[{"orgOrder":0,"company":"Orsini Specialty Pharmacy","sponsor":"Alnylam Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Partnership","leadProduct":"Vutrisiran Sodium","moa":"TTR","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Orsini Specialty Pharmacy","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Orsini Specialty Pharmacy \/ Alnylam Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Orsini Specialty Pharmacy \/ Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"Orsini Specialty Pharmacy","sponsor":"Alnylam Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Partnership","leadProduct":"Lumasiran","moa":"Glycolate oxidase","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Orsini Specialty Pharmacy","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Orsini Specialty Pharmacy \/ Alnylam Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Orsini Specialty Pharmacy \/ Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"Orsini Specialty Pharmacy","sponsor":"Amicus Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2023","type":"Agreement","leadProduct":"Cipaglucosidase Alpha","moa":"||Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Orsini Specialty Pharmacy","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orsini Specialty Pharmacy \/ Amicus Therapeutics","highestDevelopmentStatusID":"15","companyTruncated":"Orsini Specialty Pharmacy \/ Amicus Therapeutics"},{"orgOrder":0,"company":"Orsini Specialty Pharmacy","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2023","type":"Agreement","leadProduct":"Pozelimab","moa":"C5 receptor","graph1":"Rare Diseases and Disorders","graph2":"Approved FDF","graph3":"Orsini Specialty Pharmacy","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orsini Specialty Pharmacy \/ Regeneron Pharmaceuticals","highestDevelopmentStatusID":"15","companyTruncated":"Orsini Specialty Pharmacy \/ Regeneron Pharmaceuticals"},{"orgOrder":0,"company":"Orsini Specialty Pharmacy","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Large Molecule","year":"2021","type":"Partnership","leadProduct":"Avalglucosidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Orsini Specialty Pharmacy","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orsini Specialty Pharmacy \/ Sanofi","highestDevelopmentStatusID":"15","companyTruncated":"Orsini Specialty Pharmacy \/ Sanofi"},{"orgOrder":0,"company":"Orsini Specialty Pharmacy","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Partnership","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Orsini Specialty Pharmacy","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orsini Specialty Pharmacy \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"Orsini Specialty Pharmacy \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Orsini Specialty Pharmacy","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Infections and Infectious Diseases","country":"U.S.A","productType":"Microorganism","year":"2023","type":"Inapplicable","leadProduct":"Fecal Microbiota Spores, Live","moa":"Undisclosed","graph1":"Infections and Infectious Diseases","graph2":"Approved FDF","graph3":"Orsini Specialty Pharmacy","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Infectious Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Orsini Specialty Pharmacy \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Orsini Specialty Pharmacy \/ Inapplicable"},{"orgOrder":0,"company":"Orsini Specialty Pharmacy","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Blood coagulation factor","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Orsini Specialty Pharmacy","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Orsini Specialty Pharmacy \/ BioMarin Pharmaceutical","highestDevelopmentStatusID":"15","companyTruncated":"Orsini Specialty Pharmacy \/ BioMarin Pharmaceutical"}]

Find Clinical Drug Pipeline Developments & Deals by Orsini Specialty Pharmacy

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          AMWC Asia-TDAC
                          Not Confirmed
                          AMWC Asia-TDAC
                          Not Confirmed

                          Details : Roctavian (valoctocogene roxaparvovec) is a gene therapy that uses an adeno-associated virus 5, codes for human Factor VIII, together with a human liver-specific promoter. It is currently being investigated for adults with severe hemophilia A.

                          Product Name : Roctavian

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          November 07, 2023

                          Lead Product(s) : Valoctocogene Roxaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : BioMarin Pharmaceutical

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          AMWC Asia-TDAC
                          Not Confirmed
                          AMWC Asia-TDAC
                          Not Confirmed

                          Details : Under the agreement, Orsini will dispense Pombiliti™ (cipaglucosidase alfa-atga), a hydrolytic lysosomal glycogen-specific enzyme, and Opfolda™ (miglustat), a two-component treatment approved for certain patients with late-onset Pompe disease.

