[{"orgOrder":0,"company":"Sanifit","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"First patient dosed in pivotal phase 3 trial of SNF472 in calciphylaxis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Sanifit"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AMRYT Announces Positive Top Line Results from Pivotal Phase 3 \"ease\" Trial of Filsuvez\u00ae in Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Amryt Pharma"},{"orgOrder":0,"company":"Sanifit","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sanifit Presents New Data for SNF472 in Treatment of Vascular Calcification at ASN Kidney Week 2020","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Sanifit"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amryt Announces Positive Results from Phase 3 Trial of Filsuvez\u00ae in Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Amryt Pharma"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios' Phase 3 ACTIVATE Trial of Mitapivat Met Primary Endpoint in Adults with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Agios Pharmaceuticals"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Announces Phase 3 ACTIVATE-T Trial of Mitapivat Achieved Primary Endpoint in Adults with Pyruvate Kinase Deficiency","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Agios Pharmaceuticals"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Priority Review for New Drug Application for Oleogel-S10 for the Treatment of Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Amryt Pharma"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Confirms NDA for Oleogel-S10 Will not Require an Advisory Committee Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Amryt Pharma"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amryt Announces Oleogel-S10 EMA Regulatory Update","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"January 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Amryt Pharma"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amryt Receives Complete Response Letter from the FDA for Oleogel-S10 NDA","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"February 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Amryt Pharma"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"University of the Witwatersrand","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Cyclo Therapeutics Enters Research and Collaboration Agreement with Wits Commercial Enterprise (Pty) Ltd to Explore Potential Applications of Trappsol\u00ae Cyclo\u2122 to Expand Pipeline","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"March 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Cyclo Therapeutics"},{"orgOrder":0,"company":"Abionyx Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ABIONYX Pharma Receives FDA Orphan Drug Designation (ODD) for CER-001 for the Treatment of LCAT Deficiency Presenting as Kidney Dysfunction and\/or Ophthalmologic Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Abionyx Pharma"},{"orgOrder":0,"company":"Savara","sponsor":"Silicon Valley Bank","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Savara Announces Debt Refinancing of $26.5M Credit Facility With Silicon Valley Bank","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Savara"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CHMP Adopts Positive Opinion for Filsuvez for the Treatment of Dystrophic and Junctional EB","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Amryt Pharma"},{"orgOrder":0,"company":"Savara","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Savara Awarded Innovation Passport in United Kingdom (UK) for Molgramostim Nebulizer Solution (Molgramostim), a Novel Investigational Inhaled Biologic","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Savara"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cyclo Therapeutics to Present at the 2022 NPC Patient and Family Conference Hosted by the Australian NPC Disease Foundation","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Cyclo Therapeutics"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharming Group Receives Accelerated Assessment in Europe for leniolisib for the Treatment of Rare Immunodeficiency, APDS","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharming"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Announces Publication of Phase 2 Data in The Lancet Demonstrating Safety and Efficacy of PYRUKYND\u00ae (mitapivat) in Non-transfusion-dependent \u03b1- and \u03b2-Thalassemia","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"August 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Agios Pharmaceuticals"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Acer Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Relief Therapeutics and Acer Therapeutics Announce that the European Commission Has Granted Orphan Drug Designation for ACER-001 in Maple Syrup Urine Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"August 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Relief Therapeutics"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chugai Gets Priority Review for PNH Med Crovalimab in China","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Chugai Pharmaceutical"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amgen Announces Positive Top-line Results From Phase 3 Study of ABP959, Biosimilar Candidate to Soliris\u00ae (Eculizumab)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Biosimilar","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Amgen Inc"},{"orgOrder":0,"company":"Savara","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Savara Presented Data at the European Respiratory Society (ERS) International Congress 2022","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Savara"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Danicopan (ALXN2040) Add-On to Ultomiris or Soliris Met Primary Endpoint In Alpha Phase