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Diseases","amount2New":1.8,"dosageForm":"Undisclosed","sponsorNew":"Skyhawk Therapeutics \/ Ipsen","highestDevelopmentStatusID":"2","companyTruncated":"Skyhawk Therapeutics \/ Ipsen"},{"orgOrder":0,"company":"Institute of Hematology & Blood Diseases Hospital, China","sponsor":"R&D Kanglin Biotech","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"KL003","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Institute of Hematology & Blood Diseases Hospital, China","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Institute of Hematology & Blood Diseases Hospital, China \/ R&D Kanglin Biotech","highestDevelopmentStatusID":"1","companyTruncated":"Institute of Hematology & Blood Diseases Hospital, China \/ R&D Kanglin Biotech"},{"orgOrder":0,"company":"Institute of Hematology & Blood Diseases Hospital, China","sponsor":"Kanglin Biotechnology","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"KL003","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Institute of Hematology & Blood Diseases Hospital, China","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Institute of Hematology & Blood Diseases Hospital, China \/ Kanglin Biotechnology","highestDevelopmentStatusID":"1","companyTruncated":"Institute of Hematology & Blood Diseases Hospital, China \/ Kanglin Biotechnology"},{"orgOrder":0,"company":"Albireo","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Odevixibat","moa":"Ileal bile acid transporter","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Albireo","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Albireo \/ Inapplicable","highestDevelopmentStatusID":"1","companyTruncated":"Albireo \/ Inapplicable"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Ganaxolone","moa":"GABA-A receptor; anion channel","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Marinus Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Marinus Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"NovoBliss Research","sponsor":"Orgenetics, Inc.","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Biotin","moa":"Krebs cycle activatior","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"NovoBliss Research","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NovoBliss Research \/ Orgenetics, Inc.","highestDevelopmentStatusID":"1","companyTruncated":"NovoBliss Research \/ Orgenetics, Inc."},{"orgOrder":0,"company":"Hamad Medical Corporation","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"L-Cysteine","moa":"Glutamate--cysteine ligase regulatory subunit; Glutamate--cysteine ligase catalytic subunit; Aspartate aminotransferase, cytoplasmic; Aspartate aminotransferase, mitochondrial; Cystathionine gamma-lyase; Cystathionine beta-synthase; Cysteine--tRNA ligase, cytoplasmic; Methylated-DNA--protein-cysteine methyltransferase; Glutathione synthetase; Probable cysteine--tRNA ligase, mitochondrial; Cysteine sulfinic acid decarboxylase; Cysteine desulfurase; Cysteine dioxygenase type 1","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Hamad Medical Corporation","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Powder","sponsorNew":"Hamad Medical Corporation \/ Inapplicable","highestDevelopmentStatusID":"1","companyTruncated":"Hamad Medical Corporation \/ Inapplicable"},{"orgOrder":0,"company":"Oryzon Genomics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SPAIN","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Vafidemstat","moa":"Lysine-specific histone demethylase 1 (LSD)","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Oryzon Genomics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Oryzon Genomics \/ Inapplicable","highestDevelopmentStatusID":"1","companyTruncated":"Oryzon Genomics \/ Inapplicable"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Peptide","year":"2020","type":"Inapplicable","leadProduct":"Elamipretide","moa":"peptides","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Stealth Biotherapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Stealth Biotherapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Bioasis","sponsor":"Chiesi Group","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Peptide","year":"2020","type":"Licensing Agreement","leadProduct":"Transcend Peptide","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Bioasis","amount2":0.14000000000000001,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0.14000000000000001,"dosageForm":"Undisclosed","sponsorNew":"Bioasis \/ Chiesi Group","highestDevelopmentStatusID":"1","companyTruncated":"Bioasis \/ Chiesi Group"},{"orgOrder":0,"company":"ResVita Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Probiotic","year":"2022","type":"Inapplicable","leadProduct":"RVB-001","moa":"Proteolysis","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"ResVita Bio","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical","sponsorNew":"ResVita Bio \/ Inapplicable","highestDevelopmentStatusID":"1","companyTruncated":"ResVita Bio \/ Inapplicable"},{"orgOrder":0,"company":"Ovid Therapeutics","sponsor":"University of Connecticut","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Gaboxadol","moa":"||Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Ovid Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ovid