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    [{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Cipaglucosidase alfa","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Amicus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amicus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase 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Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Cipaglucosidase alfa","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Amicus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Amicus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Actelion Pharmaceuticals Ltd","sponsor":"Beyond Batten Disease Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Small molecule","year":"2021","type":"Collaboration","leadProduct":"Miglustat","moa":"GCS","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Actelion Pharmaceuticals Ltd","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Actelion Pharmaceuticals Ltd \/ Beyond Batten Disease Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Actelion Pharmaceuticals Ltd \/ Beyond Batten Disease Foundation"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Cipaglucosidase Alfa","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Amicus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amicus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Cipaglucosidase Alfa","moa":"Lysosomal alpha-glucosidase","graph1":"Genetic Disease","graph2":"Approved","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Amicus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amicus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Breckenridge Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Miglustat","moa":"Glucosylceramide synthase","graph1":"Genetic Disease","graph2":"Approved","graph3":"Breckenridge Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Breckenridge Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Breckenridge Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Cipaglucosidase Alfa","moa":"Lysosomal alpha-glucosidase","graph1":"Genetic Disease","graph2":"Approved","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Amicus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amicus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Cipaglucosidase Alfa","moa":"Lysosomal alpha-glucosidase","graph1":"Genetic Disease","graph2":"Approved","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amicus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Cipaglucosidase Alfa","moa":"Lysosomal alpha-glucosidase","graph1":"Genetic Disease","graph2":"Approved","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amicus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Cipaglucosidase Alfa","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amicus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Cipaglucosidase Alfa","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Amicus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amicus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Cipaglucosidase Alfa","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Amicus 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Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amicus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Orsini Specialty Pharmacy","sponsor":"Amicus Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2023","type":"Agreement","leadProduct":"Cipaglucosidase Alfa","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Orsini Specialty Pharmacy","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Orsini Specialty Pharmacy \/ Amicus Therapeutics","highestDevelopmentStatusID":"12","companyTruncated":"Orsini Specialty Pharmacy \/ Amicus Therapeutics"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Cipaglucosidase Alfa","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amicus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Technology","graph2":"Approved","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Technology","amount2New":0.10000000000000001,"dosageForm":"","sponsorNew":"Zevra Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Zevra Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Technology","graph2":"Approved","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Technology","amount2New":0.10000000000000001,"dosageForm":"","sponsorNew":"Zevra Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Zevra Therapeutics \/ Not Applicable"}]

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                            01

                            Breckenridge Pharmaceutical

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                            Lead Product(s) : Miglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            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.

                            Brand Name : Miglustat-Generic

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            April 21, 2022

                            Lead Product(s) : Miglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            Breckenridge Pharmaceutical

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                            02

                            Zevra Therapeutics

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                            Zevra Therapeutics

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                            Lead Product(s) : Arimoclomol,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            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.

                            Brand Name : Miplyffa

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            November 21, 2024

                            Lead Product(s) : Arimoclomol,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            03

                            Zevra Therapeutics

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                            Antibody Engineering
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                            Zevra Therapeutics

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                            Lead Product(s) : Arimoclomol,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            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.

                            Brand Name : Miplyffa

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            September 20, 2024

                            Lead Product(s) : Arimoclomol,Miglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            04

                            Orsini Specialty Pharmacy

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                            Antibody Engineering
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                            Orsini Specialty Pharmacy

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                            Lead Product(s) : Cipaglucosidase Alfa,Miglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved

                            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.

                            Brand Name : Pombiliti

                            Molecule Type : Large molecule

                            Upfront Cash : Undisclosed

                            September 28, 2023

                            Lead Product(s) : Cipaglucosidase Alfa,Miglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved

                            Sponsor : Amicus Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Agreement

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                            05

                            Amicus Therapeutics

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                            Antibody Engineering
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                            Amicus Therapeutics

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                            Lead Product(s) : Cipaglucosidase Alfa,Miglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            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.

                            Brand Name : Pombiliti

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            September 28, 2023

                            Lead Product(s) : Cipaglucosidase Alfa,Miglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            06

                            Amicus Therapeutics

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                            Amicus Therapeutics

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                            Lead Product(s) : Cipaglucosidase Alfa,Miglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            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).

                            Brand Name : Pombiliti

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            August 15, 2023

                            Lead Product(s) : Cipaglucosidase Alfa,Miglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            07

                            Amicus Therapeutics

                            U.S.A arrow
                            Antibody Engineering
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                            Amicus Therapeutics

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                            Lead Product(s) : Cipaglucosidase Alfa,Miglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            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...

                            Brand Name : Pombiliti

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            June 27, 2023

                            Lead Product(s) : Cipaglucosidase Alfa,Miglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            08

                            Theranexus

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                            Theranexus

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                            Lead Product(s) : Miglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Beyond Batten Disease Foundation

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            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...

                            Brand Name : Batten-1

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            May 09, 2023

                            Lead Product(s) : Miglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Beyond Batten Disease Foundation

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            09

                            Amicus Therapeutics

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                            Antibody Engineering
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                            Amicus Therapeutics

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                            Lead Product(s) : Cipaglucosidase Alfa,Miglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Amicus Therapeutics Receives Positive CHMP Opinion for Opfolda® (miglustat) for Late-Onset Pompe Disease

                            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...

                            Brand Name : Pombiliti

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            April 26, 2023

                            Lead Product(s) : Cipaglucosidase Alfa,Miglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Lead Product(s) : Cipaglucosidase Alfa,Miglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Amicus Therapeutics Announces European Commission Approval for Pombiliti™ in Patients with Late-Onset Pompe ...

                            Details : Pombiliti (cipaglucosidase alfa) is administered with miglustat for the treatment of pompe disease. AT-GAA is a recombinant human acid alpha-glucosidase enzyme with optimized carbohydrate structures to enhance uptake into cells. Miglustat is a stabilizes...

                            Brand Name : Pombiliti

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            March 27, 2023

                            Lead Product(s) : Cipaglucosidase Alfa,Miglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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