                          Product Name : Pombiliti

                          Product Type : Enzyme

                          Upfront Cash : Undisclosed

                          September 28, 2023

                          Lead Product(s) : Cipaglucosidase Alpha,Miglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Amicus Therapeutics

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          03

                          AMWC Asia-TDAC
                          Not Confirmed
                          AMWC Asia-TDAC
                          Not Confirmed

                          Details : Under the agreement, Orsini will be the exclusive specialty pharmacy partner for Veopoz (pozelimab-bbfg), a monoclonal antibody and the first and only treatment for those living with CHAPLE disease.

                          Product Name : Veopoz

                          Product Type : Antibody

                          Upfront Cash : Undisclosed

                          August 23, 2023

                          Lead Product(s) : Pozelimab

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Regeneron Pharmaceuticals

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          04

                          AMWC Asia-TDAC
                          Not Confirmed
                          AMWC Asia-TDAC
                          Not Confirmed

                          Details : Vowst (fecal microbiota spores, live-brpk) is approved by FDA as the first orally administered microbiota-based therapeutic for the prevention of recurrent C. difficile infection (CDI).

                          Product Name : Vowst

                          Product Type : Microorganism

                          Upfront Cash : Inapplicable

                          June 23, 2023

                          Lead Product(s) : Fecal Microbiota Spores, Live

                          Therapeutic Area : Infections and Infectious Diseases

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          AMWC Asia-TDAC
                          Not Confirmed
                          AMWC Asia-TDAC
                          Not Confirmed

                          Details : AMVUTTRA (vutrisiran), is indicated to treat the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The drug works to reduce the production of the transthyretin (TTR) protein in the liver, thereby reducing the levels of TT...

                          Product Name : Amvuttra

                          Product Type : Oligonucleotide

                          Upfront Cash : Undisclosed

                          July 20, 2022

                          Lead Product(s) : Vutrisiran Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Alnylam Pharmaceuticals

                          Deal Size : Undisclosed

                          Deal Type : Partnership

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                          06

                          AMWC Asia-TDAC
                          Not Confirmed
                          AMWC Asia-TDAC
                          Not Confirmed

                          Details : Sanofi has selected Orsini Specialty Pharmacy as a distribution partner for Nexviazyme® (avalglucosidase alfa-ngpt), an enzyme replacement therapy (ERT), used for treating patients one year of age and older with late-onset Pompe disease.

                          Product Name : Nexviazyme

                          Product Type : Other Large Molecule

                          Upfront Cash : Undisclosed

                          September 27, 2021

                          Lead Product(s) : Avalglucosidase Alpha

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Sanofi

                          Deal Size : Undisclosed

                          Deal Type : Partnership

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                          07

                          AMWC Asia-TDAC
                          Not Confirmed
                          AMWC Asia-TDAC
                          Not Confirmed

                          Details : Orsini Pharmaceutical Services has been selected by Ultragenyx Pharmaceutical as a limited distribution specialty pharmacy partner for Dojolvi (triheptanoin). Dojolvi™ is indicated as a source of calories and fatty acids for the treatment of molecularl...

                          Product Name : Dojolvi

                          Product Type : Other Small Molecule

                          Upfront Cash : Undisclosed

                          October 07, 2020

                          Lead Product(s) : Triheptanoin

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Ultragenyx Pharmaceutical

                          Deal Size : Undisclosed

                          Deal Type : Partnership

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                          08

                          AMWC Asia-TDAC
                          Not Confirmed
                          AMWC Asia-TDAC
                          Not Confirmed

                          Details : Orsini Specialty Pharmacy been selected by Alnylam® Pharmaceuticals as a limited distribution partner for OXLUMO™. OXLUMO is the first and only treatment approved for primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and ...

                          Product Name : Oxlumo

                          Product Type : Oligonucleotide

                          Upfront Cash : Undisclosed

                          March 12, 2020

                          Lead Product(s) : Lumasiran

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved FDF

                          Sponsor : Alnylam Pharmaceuticals

                          Deal Size : Undisclosed

                          Deal Type : Partnership

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