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Children's Hospital"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Medipal Holdings Corporation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Protein","year":"2025","type":"Licensing Agreement","leadProduct":"JR-479","moa":"HEXA","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"JCR Pharmaceuticals \/ Medipal Holdings Corporation","highestDevelopmentStatusID":"4","companyTruncated":"JCR Pharmaceuticals \/ Medipal Holdings Corporation"},{"orgOrder":0,"company":"Mytos","sponsor":"Novadip Biosciences","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Collaboration","leadProduct":"NVD-003","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Mytos","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Mytos \/ Novadip Biosciences","highestDevelopmentStatusID":"10","companyTruncated":"Mytos \/ Novadip Biosciences"},{"orgOrder":0,"company":"Alcyone Therapeutics","sponsor":"Biogen","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Acquisition","leadProduct":"CLN-301","moa":"CLN3","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Alcyone Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Alcyone Therapeutics \/ Biogen","highestDevelopmentStatusID":"7","companyTruncated":"Alcyone Therapeutics \/ Biogen"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Elamipretide","moa":"Cardiolipin","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Stealth Biotherapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Stealth Biotherapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Unravel Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Vorinostat","moa":"Histone deacetylase 2 | Histone deacetylase 3 | Histone deacetylase 6 | Histone deacetylase 1","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Unravel Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Unravel Biosciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Unravel Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Unravel Biosciences","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Vorinostat","moa":"Histone deacetylase 2 | Histone deacetylase 3 | Histone deacetylase 6 | Histone deacetylase 1","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Unravel Biosciences","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Unravel Biosciences \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Unravel Biosciences \/ Undisclosed"},{"orgOrder":0,"company":"Haisco Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"HSK39297","moa":"Unknown","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Haisco Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Haisco Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Haisco Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Pozelimab","moa":"Complement C5","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Regeneron Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Regeneron Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Steroid","year":"2025","type":"Inapplicable","leadProduct":"Apazunersen","moa":"Glucocorticoid receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ultragenyx Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Ultragenyx Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"CSPC ZhongQi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Hydroxocobalamin Hydrochloride","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"CSPC ZhongQi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"CSPC ZhongQi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"CSPC ZhongQi \/ Undisclosed"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Avacopan","moa":"C5a anaphylatoxin chemotactic receptor","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Amgen Inc \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Amgen Inc \/ Undisclosed"},{"orgOrder":0,"company":"Nanjing Chia-tai Tianqing Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"NTQ5082","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Nanjing Chia-tai Tianqing Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Nanjing Chia-tai Tianqing Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Nanjing Chia-tai Tianqing Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Nanjing Chia-tai Tianqing Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"NTQ5082","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Nanjing Chia-tai Tianqing Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Nanjing Chia-tai Tianqing Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Nanjing Chia-tai Tianqing Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"MavriX Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"MVX-220","moa":"UBE3A gene","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"MavriX Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"MavriX Bio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"MavriX Bio \/ Undisclosed"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"ALN-CFB","moa":"CFB","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regeneron Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Regeneron Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"ResVita Bio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"RVB-003","moa":"KLK5\/7","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"ResVita Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ResVita Bio \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"ResVita Bio \/ Undisclosed"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Breckenridge Pharmaceutical

                            U.S.A
                            FNCE
                            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) : Everolimus

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Breckenridge Gains FDA Nod for Everolimus, Afinitor Disperz Generic

                            Details : Afinitor Disperz (everolimus) is an mTOR inhibitor small molecule drug candidate, which is currently being evaluated for the treatment of tuberous sclerosis complex.

                            Product Name : Afinitor Disperz-Generic

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            January 29, 2025

                            Lead Product(s) : Everolimus

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Breckenridge Pharmaceutical

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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) : Avalglucosidase Alfa

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD

                            Details : Avalglucosidase Alfa is a Enzyme drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Glycogen Storage Disease Type II.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            October 30, 2024

                            Lead Product(s) : Avalglucosidase Alfa

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            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) : SAR443809

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Test if SAR443809 is Tolerated and Safe When Taken as a Single Dose in Healthy Adults

                            Details : SAR443809 is a Antibody drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Hemoglobinuria, Paroxysmal.

                            Product Name : Undisclosed

                            Product Type : Antibody, Unconjugated

                            Upfront Cash : Inapplicable

                            March 22, 2024

                            Lead Product(s) : SAR443809

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            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) : Avalglucosidase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            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 : Enzyme

                            Upfront Cash : Inapplicable

                            February 05, 2024

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Orion Corporation

                            Finland
                            FNCE
                            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) : Ganaxolone

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Marinus Pharmaceuticals

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            European Commission Approval of Ztalmy (ganaxolone) for the Adjunctive Treatment of Epileptic Seizures Associa...

                            Details : Ztalmy (ganaxolone) is a positive allosteric modulation of the gamma-aminobutyric acid type A (GABAA) receptor in the CNS. It is used for the treatment of epileptic seizures associated with CDKL5 deficiency disorder.

                            Product Name : Ztalmy

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            July 31, 2023

                            Lead Product(s) : Ganaxolone

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Marinus Pharmaceuticals

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Fermion Orion Company Banner

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                            06

                            Zenara Pharma

                            India
                            FNCE
                            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) : Cannabidiol

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Biophore, Its Subsidiary Zenara Pharma, Receive First Approval for Cannabidiol in India

                            Details : Cannabinoid oral solution has received approval for the treatment of several neurological disorders. It functions by stimulating two receptors, cannabinoid receptor type 1 and type 2, within the endocannabinoid system, maintaining body's homeostatis.

                            Product Name : Undisclosed

                            Product Type : Controlled Substance

                            Upfront Cash : Inapplicable

                            May 15, 2023

                            Lead Product(s) : Cannabidiol

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Biophore CB

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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 : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            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 : Enzyme

                            Upfront Cash : Inapplicable

                            February 24, 2023

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            08

                            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) : Olipudase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Xenpozyme TM (Olipudase Alfa-rpcp) Approved by FDA as First Disease-specific Treatment for ASMD (Non-CNS Manif...

                            Details : Xenpozyme, (olipudase Alfa-rpcp) hydrolytic lysosomal sphingomyelin-specific enzyme replacement therapy, is designed to replace deficient or defective acid sphingomyelinase, an enzyme that allows for the breakdown of the lipid sphingomyelin.

                            Product Name : Xenpozyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            August 31, 2022

                            Lead Product(s) : Olipudase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            09

                            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) : Caplacizumab

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune...

                            Details : Caplacizumab is a Antibody drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Purpura, Thrombotic Thrombocytopenic.

                            Product Name : Undisclosed

                            Product Type : Antibody, Unconjugated

                            Upfront Cash : Inapplicable

                            July 21, 2022

                            Lead Product(s) : Caplacizumab

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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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 : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            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 : Enzyme

                            Upfront Cash : Inapplicable

                            June 28, 2022

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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