Quotient Sciences Translational Pharmaceuticals Quotient Sciences Translational Pharmaceuticals

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Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Veopoz\u2122 (pozelimab-bbfg) Receives FDA Approval as the First Treatment for Children and Adults with CHAPLE Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"August 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Regeneron Pharmaceuticals"},{"orgOrder":0,"company":"Orsini Specialty Pharmacy","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orsini Specialty Pharmacy Selected to be the Exclusive Specialty Pharmacy Partner for VEOPOZ\u2122 (pozelimab-bbfg), the First and Only Treatment for CHAPLE Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"August 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Orsini Specialty Pharmacy"},{"orgOrder":0,"company":"Daewoong Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Daewoong\u2019s Botulinum Toxin Wins 'World\u2019s 1st' Nod to Treat Square Jaw","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"SOUTH KOREA","productType":"Large molecule","productStatus":"Approved","date":"August 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Daewoong Pharmaceutical"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Update on US Regulatory Review of Ultomiris in NMOSD","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"Approved","date":"September 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"AstraZeneca"}]

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            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.

            Lead Product(s): Olipudase Alfa-rpcp

            Therapeutic Area: Rare Diseases and Disorders Product Name: Xenpozyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 31, 2022

            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.

            Lead Product(s): Olipudase Alfa

            Therapeutic Area: Rare Diseases and Disorders Product Name: Xenpozyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 28, 2022

            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.

            Lead Product(s): Olipudase Alfa

            Therapeutic Area: Rare Diseases and Disorders Product Name: Xenpozyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 19, 2022

            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.

            Lead Product(s): Olipudase Alfa

            Therapeutic Area: Rare Diseases and Disorders Product Name: Xenpozyme

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 28, 2022

            Details:

            Ultomiris (ravulizumab), the first and only long-acting C5 complement inhibitor, provides immediate, complete and sustained complement inhibition. It is being recommended for the treatment of neuromyelitis optica spectrum disorder.

            Lead Product(s): Ravulizumab

            Therapeutic Area: Rare Diseases and Disorders Product Name: Ultomiris

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 06, 2023

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            Daewoong Pharmaceutical has added benign masseteric hypertrophy (BMH), commonly known as the square jaw, to the indications for its botulinum toxin (BTX) product, Nabota.

            Lead Product(s): Botulinum toxin type A

            Therapeutic Area: Rare Diseases and Disorders Product Name: Nabota

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 28, 2023

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            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.

            Lead Product(s): Pozelimab-bbfg

            Therapeutic Area: Rare Diseases and Disorders Product Name: Veopoz

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Regeneron Pharmaceuticals

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement August 23, 2023

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            Details:

            Veopoz™ (pozelimab-bbfg) is the proprietary VelocImmune® technology and is a fully human, monoclonal antibody designed to block the activity of complement factor C5 and prevent the CHAPLE Disease.

            Lead Product(s): Pozelimab-bbfg

            Therapeutic Area: Rare Diseases and Disorders Product Name: Veopoz

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 18, 2023

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            Under the agreement, Pharming will sell its Rare Pediatric Disease Priority Review Voucher, in connection with the approval of Joenja® (leniolisib), an oral, selective PI3Kδ inhibitor, for activated PI3Kδ syndrome, a rare and progressive primary immunodeficiency, to Novartis.

            Lead Product(s): Leniolisib

            Therapeutic Area: Rare Diseases and Disorders Product Name: Joenja

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Novartis Pharmaceuticals Corporation

            Deal Size: $21.1 million Upfront Cash: Undisclosed

            Deal Type: Divestment June 01, 2023

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            Details:

            Ultomiris (ravulizumab), the first and only long-acting C5 complement inhibitor, provides immediate, complete and sustained complement inhibition. It is being recommended for the treatment of neuromyelitis optica spectrum disorder.

            Lead Product(s): Ravulizumab

            Therapeutic Area: Rare Diseases and Disorders Product Name: Ultomiris

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 26, 2023

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