CSBio CSBio

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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 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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 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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 Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"New Data Demonstrate Clinical Safety, Benefit and Durability of Nexviazyme\u00ae Across a Wide-Range of Pompe Disease Patient Groups","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Voydeya Recommended for Approval in the EU by CHMP as Add-On Treatment to Ravulizumab or Eculizumab for Adults with Pnh Who Have Residual Haemolytic Anaemia","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"February 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

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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

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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

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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

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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

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            TMF Summit 2024

            Not Confirmed

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            Voydeya (danicopan) is an investigational 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 February 26, 2024

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            Through the acquisition, Kyowa expands its portfolio, enables the development of promising candidates with a clinically differentiated platform, and helps resource the ongoing and future launches of Libmeldy (atidarsagene autotemcel) for metachromatic leukodystrophy.

            Lead Product(s): Atidarsagene Autotemcel

            Therapeutic Area: Rare Diseases and Disorders Product Name: Libmeldy

            Highest Development Status: Approved Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Kyowa Kirin

            Deal Size: $477.6 million Upfront Cash: $387.4 million

            Deal Type: Acquisition January 24, 2024

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            Epidiolex (cannabidiol) is an oral CB receptor CB1 receptor negative allosteric modulator. It is now approved for the treatment of seizures associated with Lennox-Gastaut syndrome & Dravet syndrome in patients 2 years of age and older.

            Lead Product(s): Cannabidiol

            Therapeutic Area: Rare Diseases and Disorders Product Name: Epidiolex

            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 November 23, 2023

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            ADZYNMA (ADAMTS13, recombinant-krhn) for the prophylactic and on-demand treatment of adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP).

            Lead Product(s): ADAMTS13 Recombinant-krhn

            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 November 09, 2023

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            OMERS Life Sciences

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            OMERS has acquired royalties for Ultomiris (ravulizumab-cwvz), a humanized monoclonal antibody complement inhibitor for paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome, and Monjuvi (tafasitamab-cxix), used in combination with lenalidomide.

            Lead Product(s): Ravulizumab

            Therapeutic Area: Rare Diseases and Disorders Product Name: Ultomiris

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Xencor

            Deal Size: $215.0 million Upfront Cash: Undisclosed

            Deal Type: Divestment November 07, 2023

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