III Trial For Patients With Paroxysmal Nocturnal Haemoglobinuria Who Experience Clinically Significant Extravascular Haemolysis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Danicopan (ALXN2040) Add-On to ULTOMIRIS\u00ae (ravulizumab-cwvz) or SOLIRIS\u00ae (eculizumab) Met Primary Endpoint in ALPHA Phase III Trial for Patients with Paroxysmal Nocturnal Hemoglobinuria Who Experience Clinically Significant Extravascular Hemolysis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Saol therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saol Therapeutics Announces Enrollment Achievement in Pivotal Phase 3 Trial for Rare Mitochondrial Disease - Pyruvate Dehydrogenase Complex Deficiency (PDCD)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Saol therapeutics"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alexion Aims To Advance NMOSD Treatment Landscape With Exceptional ULTOMIRIS\u00ae (ravulizumab-cwvz) Efficacy Data at ECTRIMS 2022","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alexion Aims To Advance NMOSD Treatment Landscape With Exceptional ULTOMIRIS (ravulizumab-cwvz) Efficacy Data At ECTRIMS 2022","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cyclo Therapeutics Announces Publication of Phase 1 Data for Trappsol\u00ae Cyclo\u2122 for the Treatment of Niemann-Pick Disease Type C1 (NPC1)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Cyclo Therapeutics"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alexion Aims To Advance NMOSD Treatment Landscape With Exceptional Ultomiris Efficacy Data At ECTRIMS 2022","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Novartis Investigational Oral Monotherapy Iptacopan Demonstrates Clinically Meaningful Superiority Over Anti-C5 Treatment in Phase III APPLY-PNH Study","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2022","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":"Ultomiris Showed Zero Relapses In Adults With Neuromyelitis Optica Spectrum Disorder (NMOSD) With Median Treatment Duration Of 73 Weeks","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"AstraZeneca"},{"orgOrder":0,"company":"IntraBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"IntraBio Reaches Target Enrollment for IB1001 NPC Pivotal Clinical Trial","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"IntraBio"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharming Group Announces Presentation Of New Leniolisib Data At 2022 ASH Annual Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharming"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Piper Sandler","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"X4 Pharmaceuticals Announces Proposed Public Offering","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"X4 Pharmaceuticals"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Piper Sandler","pharmaFlowCategory":"D","amount":"$65.0 million","upfrontCash":"Undisclosed","newsHeadline":"X4 Pharmaceuticals Prices $65.0 Million Public Offering","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"X4 Pharmaceuticals"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharming Announces Publication Of Data From Phase 3 Study Of Leniolisib In Patients With APDS in ASH's Blood","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharming"},{"orgOrder":0,"company":"IntraBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"IntraBio Completes Recruitment for IB1001 NPC Pivotal Clinical Trial","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"IntraBio"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Novartis Presents Pivotal Phase III APPLY-PNH Data at ASH Demonstrating Investigational Oral Monotherapy Iptacopan Superiority Over Anti-C5","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Roche\u2019s Subcutaneous Crovalimab Given Every Four Weeks Achieves Disease Control In People With PNH, a Life-threatening Blood Condition","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Novartis Investigational Iptacopan Provides Clinically Meaningful Increases In Hemoglobin Levels In Complement-inhibitor-na\u00efve Patients With PNH","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharming Announces Positive Interim Analysis Data from Open-Label Extension Study of Leniolisib in Presentation at The 64th American Society of Hematology (ASH) Annual Meeting and Exposition English","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharming"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"H.C. Wainwright & Co.","pharmaFlowCategory":"D","amount":"$4.2 million","upfrontCash":"Undisclosed","newsHeadline":"Cyclo Therapeutics Announces $4.2 Million Registered Direct Offering Priced At-The-Market Under Nasdaq Rules","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Cyclo Therapeutics"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"H.C. Wainwright & Co.","pharmaFlowCategory":"D","amount":"$4.2 million","upfrontCash":"Undisclosed","newsHeadline":"Cyclo Therapeutics Announces Closing of $4.2 Million Registered Direct Offering Priced At-The-Market under Nasdaq Rules","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Cyclo Therapeutics"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cyclo Therapeutics Announces Abstract Accepted for Poster Presentation at the Society for Inherited Metabolic Disorders (SIMD) 44th Annual Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Cyclo Therapeutics"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Takeda Announces Favorable Phase 3 Safety and Efficacy Results of TAK-755 as Compared to Standard of Care in Congenital Thrombotic Thrombocytopenic Purpura (cTTP)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Takeda Pharmaceutical"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cyclo Therapeutics Provides Clinical Program Update and Highlights Recent Achievements","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Cyclo