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Lyophilized","sponsorNew":"Sanofi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sanofi \/ Undisclosed"},{"orgOrder":0,"company":"KORU Medical Systems","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"KORU Medical Systems","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"KORU Medical Systems \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"KORU Medical Systems \/ Undisclosed"},{"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":"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":"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":"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"}]

    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) : 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

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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 III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase ...

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

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            June 02, 2021

                            Lead Product(s) : Avalglucosidase Alfa

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            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) : Avalglucosidase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

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

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

                            Product Name : Nexviazyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            November 18, 2020

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            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 : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            EMA Accepts Regulatory Submission for Avalglucosidase Alfa, a Potentially new Standard of Care Enzyme Replacem...

                            Details : The MAA is based on positive data from two trials Phase 3 pivotal COMET trial, which evaluated the safety and efficacy of avalglucosidase alfa compared to alglucosidase alfa in patients with late-onset Pompe disease and Phase 2 mini-COMET trial.

                            Product Name : Nexviazyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            October 02, 2020

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            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) : Avalglucosidase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            EMA Accepts Sanofi's Regulatory Submission for Avalglucosidase Alfa for Pompe Disease

                            Details : The MAA is based on positive data from two trials: Phase 3, pivotal COMET trial, which evaluated the safety and efficacy of avalglucosidase alfa compared to alglucosidase alfa (standard of care) in patients with late-onset Pompe disease, and Phase 2 mini...

                            Product Name : Nexviazyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            October 02, 2020

                            Lead Product(s) : Avalglucosidase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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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 : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Sanofi’s Investigational Enzyme Replacement Therapy Improves Critical Manifestations of Late-Onset Pompe Dis...

                            Details : Avalglucosidase alfa showed a 2.4-point improvement in percent-predicted forced vital capacity, an important measure of respiratory function in Pompe disease, compared to alglucosidase alfa (standard of care).

                            Product Name : Nexviazyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            June 16, 2020

                            Lead Product(s) : Avalglucosidase Alpha

                            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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                            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 : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Sanofi To Present Phase 3 Results of Avalglucosidase Alfa in Patients with Late-Onset Pompe Disease

                            Details : Sanofi will host a virtual scientific session to present data from the Phase 3 COMET trial of investigational enzyme replacement therapy (ERT) avalglucosidase alfa in patients with late-onset Pompe disease (LOPD).

                            Product Name : Nexviazyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            June 08, 2020

                            Lead Product(s) : Avalglucosidase Alpha

                            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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                            08

                            Nanjing Chia-tai Tianqing Pharmaceutical

                            Country arrow
                            FNCE
                            Not Confirmed

                            Nanjing Chia-tai Tianqing Pharmaceutical

                            Country arrow
                            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) : NTQ5082

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients

                            Details : (NTQ5082) is currently being evaluated in Phase III clinical studies for the treatment of Hemoglobinuria, Paroxysmal.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            September 17, 2025

                            Lead Product(s) : NTQ5082

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Nanjing Chia-tai Tianqing Pharmaceutical

                            Country arrow
                            FNCE
                            Not Confirmed

                            Nanjing Chia-tai Tianqing Pharmaceutical

                            Country arrow
                            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) : NTQ5082

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Long-term Efficacy and Safety of NTQ5082 Capsules

                            Details : (NTQ5082) is currently being evaluated in Phase III clinical studies for the treatment of Hemoglobinuria, Paroxysmal.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            September 17, 2025

                            Lead Product(s) : NTQ5082

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Regeneron Pharmaceuticals

                            U.S.A arrow
                            FNCE
                            Not Confirmed

                            Regeneron Pharmaceuticals

                            U.S.A arrow
                            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) : Pozelimab

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Noc...

                            Details : Pozelimab is a Antibody, Unconjugated drug candidate, which is currently being evaluated in Phase III clinical studies for the treatment of Hemoglobinuria, Paroxysmal.

                            Product Name : Undisclosed

                            Product Type : Antibody, Unconjugated

                            Upfront Cash : Inapplicable

                            September 04, 2025

                            Lead Product(s) : Pozelimab

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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