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    [{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","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":"U.S.A","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":"U.S.A","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":"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":"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":"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":"Amicus Therapeutics","sponsor":"ARYA Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2021","type":"Acquisition","leadProduct":"Migalastat","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved","graph3":"Amicus Therapeutics","amount2":0.59999999999999998,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.59999999999999998,"dosageForm":"Capsule","sponsorNew":"Amicus Therapeutics \/ ARYA Sciences","highestDevelopmentStatusID":"12","companyTruncated":"Amicus Therapeutics \/ ARYA Sciences"},{"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":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Migalastat","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","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":"","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 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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":"Infusion","sponsorNew":"Amicus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amicus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Orsini Specialty Pharmacy","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":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Amicus Therapeutics \/ Amicus Therapeutics","highestDevelopmentStatusID":"12","companyTruncated":"Amicus Therapeutics \/ Amicus Therapeutics"},{"orgOrder":0,"company":"Amicus 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Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic 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

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                            Amicus Therapeutics

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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

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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

                            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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                            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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                            04

                            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 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

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                            Antibody Engineering
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                            Amicus Therapeutics

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                            Antibody Engineering
                            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

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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

                            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

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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 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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                            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

                            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

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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

                            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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                            Amicus Therapeutics

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                            Antibody Engineering
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                            Amicus Therapeutics

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                            Antibody Engineering
                            Not Confirmed
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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            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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