Therapeutics"},{"orgOrder":0,"company":"Roche Diagnostics GmbH","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Roche Announces Positive Data from Global Phase III Programme for Crovalimab In PNH, A Rare Life-Threatening Blood Condition","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"GERMANY","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Roche Diagnostics GmbH"},{"orgOrder":0,"company":"Genentech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Genentech Announces Positive Data From Global Phase III Program for Crovalimab in PNH, a Rare, Life-Threatening Blood Condition","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Genentech"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chugai Announces Positive Data from Global Phase III Program for Crovalimab in PNH, a Rare Life-Threatening Blood Condition","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Chugai Pharmaceutical"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharming Provides Update on EMA Regulatory Review of Leniolisib For APDS in Europe","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharming"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Zevra Therapeutics Announces Two Abstracts Accepted for Presentation at the 19th Annual WORLDSymposium 2023","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Zevra Therapeutics"},{"orgOrder":0,"company":"Endo International","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Endo Provides Top-Line Results from Phase 2 Study of Collagenase Clostridium Histolyticum (CCH) in Participants With Plantar Fibromatosis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Endo International"},{"orgOrder":0,"company":"Samsung Bioepis","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Samsung Bioepis Receives Positive CHMP Opinion for Eculizumab Biosimilar, EPYSQLI\u2122","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"SOUTH KOREA","productType":"Large molecule","productStatus":"Biosimilar","date":"March 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Samsung Bioepis"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultomiris Recommended for Approval in The EU By CHMP For the Treatment of Adults with Neuromyelitis Optica Spectrum Disorder","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"Approved","date":"April 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"AstraZeneca"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Novartis Phase III APPOINT-PNH Trial Shows Investigational Oral Monotherapy Iptacopan Improves Hemoglobin to Near-Normal Levels, Leading to Transfusion Independence in All Treatment-Na\u00efve PNH Patients","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"U.S. Food & Drug Administration Grants Priority Review of TAK-755 for the Treatment of Congenital Thrombotic Thrombocytopenic Purpura (cTTP)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Takeda Pharmaceutical"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Govorestat (AT-007) Receives Orphan Medicinal Product Designation from the EMA for Treatment of SORD Deficiency","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Applied Therapeutics"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Pharmanovia","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Stealth BioTherapeutics Enters into Exclusive Licensing Agreement with Pharmanovia to Commercialize Elamipretide for the Treatment of Barth Syndrome in Europe and Key Global Territories","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Peptide","graph2":"Stealth Biotherapeutics"}]
Find Clinical Drug Development Pipelines & Deals | PipelineProspector
Pharmanovia will commercialize MTP-131 (elamipretide), an investigational mitochondrial protective agent, in Europe for the treatment of Barth syndrome, an ultra-rare cardiac condition caused by mitochondrial cardiolipin deficiency for which there are no approved treatments.
AT-007 (govorestat) is a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in development for the treatment of several rare neurological diseases, including Galactosemia, SORD Deficiency, and PMM2-CDG.
TAK-755 (apadamtase alfa) is the first and only recombinant ADAMTS13 protein in development. It provides targeted therapy to address an unmet medical need in patients with thrombotic thrombocytopenic purpura (TTP), by replacing the missing or deficient ADAMTS13 enzyme.
LNP023 (iptacopan) is an investigational first-in-class, orally administered targeted factor B inhibitor of the alternative complement pathway. It acts upstream of the C5 terminal pathway, preventing not only intravascular but also extravascular hemolysis in PNH.
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.
Epysqli™ (eculizumab) is a mAb that specifically binds to the complement protein C5, thereby inhibiting its cleavage to C5a and C5b and preventing the generation of the terminal complement complex C5b-9 mediated intravascular hemolysis in PNH patients.
EN3835 (collagenase clostridium histolyticum) is a collagenase enzyme used to promote debridement of necrotic tissue in burns and skin ulcers. It is being investigated for plantar fibromatosis.
Arimoclomol is believed to function by stimulating a normal cellular protein repair pathway through the activation of molecular chaperones. It is being developed as a treatment for Niemann-Pick disease type C (NPC).
Leniolisib is a small-molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K. PI3K delta is a valid and potentially effective therapeutic target for immune diseases such as APDS.
RG6107 (crovalimab) is an investigational, novel anti-C5 recycling monoclonal antibody designed to block the complement system which plays a vital part of the innate immune system that acts as the body’s first line of defence against infection.
Market Place
Upload your Marketing & Sales content on your company Virtual Booth, click HERE
