[{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"ARYA Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Acquisition","leadProduct":"Migalastat","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Amicus Therapeutics \/ ARYA Sciences","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ ARYA Sciences"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Blackstone Life Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Financing","leadProduct":"Migalastat","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Amicus Therapeutics \/ Blackstone Life Sciences","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Blackstone Life Sciences"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Migalastat","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","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 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":"2020","type":"Inapplicable","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Amicus Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Amicus Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Amicus Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Amicus Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Amicus Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amicus 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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":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2020","type":"Financing","leadProduct":"Cipaglucosidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Amicus 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Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"AT-GTX-501","moa":"Undisclosed","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 \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Amicus Therapeutics \/ Inapplicable"}]

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                          01

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : The U.S. FDA has approved Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules. This two-component therapy is indicated for adults living with late-onset Pompe disease (LOPD) weighing ≥40 kg.

                          Product Name : AT-GAA

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          September 28, 2023

                          Lead Product(s) : Cipaglucosidase Alpha,Miglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : MHRA of the United Kingdom has granted marketing authorizations for Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) for adults living with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency).

                          Product Name : AT-GAA

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          August 15, 2023

                          Lead Product(s) : Cipaglucosidase Alpha,Miglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : Upon approval of enzyme replacement therapy (cipaglucosidase alfa) + opfolda (miglustat), the enzyme stabilizer component of AT-GAA, will be the first two-component therapy available in the EU for the treatment of adults living with late-onset pompe dise...

                          Product Name : AT-GAA

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          June 27, 2023

                          Lead Product(s) : Cipaglucosidase Alpha,Miglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : Upon approval of enzyme replacement therapy (cipaglucosidase alfa) + opfolda (miglustat), the enzyme stabilizer component of AT-GAA, will be the first two-component therapy available in the EU for the treatment of adults living with late-onset pompe dise...

                          Product Name : AT-GAA

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          April 26, 2023

                          Lead Product(s) : Cipaglucosidase Alpha,Miglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          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...

                          Product Name : AT-GAA

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          March 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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                          06

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          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...

                          Product Name : AT-GAA

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          February 13, 2023

                          Lead Product(s) : Cipaglucosidase Alpha,Miglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          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.

                          Product Name : Galafold

                          Product Type : Other Small Molecule

                          Upfront Cash : Undisclosed

                          February 10, 2023

                          Lead Product(s) : Migalastat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Blackstone Life Sciences

                          Deal Size : $30.0 million

                          Deal Type : Financing

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                          08

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          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...

                          Product Name : AT-GAA

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          October 28, 2022

                          Lead Product(s) : Cipaglucosidase Alpha,Miglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          Details : AT-GAA, consists of cipaglucosidase alfa, a unique rhGAA enzyme with optimized carbohydrate structures, particularly bis-phosphorylated mannose-6 phosphate glycans, to enhance uptake into cells, administered in conjunction with miglustat, a stabilizer of...

                          Product Name : AT-GAA

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          October 05, 2022

                          Lead Product(s) : Cipaglucosidase Alpha,Miglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          AACR Annual meeting
                          Not Confirmed
                          AACR Annual meeting
                          Not Confirmed

                          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.

                          Product Name : AT-GAA

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          July 11, 2022

                          Lead Product(s) : Cipaglucosidase Alpha,Miglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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