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[{"orgOrder":0,"company":"LFB","sponsor":"HEMA Biologics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"LFB Announces FDA Approval of SEVENFACT for the Treatment of Adults and Adolescents with Hemophilia A or B with Inhibitors","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"LFB"},{"orgOrder":0,"company":"Ipsen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Health Canada Approves Ipsen\u2019s Sohonos (palovarotene capsules) as the First Approved Treatment for Fibrodysplasia Ossificans Progressiva","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Small molecule","productStatus":"Approved","date":"January 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Ipsen"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Nexviazyme\u00ae (Avalglucosidase Alfa) Shows Sustained Improvements in Respiratory Function and Mobility in Patients With Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"February 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Orphalan","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphalan Announces FD Approval of Cuvrior\u2122 for the Treatment of Adult Patients With Stable Wilson\u2019s Disease Who Are De-coppered and Tolerant to Penicillamine","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Small molecule","productStatus":"Approved","date":"May 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Orphalan"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Approves Nexviazyme\u00ae (avalglucosidase alfa-ngpt), an Important New Treatment Option for Late-Onset Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"August 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sanofi Announces Results of CHMP Re-Examination of the New Active Substance status for Avalglucosidase alfa, a Potential New Standard of Care for The Treatment of Pompe disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"November 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Orphalan","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphalan Announces Data From Secondary Analyses of the CHELATE Trial for Maintenance Patients With Wilson\u2019s Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Small molecule","productStatus":"Approved","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Orphalan"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not 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molecule","graph2":"Ipsen"},{"orgOrder":0,"company":"LFB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CHMP Recommends Cevenfacta to Control Bleeding Episodes in Patients With Congenital Haemophilia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"May 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"LFB"},{"orgOrder":0,"company":"LFB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"LFB Announces the Approval of CEVENFACTA\u00ae (eptacog beta) in the European Union","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Large 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Disease","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"February 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Ipsen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ipsen Receives New FDA PDUFA Date for Investigational Palovarotene for The Treatment of People With FOP","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Small molecule","productStatus":"Approved","date":"March 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Ipsen"},{"orgOrder":0,"company":"Orphalan","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphalan Announces US Commercial Launch of Cuvrior\u2122 for the Treatment of Wilson Disease","therapeuticArea":"Genetic 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            Details:

            Altuviiio (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels, extending bleed protection in a once-weekly dose for people with haemophilia A.

            Lead Product(s): Efanesoctocog Alfa

            Therapeutic Area: Genetic Disease Product Name: Altuviiio

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 25, 2023

            Sanofi Company Banner

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            Cerdelga (eliglustat tartrate) is a prescription medicine used for the long-term treatment of Gaucher disease type 1 (GD1) in adults who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs).

            Lead Product(s): Eliglustat

            Therapeutic Area: Genetic Disease Product Name: Cerdelga

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 01, 2023

            Sanofi Company Banner

            TMF Summit 2024

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            Details:

            Nexviazyme (avalglucosidase alfa) is an enzyme replacement therapy (ERT) designed to target the mannose-6-phosphate (M6P) receptor, the key pathway for uptake and transport of ERT. It is being developed for treatment of people living with pompe disease.

            Lead Product(s): Avalglucosidase Alfa

            Therapeutic Area: Genetic Disease Product Name: Nexviazyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 24, 2023

            Sanofi Company Banner

            TMF Summit 2024

            Not Confirmed

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            Details:

            Altuviiio (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 haemophilia A.

            Lead Product(s): Efanesoctocog Alfa

            Therapeutic Area: Genetic Disease Product Name: Altuviiio

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 23, 2023

            Sanofi Company Banner

            TMF Summit 2024

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            Details:

            Nexviadyme (avalglucosidase alfa), is the first and only newly approved medicine for Pompe disease in Europe since 2006, when the European Commission authorized the marketing of alglucosidase alfa, branded Myozyme®.

            Lead Product(s): Avalglucosidase Alfa

            Therapeutic Area: Genetic Disease Product Name: Nexviadyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 28, 2022

            Sanofi Company Banner

            TMF Summit 2024

            Not Confirmed

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            Details:

            During the extension period of COMET, patients who had initially received Nexviazyme continued their treatment, while patients who were previously treated with alglucosidase alfa switched to Nexviazyme 20 mg/kg.

            Lead Product(s): Avalglucosidase Alfa-ngpt

            Therapeutic Area: Genetic Disease Product Name: Nexviazyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 08, 2022

            Sanofi Company Banner

            TMF Summit 2024

            Not Confirmed

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            Details:

            Nexviazyme® (avalglucosidase alfa-ngpt), is an enzyme replacement therapy designed to target the mannose6-phosphate (M6P) receptor, ) for treatment of late-onset Pompe disease in patients one year of age and older.

            Lead Product(s): Avalglucosidase Alfa

            Therapeutic Area: Genetic Disease Product Name: Nexviazyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 12, 2021

            Sanofi Company Banner

            TMF Summit 2024

            Not Confirmed

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            Details:

            Nexviazyme, (avalglucosidase alfa-ngpt) has demonstrated improvements for people living with late-onset Pompe disease. In pivotal Phase 3 trial, showed improvements in respiratory function and walking distance measures in people with LOPD and established its safety profile.

            Lead Product(s): Avalglucosidase Alfa

            Therapeutic Area: Genetic Disease Product Name: Nexviazyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 06, 2021

            Sanofi Company Banner

            TMF Summit 2024

            Not Confirmed

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            Details:

            Fabrazyme (agalsidase beta) works by replacing a naturally occurring enzyme (alpha-galactosidase A) to help clear GL-3 build-up in cells, including those lining the blood vessels of the kidneys, heart and skin. It is the only FDA-approved therapy for Fabry disease.

            Lead Product(s): Agalsidase Beta

            Therapeutic Area: Genetic Disease Product Name: Fabrazyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 12, 2020

            Sanofi Company Banner

            TMF Summit 2024

            Not Confirmed

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            Details:

            Cuprior (trientine tetrahydrochloride) is a copper chelator which eliminates absorbed copper from the body by forming a stable complex that is then eliminated through urinary excretion. It is approved for the treatment of Wilson disease.

            Lead Product(s): Trientine Tetrahydrochloride

            Therapeutic Area: Genetic Disease Product Name: Cuprior

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 18, 2024

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