Therapeutics \/ University of Connecticut","highestDevelopmentStatusID":"1","companyTruncated":"Ovid Therapeutics \/ University of Connecticut"},{"orgOrder":0,"company":"University of North Carolina","sponsor":"AskBio","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"University of North Carolina","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of North Carolina \/ AskBio","highestDevelopmentStatusID":"4","companyTruncated":"University of North Carolina \/ AskBio"},{"orgOrder":0,"company":"Medable","sponsor":"Every Cure","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Medable","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Medable \/ Every Cure","highestDevelopmentStatusID":"1","companyTruncated":"Medable \/ Every Cure"},{"orgOrder":0,"company":"XOMA","sponsor":"Chiesi Group","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"XOMA","amount2":0.14999999999999999,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0.14999999999999999,"dosageForm":"Undisclosed","sponsorNew":"XOMA \/ Chiesi Group","highestDevelopmentStatusID":"1","companyTruncated":"XOMA \/ Chiesi Group"},{"orgOrder":0,"company":"Selecta Biosciences","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Selecta Biosciences","amount2":1.1200000000000001,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":1.1200000000000001,"dosageForm":"Undisclosed","sponsorNew":"Selecta Biosciences \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"1","companyTruncated":"Selecta Biosciences \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Minovia Therapeutics","sponsor":"Astellas Pharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"ISRAEL","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Minovia Therapeutics","amount2":0.41999999999999998,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0.41999999999999998,"dosageForm":"Undisclosed","sponsorNew":"Minovia Therapeutics \/ Astellas Pharma","highestDevelopmentStatusID":"1","companyTruncated":"Minovia Therapeutics \/ Astellas Pharma"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Organovo Holdings","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Undisclosed","year":"2021","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Hemab Therapeutics","amount2":0.059999999999999998,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0.059999999999999998,"dosageForm":"Undisclosed","sponsorNew":"Hemab Therapeutics \/ Organovo Holdings","highestDevelopmentStatusID":"1","companyTruncated":"Hemab Therapeutics \/ Organovo Holdings"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Inapplicable","highestDevelopmentStatusID":"1","companyTruncated":"CSL Behring \/ Inapplicable"},{"orgOrder":0,"company":"Azienda Ospedaliera Universitaria Senese","sponsor":"Kolfarma s.r.l. - Italy | Gazi University","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"ALAC","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Azienda Ospedaliera Universitaria Senese","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Azienda Ospedaliera Universitaria Senese \/ Kolfarma s.r.l. - Italy | Gazi University","highestDevelopmentStatusID":"1","companyTruncated":"Azienda Ospedaliera Universitaria Senese \/ Kolfarma s.r.l. - Italy | Gazi University"},{"orgOrder":0,"company":"Bioray Laboratories","sponsor":"First Affiliated Hospital of Guangxi Medical University","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"\u0392-Globin Reactivated Autologous Hematopoietic Stem Cell","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Bioray Laboratories","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Bioray Laboratories \/ First Affiliated Hospital of Guangxi Medical University","highestDevelopmentStatusID":"1","companyTruncated":"Bioray Laboratories \/ First Affiliated Hospital of Guangxi Medical University"},{"orgOrder":0,"company":"Institute of Hematology & Blood Diseases Hospital, China","sponsor":"Shanghai Vitalgen BioPharma Co., Ltd.","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"VGB-Ex01","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Institute of Hematology & Blood Diseases Hospital, China","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Institute of Hematology & Blood Diseases Hospital, China \/ Shanghai Vitalgen BioPharma Co., Ltd.","highestDevelopmentStatusID":"1","companyTruncated":"Institute of Hematology & Blood Diseases Hospital, China \/ Shanghai Vitalgen BioPharma Co., Ltd."},{"orgOrder":0,"company":"Genecombio Ltd.","sponsor":"Xinhua Hospital, Shanghai Jiaotong University School of Medicine","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Undisclosed","year":"2024","type":"Inapplicable","leadProduct":"GCB-002","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Genecombio Ltd.","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Genecombio Ltd. \/ Xinhua Hospital, Shanghai Jiaotong University School of Medicine","highestDevelopmentStatusID":"1","companyTruncated":"Genecombio Ltd. \/ Xinhua Hospital, Shanghai Jiaotong University School of Medicine"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Breckenridge Pharmaceutical

