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Molecule","year":"2022","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Ftase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ The Progeria Research Foundation","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ The Progeria Research Foundation"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Ftase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Ftase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Lonafarnib","moa":"Ftase","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Eiger BioPharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Eiger BioPharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Fosdenopterin","moa":"cPMP","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"BridgeBio Pharma \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Inapplicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Fosdenopterin","moa":"cPMP","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"BridgeBio Pharma \/ Sentynl Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Fosdenopterin","moa":"cPMP","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"BridgeBio Pharma \/ Sentynl Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Fosdenopterin","moa":"cPMP","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"BridgeBio Pharma \/ Sentynl Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Agreement","leadProduct":"Fosdenopterin","moa":"cPMP","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"BridgeBio Pharma \/ Sentynl Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Sentynl Therapeutics"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Fosdenopterin","moa":"cPMP","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"BridgeBio Pharma","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"BridgeBio Pharma \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"BridgeBio Pharma \/ Inapplicable"},{"orgOrder":0,"company":"KYE Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Steroid","year":"2025","type":"Inapplicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"KYE Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"KYE Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"KYE Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Zydus Lifesciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Steroid","year":"2025","type":"Inapplicable","leadProduct":"Deflazacort","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Zydus Lifesciences","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Zydus Lifesciences \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Zydus Lifesciences \/ Inapplicable"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Maralixibat Chloride","moa":"IBAT","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Mirum Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Mirum Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Diazoxide","moa":"Potassium channel","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Soleno Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet, Extended Release","sponsorNew":"Soleno Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Soleno Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Belief BioMed","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"Dalnacogene Ponparvovec","moa":"FIX gene","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Belief BioMed","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Belief BioMed \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"Belief BioMed \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Italfarmaco S.p.A","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Givinostat HCl","moa":"HDAC","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Italfarmaco S.p.A","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"Italfarmaco S.p.A \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Italfarmaco S.p.A \/ Inapplicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"||CFTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Lupin Ltd","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Tolvaptan","moa":"V2 receptor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Lupin Ltd","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Immediate-Release Tablet","sponsorNew":"Lupin Ltd \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Lupin Ltd \/ Inapplicable"},{"orgOrder":0,"company":"Lonza Group","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell and Gene therapy","year":"2024","type":"Agreement","leadProduct":"Exagamglogene Autotemcel","moa":"BCL11A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Lonza Group","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Lonza Group \/ Vertex Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Lonza Group \/ Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Inapplicable"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • 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) : Avalglucosidase Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            New Data Show Safety, Benefit, and Durability of Nexviazyme in Pompe Disease Patients

                            Details : Nexviazyme (avalglucosidase alfa) is a USFDA approved GAA stimulator enzyme replacement therapy. It is being evaluated for the treatment of treatment-naïve Infantile-onset Pompe disease.

                            Product Name : Nexviazyme

                            Product Type : Other Large Molecule

                            Upfront Cash : Inapplicable

                            May 02, 2024

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Virtual Booth

                            02

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • 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) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Once-weekly ALTUVIIIO® Approved in Japan as a New Class of Factor VIII Therapy for Hemophilia A

                            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.

                            Product Name : BIVV001

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            September 25, 2023

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Virtual Booth

                            03

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • 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) : Avalglucosidase Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            New Phase 3 Data Presented At WORLD Symposium™ Reinforce Nexviazyme® (avalglucosidase alfa) As Potential Ne...

                            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.

                            Product Name : Nexviazyme

                            Product Type : Other Large Molecule

                            Upfront Cash : Inapplicable

                            February 24, 2023

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            Virtual Booth

                            04

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • 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) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            FDA Approves Once-Weekly ALTUVIIIO A New Class of Factor VIII Therapy for Hemophilia a That Offers Significant...

                            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 haemophi...

                            Product Name : BIVV001

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            February 23, 2023

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            Virtual Booth

                            05

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • 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) : Eliglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Major Life Saving Drugs Program Funding for Rare Genetic Disease

                            Details : 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).

                            Product Name : Cerdelga

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 04, 2023

                            Lead Product(s) : Eliglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            Virtual Booth

                            06

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • 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) : Avalglucosidase Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Nexviazyme® (Avalglucosidase Alfa) Shows Sustained Improvements in Respiratory Function and Mobility in Patie...

                            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.

                            Product Name : Nexviazyme

                            Product Type : Other Large Molecule

                            Upfront Cash : Inapplicable

                            August 02, 2022

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            Virtual Booth

                            07

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • 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) : Avalglucosidase Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Nexviadyme® (avalglucosidase alfa) Approved by European Commission as a Potential New Standard of Care for th...

                            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®.

                            Product Name : Nexviadyme

                            Product Type : Other Large Molecule

                            Upfront Cash : Inapplicable

                            June 28, 2022

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            Virtual Booth

                            08

                            Breckenridge Pharmaceutical

                            U.S.A
                            STLE Annual Meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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) : Miglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Breckenridge Announces Approval of its ANDA for Miglustat Capsules (generic for Zavesca®)

                            Details : Miglustat Capsules (generic for Zavesca), 100mg strength, is an inhibitor of the enzyme glucosylceramide synthase, which is a glucosyl transferase enzyme responsible for first step in synthesis of most glycosphingolipids.

                            Product Name : Miglustat-Generic

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            April 21, 2022

                            Lead Product(s) : Miglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Breckenridge Pharmaceutical

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                            Virtual Booth

                            09

                            Breckenridge Pharmaceutical

                            U.S.A
                            STLE Annual Meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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) : Penicillamine

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Breckenridge Pharmaceutical, Inc. Announces Launch of Penicillamine Capsules, USP (generic for Cuprimine®)

                            Details : Cuprimine-Generic (penicillamine) is a copper chelating agent, small molecule drug candidate, which is being evauated for the treatment of Wilson's disease.

                            Product Name : Cuprimine-Generic

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            April 20, 2022

                            Lead Product(s) : Penicillamine

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Breckenridge Pharmaceutical

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                            Virtual Booth

                            10

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • 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) : Avalglucosidase Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Sanofi Announces Results of CHMP Re-Examination of the New Active Substance status for Avalglucosidase alfa, a...

                            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.

                            Product Name : Nexviazyme

                            Product Type : Other Large Molecule

                            Upfront Cash : Inapplicable

                            December 11, 2021

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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