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Molecule","year":"2025","type":"Inapplicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"Cholesterol and lipid accumulation","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"ZyVersa Therapeutics, Inc","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ZyVersa Therapeutics, Inc \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"ZyVersa Therapeutics, Inc \/ Undisclosed"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Sepiapterin","moa":"eNOS","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder","sponsorNew":"PTC Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"PTC Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"Concizumab","moa":"Tissue factor pathway inhibitor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Novo Nordisk","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Novo Nordisk \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Novo Nordisk \/ Undisclosed"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"Donidalorsen Sodium","moa":"antisense oligonucleotides","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ionis Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Ionis Pharmaceuticals \/ Undisclosed"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • 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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                            02

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • 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 : Undisclosed

                            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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                            03

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • 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

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                            04

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • 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 : Undisclosed

                            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

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                            05

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • 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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                            06

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • 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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                            07

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • 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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                            08

                            Breckenridge Pharmaceutical

                            U.S.A
                            BioProcess International
                            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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                            09

                            Breckenridge Pharmaceutical

                            U.S.A
                            BioProcess International
                            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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                            10

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • 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

                            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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