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    [{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Fitusiran","moa":"Antithrombin III","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Fitusiran","moa":"Antithrombin III","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Alpha Blue Ocean","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2021","type":"Financing","leadProduct":"Baclofen","moa":"||PMP22 overexpression","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Alpha Blue Ocean","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Alpha Blue Ocean"},{"orgOrder":0,"company":"Pharnext","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Baclofen","moa":"||PMP22 overexpression","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Inapplicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Baclofen","moa":"||PMP22 overexpression","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Inapplicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Baclofen","moa":"||PMP22 overexpression","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Inapplicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Baclofen","moa":"||PMP22 overexpression","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Inapplicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Baclofen","moa":"||PMP22 overexpression","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Inapplicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Baclofen","moa":"||PMP22 overexpression","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Inapplicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Baclofen","moa":"||PMP22 overexpression","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Inapplicable"},{"orgOrder":0,"company":"Pharnext","sponsor":"Neovacs","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2022","type":"Financing","leadProduct":"Baclofen","moa":"||PMP22 overexpression","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Neovacs","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Neovacs"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Givosiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Givosiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Givosiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Givosiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Inventiva Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Odiparcil","moa":"Glycosaminoglycans","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Inventiva Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Inventiva Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Inventiva Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Inventiva Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Odiparcil","moa":"Glycosaminoglycans","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Inventiva Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Inventiva Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Inventiva Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Inventiva Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Odiparcil","moa":"Glycosaminoglycans","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Inventiva Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Inventiva Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Inventiva Pharma \/ Inapplicable"},{"orgOrder":0,"company":"GenSight Biologics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"Lenadogene Nolparvovec","moa":"MTND4","graph1":"Genetic Disease","graph2":"Phase III","graph3":"GenSight Biologics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"GenSight Biologics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"GenSight Biologics \/ Inapplicable"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Sanofi

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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Fitusiran

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Two Fitusiran Phase 3 Studies Published in The Lancet and The Lancet Haematology Highlight Potential to Addres...

                            Details : SAR-439774 (fitusiran) is an investigational, subcutaneously administered small interference RNA therapeutic in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors.

                            Product Name : SAR439774

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            April 04, 2023

                            Lead Product(s) : Fitusiran

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Sanofi

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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            NEJM Publishes Once-Weekly Efanesoctocog Alfa Phase 3 Data Demonstrating Its Potential to Transform the Treatm...

                            Details : BIVV001 (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for most of the week, extending bleed protection in a once-weekly dose for people with haemophili...

                            Product Name : BIVV001

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            January 25, 2023

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Sanofi

                            France
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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Sanofi to present new clinical data reinforcing novel therapies across rare blood disorders at ASH 2022

                            Details : BIVV001 (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy that is designed to extend protection from bleeds with once-weekly prophylactic dosing for people with hemophilia A.

                            Product Name : BIVV001

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            November 30, 2022

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Sanofi

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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Fitusiran

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Fitusiran Prophylaxis Reduced Bleeds by 61% in People With Hemophilia a or B, With or Without Inhibitors, Comp...

                            Details : SAR-439774 (fitusiran) is an investigational, subcutaneously administered small interference RNA therapeutic in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors.

                            Product Name : SAR439774

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            October 07, 2022

                            Lead Product(s) : Fitusiran

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Sanofi

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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Pivotal Data Demonstrate Once-weekly Efanesoctocog Alfa Provides Superior Bleed Protection Compared to Prior F...

                            Details : Investigational once-weekly BIVV001 (efanesoctocog alfa) prophylaxis met the primary efficacy endpoint providing clinically meaningful bleed protection for people with severe hemophilia A.

                            Product Name : BIVV001

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            October 07, 2022

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Sanofi

                            France
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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Efanesoctocog Alfa Met Primary and Key Secondary Endpoints in Pivotal Study in Hemophilia a, Demonstrating Sup...

                            Details : The study met the primary endpoint, showing a clinically meaningful prevention of bleeds in people with severe hemophilia A receiving weekly prophylaxis with BIVV001 (efanesoctocog alfa) over a period of 52 weeks.

                            Product Name : BIVV001

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            September 03, 2022

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Sanofi

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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            FDA Grants Efanesoctocog Alfa Breakthrough Therapy Designation for Hemophilia A

                            Details : BIVV001 (efanesoctocog alfa) is a novel and investigational factor VIII therapy designed to provide normal to near-normal factor activity levels for the majority of the week in a once-weekly prophylactic treatment regimen.

                            Product Name : BIVV001

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            January 06, 2022

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Sanofi

                            France
                            ChemOutsourcing
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                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Givosiran

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Data From Two Phase 3 Studies Demonstrating Fitusiran Significantly Reduced Bleeds in People with Hemophilia A...

                            Details : Data from phase 3 studies achieved primary and secondary endpoints for ALN-AT3SC (Fitusiran) prophylaxis demonstrated significant and clinically meaningful improvements in bleed protection across all study populations.

                            Product Name : ALN-AT3SC

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            December 14, 2021

                            Lead Product(s) : Givosiran

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Sanofi

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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Sanofi's Efanesoctocog Alfa Granted FDA Fast Track Designation for treatment of hemophilia A

                            Details : Efanesoctocog alfa, previously known as BIVV001, is an investigational factor VIII replacement therapy that has the potential to transform therapy and provide high sustained factor activity levels for people with hemophilia A.

                            Product Name : BIVV001

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            February 18, 2021

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Sanofi

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                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            FDA Grants Priority Review for Avalglucosidase Alfa, a Potential New Therapy for Pompe Disease

                            Details : In patients with late-onset Pompe disease (LOPD), 5.5 years of safety and exploratory efficacy results showed avalglucosidase alfa was well-tolerated and stabilized pulmonary and motor function.

                            Product Name : Nexviazyme

                            Product Type : Other Large Molecule

                            Upfront Cash : Inapplicable

                            November 18, 2020

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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