Quotient Sciences Quotient Sciences

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Japan for the Prevention of Relapses in Patients with Neuromyelitis Optica Spectrum Disorder (NMOSD)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"Approved","date":"May 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"AstraZeneca"},{"orgOrder":0,"company":"Pharming","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","amount":"$21.1 million","upfrontCash":"Undisclosed","newsHeadline":"Pharming Announces Sale of Priority Review Voucher","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"NETHERLANDS","productType":"Small molecule","productStatus":"Approved","date":"June 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharming"},{"orgOrder":0,"company":"Greenwich Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Approves EPIDIOLEX (cannabidiol) Oral Solution to Treat Seizures Associated with Tuberous Sclerosis Complex","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"August 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Greenwich Biosciences"},{"orgOrder":0,"company":"Jazz Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Jazz Pharmaceuticals Gets NICE Backing for Its Cannabinoid-Based Medicine","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"January 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Jazz Pharmaceuticals"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Not 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"},{"orgOrder":0,"company":"Apellis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Apellis Announces U.S. FDA Approval of the EMPAVELI\u00ae Injector, a Device to Streamline Self-Administration","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Peptide","productStatus":"Approved","date":"October 2023","url1":"","url2":"","graph1":"Peptide","graph2":"Apellis Pharmaceuticals"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chugai\u2019s Enspryng Launched in Taiwan as First Indication for Neuromyelitis Optica Spectrum Disorder (NMOSD)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"October 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Chugai Pharmaceutical"},{"orgOrder":0,"company":"American Regent","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"American Regent Introduces Levocarnitine Injection, USP; FDA-Approved, \"AP\" Rated, and Therapeutically Equivalent to Carnitor\u00ae\u00b9","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"October 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"American Regent"},{"orgOrder":0,"company":"Pint Pharma","sponsor":"OrphanDC","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Empaveli is the First Treatment for PNH that Binds to the Complement Protein C3","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"AUSTRIA","productType":"Peptide","productStatus":"Approved","date":"October 2023","url1":"","url2":"","graph1":"Peptide","graph2":"Pint Pharma"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Approves Rivfloza\u2122 for Children \u22659 Years Old and Adults Living with Primary Hyperoxaluria Type 1 (PH1), a Rare Genetic Condition","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"DENMARK","productType":"Large molecule","productStatus":"Approved","date":"October 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Novo Nordisk"},{"orgOrder":0,"company":"EA Pharma","sponsor":"Eisai","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"All-case Surveillance Condition for Approval of \u201cActonel\u00ae 17.5 mg tablets\u201d for Treatment of Paget\u2019s Disease of Bone Cleared in Japan","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Small molecule","productStatus":"Approved","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"EA Pharma"},{"orgOrder":0,"company":"OMERS Life Sciences","sponsor":"Xencor","pharmaFlowCategory":"D","amount":"$215.0 million","upfrontCash":"Undisclosed","newsHeadline":"Xencor Sells Portion of Royalties and Milestones from Ultomiris\u00ae and Monjuvi\u00ae to OMERS Life Sciences for $215 Million","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"","productType":"Large molecule","productStatus":"Approved","date":"November 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"OMERS Life Sciences"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Takeda\u2019s ADZYNMA (ADAMTS13, recombinant-krhn) Approved by U.S. FDA as the First and Only Recombinant ADAMTS13 Enzyme Replacement Therapy for the Treatment of Congenital Thrombotic Thrombocytopenic Purpura (cTTP)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"November 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Takeda Pharmaceutical"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Kyowa Kirin","pharmaFlowCategory":"D","amount":"$477.6 million","upfrontCash":"$387.4 million","newsHeadline":"Kyowa Kirin Successfully Completes Acquisition of Orchard Therapeutics, a Global Gene Therapy Leader for Rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"January 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Jazz Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Jazz Pharmaceuticals Receives Health Canada Approval for Epidiolex\u00ae (Cannabidiol Oral Solution) for the Treatment of Seizures Associated with Three Rare Forms of Epilepsy","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"November 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Jazz Pharmaceuticals"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not 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Treatment of Adults with Neuromyelitis Optica Spectrum Disorder (NMOSD)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"March 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Takeda Announces Approval of ADZYNMA\u00ae Intravenous Injection 1500 (apadamtase alfa \/cinaxadamtase alfa) in Japan for Patients with Congenital Thrombotic Thrombocytopenic Purpura (cTTP)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"March 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Takeda Pharmaceutical"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"F. 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Hoffmann-La Roche","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Crovalimab Approved in China as the First Country, for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Chugai Pharmaceutical"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Harrow","pharmaFlowCategory":"D","amount":"$130.0 million","upfrontCash":"Undisclosed","newsHeadline":"Harrow Completes Transfer of the TRIESENCE\u00ae New Drug Application","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"November 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Novartis receives FDA Approval for Fabhalta\u00ae (iptacopan), offering Superior Hemoglobin Improvement in the Absence of Transfusions as the First Oral Monotherapy for Adults with PNH","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"VOYDEYA\u2122 Approved in the US as Add-On Therapy to Ravulizumab or Eculizumab for Treatment of Extravascular Hemolysis in Adults with the Rare Disease PNH","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"April 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"AstraZeneca"}]

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            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.

            Lead Product(s): Avalglucosidase Alfa

            Therapeutic Area: Rare Diseases and Disorders Product Name: Nexviazyme

            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 February 05, 2024

            Sanofi Company Banner

            BePharma 2024

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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

            Sanofi Company Banner

            BePharma 2024

            Not Confirmed

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            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

            Sanofi Company Banner

            BePharma 2024

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            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

            Sanofi Company Banner

            BePharma 2024

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            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

            Sanofi Company Banner

            BePharma 2024

            Not Confirmed

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            Voydeya (danicopan) is a first-in-class, oral medicine in development as an add-on to C5 inhibitor therapy eculizumab or ravulizumab for patients with paroxysmal nocturnal hemoglobinuria (PNH) who have residual haemolytic anaemia.

            Lead Product(s): Danicopan,Ravulizumab

            Therapeutic Area: Rare Diseases and Disorders Product Name: Voydeya

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 01, 2024

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            Adzynma (apadamtase alfa /cinaxadamtase alfa) is the first and only recombinant ADAMTS13 enzyme replacement therapy, which is approved for the treatment of congenital thrombotic thrombocytopenic purpura in patients 12 years of age and older.

            Lead Product(s): Apadamtase Alfa,Cinaxadamtase Alfa

            Therapeutic Area: Rare Diseases and Disorders Product Name: Adzynma

            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 26, 2024

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            Piasky (crovalimab) is a humanized complement inhibitor C5 monoclonal antibody. It is being developed for the treatment of adults and adolescents (12 years of age and above) with Paroxysmal Nocturnal Hemoglobinuria.

            Lead Product(s): Crovalimab

            Therapeutic Area: Rare Diseases and Disorders Product Name: Piasky

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: F. Hoffmann-La Roche

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 26, 2024

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            Ultomiris (ravulizumab) has been approved in the US as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder.

            Lead Product(s): Ravulizumab