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    [{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2020","type":"Financing","leadProduct":"AT-GAA","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amicus Therapeutics","amount2":0.40000000000000002,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.40000000000000002,"dosageForm":"","sponsorNew":"Amicus Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Amicus Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"AT-GTX-501","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Infusion","sponsorNew":"Amicus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Amicus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"AT-GTX-501","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Infusion","sponsorNew":"Amicus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Amicus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","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":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Cipaglucosidase alfa","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Amicus 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Sciences"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","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":"","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Cipaglucosidase Alfa","moa":"Lysosomal alpha-glucosidase","graph1":"Genetic 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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":"","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 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Disease","amount2New":0.029999999999999999,"dosageForm":"Capsule","sponsorNew":"Amicus Therapeutics \/ Blackstone Life Sciences","highestDevelopmentStatusID":"12","companyTruncated":"Amicus Therapeutics \/ Blackstone Life Sciences"}]

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                            01

                            Amicus Therapeutics

                            U.S.A arrow
                            euroPLX 86 Munich
                            Not Confirmed

                            Amicus Therapeutics

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                            euroPLX 86 Munich
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                            Lead Product(s) : Migalastat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved

                            Sponsor : Blackstone Life Sciences

                            Deal Size : $30.0 million

                            Deal Type : Financing

                            DEALS_DEV arrow-down

                            Amicus Therapeutics and Blackstone Enter into $430 Million Strategic Financing Collaboration

                            Details : The proceeds will be used to fund the ongoing operations of the company, including Galafold (migalastat), a small molecule and the first and only oral therapy for adults with Fabry disease and an amenable GLA variant.

                            Brand Name : Galafold

                            Molecule Type : Small molecule

                            Upfront Cash : Undisclosed

                            October 02, 2023

                            Lead Product(s) : Migalastat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved

                            Sponsor : Blackstone Life Sciences

                            Deal Size : $30.0 million

                            Deal Type : Financing

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                            02

                            Amicus Therapeutics

                            U.S.A arrow
                            euroPLX 86 Munich
                            Not Confirmed

                            Amicus Therapeutics

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                            euroPLX 86 Munich
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                            Lead Product(s) : Cipaglucosidase Alfa,Miglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved

                            Recipient : Orsini Specialty Pharmacy

                            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

                            Recipient : Orsini Specialty Pharmacy

                            Deal Size : Undisclosed

                            Deal Type : Agreement

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                            03

                            Amicus Therapeutics

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                            euroPLX 86 Munich
                            Not Confirmed

                            Amicus Therapeutics

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                            euroPLX 86 Munich
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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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                            04

                            Amicus Therapeutics

                            U.S.A arrow
                            euroPLX 86 Munich
                            Not Confirmed

                            Amicus Therapeutics

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                            euroPLX 86 Munich
                            Not Confirmed
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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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                            05

                            Amicus Therapeutics

                            U.S.A arrow
                            euroPLX 86 Munich
                            Not Confirmed

                            Amicus Therapeutics

                            U.S.A arrow
                            euroPLX 86 Munich
                            Not Confirmed
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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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                            06

                            Amicus Therapeutics

                            U.S.A arrow
                            euroPLX 86 Munich
                            Not Confirmed

                            Amicus Therapeutics

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                            euroPLX 86 Munich
                            Not Confirmed
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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

                            DEALS_DEV arrow-down

                            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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                            07

                            Amicus Therapeutics

                            U.S.A arrow
                            euroPLX 86 Munich
                            Not Confirmed

                            Amicus Therapeutics

                            U.S.A arrow
                            euroPLX 86 Munich
                            Not Confirmed
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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 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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                            08

                            Amicus Therapeutics

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                            euroPLX 86 Munich
                            Not Confirmed

                            Amicus Therapeutics

                            U.S.A arrow
                            euroPLX 86 Munich
                            Not Confirmed
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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

                            DEALS_DEV arrow-down

                            Amicus Therapeutics Announces Presentations and Posters at the 19th Annual WORLDSymposium™ 2023

                            Details : AT-GAA (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 AT...

                            Brand Name : AT-GAA

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            February 13, 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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                            09

                            Amicus Therapeutics

                            U.S.A arrow
                            euroPLX 86 Munich
                            Not Confirmed

                            Amicus Therapeutics

                            U.S.A arrow
                            euroPLX 86 Munich
                            Not Confirmed
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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

                            DEALS_DEV arrow-down

                            Amicus Therapeutics Provides EU Regulatory Update for AT-GAA

                            Details : AT-GAA is an investigational two-component therapy that consists of cipaglucosidase alfa (ATB200), a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures.

                            Brand Name : AT-GAA

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            November 07, 2022

                            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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                            10

                            Amicus Therapeutics

                            U.S.A arrow
                            euroPLX 86 Munich
                            Not Confirmed

                            Amicus Therapeutics

                            U.S.A arrow
                            euroPLX 86 Munich
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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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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                            U.S. FDA Defers Action on Filing for AT-GAA in Late-onset Pompe Disease

                            Details : AT-GAA is an investigational two-component therapy that consists of cipaglucosidase alfa, a unique rhGAA enzyme with optimized carbohydrate structures, particularly bis-M6P glycans, to enhance uptake into cells, administered in conjunction with miglustat...

                            Brand Name : AT-GAA

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            October 28, 2022

                            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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