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 Citrate","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":"Orphazyme","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Other Small Molecule","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":"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 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":"Oral Capsule","sponsorNew":"Orphazyme \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Inapplicable"},{"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 Citrate","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":"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 Citrate","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":"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 Citrate","moa":"||TFEB","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"}]

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                            01

                            Breckenridge Pharmaceutical

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                            Lead Product(s) : Miglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Breckenridge Announces Approval of its ANDA for Miglustat Capsules (generic for Zavesca®)

                            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

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                            02

                            Zevra Therapeutics

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                            Lead Product(s) : Arimoclomol Citrate,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : $150.0 million

                            Deal Type : Agreement

                            DEALS_DEV arrow-down

                            Zevra Sells Rare Disease Review Voucher For $150 Million

                            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

                            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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                            03

                            Zevra Therapeutics

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                            Lead Product(s) : Arimoclomol Citrate,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : $150.0 million

                            Deal Type : Agreement

                            DEALS_DEV arrow-down

                            Zevra to Sell Rare Pediatric Disease Voucher for $150M

                            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 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

                            Zevra Therapeutics

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                            Zevra Therapeutics

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                            Lead Product(s) : Arimoclomol Citrate,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Zevra Launches MIPLYFFA™ in US for Niemann-Pick Disease Type C Treatment

                            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 Citrate,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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                            05

                            Zevra Therapeutics

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                            Zevra Therapeutics

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                            Lead Product(s) : Arimoclomol Citrate,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            FDA Approves First Treatment for Niemann-Pick Disease, Type C

                            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 Citrate,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Orsini Specialty Pharmacy

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                            Orsini Specialty Pharmacy

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                            Lead Product(s) : Cipaglucosidase Alpha,Miglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Amicus Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Agreement

                            DEALS_DEV arrow-down

                            Orsini Specialty Pharmacy Partners with Amicus Therapeutics to Dispense New Therapy for the Treatment of Late-...

                            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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                            07

                            Amicus Therapeutics

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                            Lead Product(s) : Cipaglucosidase Alpha,Miglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Amicus Therapeutics Announces FDA Approval and Launch of New Treatment for Pompe Disease

                            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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                            08

                            Theranexus

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                            Theranexus

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                            Lead Product(s) : Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Beyond Batten Disease Foundation

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Theranexus and BBDF Win FDA Approval on Efficacy Endpoints for the Phase III Trial to Evaluate Batten-1 in CLN...

                            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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                            09

                            Amicus Therapeutics

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                            Amicus Therapeutics

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                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Cipaglucosidase Alpha,Miglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Amicus Therapeutics Announces Approval and Launch of New Pompe Disease Therapy in the United Kingdom

                            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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                            10

                            Amicus Therapeutics

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                            Amicus Therapeutics

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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Cipaglucosidase Alpha,Miglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Amicus Therapeutics Announces Approval and Launch of New Pompe Disease Therapy in the European Union

                            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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