                            U.S.A
                            Pharmaconex
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                            • 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 : Inapplicable

                            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 : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 29, 2025

                            Lead Product(s) : Everolimus

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Sanofi

                            France
                            ChemOutsourcing
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                            • EDQM
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                            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 : Inapplicable

                            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 : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            March 22, 2024

                            Lead Product(s) : SAR443809

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Orion Corporation

                            Finland
                            Pharmaconex
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                            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 : Other Small Molecule

                            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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                            04

                            Zenara Pharma

                            India
                            Pharmaconex
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                            Lead Product(s) : Cannabidiol

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Approved FDF

                            Sponsor : Inapplicable

                            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 : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Biophore CB

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                            05

                            Sanofi

                            France
                            ChemOutsourcing
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                            • fda
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                            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 : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Olipudase alfa Shown to Provide Sustained Improvement Across Multiple Clinical Manifestations of ASMD

                            Details : Investigational data from long-term, follow-up studies showed that olipudase alfa provided sustained improvement in lung function (as measured by DLco) and reduction of spleen and liver volumes over time in patients with ASMD.

                            Product Name : Xenpozyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            September 02, 2022

                            Lead Product(s) : Olipudase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Sanofi

                            France
                            ChemOutsourcing
                            Attending
                            • fda
                            • EDQM
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                            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 : Inapplicable

                            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 : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            07

                            Sanofi

                            France
                            ChemOutsourcing
                            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 : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

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

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            July 21, 2022

                            Lead Product(s) : Caplacizumab

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            08

                            Sanofi

                            France
                            ChemOutsourcing
                            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 : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Xenpozyme® (olipudase alfa) Approved by European Commission as First and Only treatment for ASMD

                            Details : Xenpozyme (Olipudase alfa), is an enzyme replacement therapy designed to replace deficient or defective acid sphingomyelinase (ASM), an enzyme that allows for the breakdown of the lipid sphingomyelin.

                            Product Name : Xenpozyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            June 28, 2022

                            Lead Product(s) : Olipudase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Sanofi

                            France
                            ChemOutsourcing
                            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 : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            CHMP Recommends Approval of Xenpozyme® (Olipudase Alfa), the First and Only Treatment for ASMD

                            Details : Recommendation based on positive results from two clinical trials in which Xenpozyme (olipudase alfa) provided improvement across multiple non-CNS clinical manifestations of ASMD in pediatric and adult patients.

                            Product Name : Xenpozyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            May 19, 2022

                            Lead Product(s) : Olipudase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Sanofi

                            France
                            ChemOutsourcing
                            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 : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Xenpozyme® (olipudase alfa), Approved in Japan, First and Only Approved Therapy Indicated to Treat Acid Sphin...

                            Details : Approval based on positive results of Xenpozyme® (olipudase alfa) from two separate clinical trials in children and adults demonstrating improvement in lung function (as measured by DLco) and reduction of spleen and liver volumes.

                            Product Name : Xenpozyme

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            March 28, 2022

                            Lead Product(s) : Olipudase Alpha

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Approved FDF

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            #PipelineProspector

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