[{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Approves First Treatment for Adult Onset Still\u2019s Disease, a Severe and Rare Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"SWITZERLAND","productType":"Large molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"GSK","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GlaxoSmithKline's Nucala Filings Accepted by European Medicines Agency for Three Additional Eosinophil-Driven Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"Approved","date":"October 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"GSK"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"NICE Issues Positive Recommendation for Use of Kineret to Treat Still\u2019s Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"SWEDEN","productType":"Large molecule","productStatus":"Approved","date":"March 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ilaris\u00ae Receives Health Canada Approval as First of Its Kind Treatment for Rare, Inflammatory Disorder, Adult-Onset Still's Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"SWITZERLAND","productType":"Large molecule","productStatus":"Approved","date":"April 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Strongbridge Biopharma","sponsor":"Xeris Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Xeris Pharmaceuticals, Inc. to Acquire Strongbridge Biopharma in Stock and CVR Transaction","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"May 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Strongbridge Biopharma"},{"orgOrder":0,"company":"Enzyvant","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Enzyvant Receives FDA Approval for RETHYMIC\u00ae (allogeneic processed thymus tissue-agdc), a One-Time Regenerative Tissue-Based Therapy for Pediatric Congenital Athymia","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"October 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Enzyvant"},{"orgOrder":0,"company":"Kedrion","sponsor":"Kedrion","pharmaFlowCategory":"D","amount":"$17.0 million","upfrontCash":"Undisclosed","newsHeadline":"Kedrion Biopharma Grows in North America As it Completes Acquisition of Prometic","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"October 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Kedrion"},{"orgOrder":0,"company":"Kedrion","sponsor":"Kedrion","pharmaFlowCategory":"D","amount":"$17.0 million","upfrontCash":"Undisclosed","newsHeadline":"Kedrion enters into Agreement with Liminal BioSciences","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"May 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Kedrion"},{"orgOrder":0,"company":"GSK","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"European Commission Approves Nucala (Mepolizumab) in Three Additional Eosinophil-Driven Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"Approved","date":"November 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"GSK"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"New Analysis Published in Multiple Sclerosis and Related Disorders Supports the Efficacy of UPLIZNA\u00ae (Inebilizumab-cdon) in Patients Previously Treated with Rituximab for Neuromyelitis Optica Spectrum Disorder (NMOSD)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Large molecule","productStatus":"Approved","date":"November 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Horizon Therapeutics"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"BridgeBio Pharma and Sentynl Therapeutics Announce Asset Purchase Agreement for BridgeBio Pharma\u2019s NULIBRY\u2122 (Fosdenopterin)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"BridgeBio Pharma"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Marinus Pharmaceuticals Announces FDA Approval of ZTALMY\u00ae (ganaxolone) for CDKL5 Deciency Disorder","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Marinus Pharmaceuticals"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Xenpozyme\u00ae (olipudase alfa), Approved in Japan, First and Only Approved Therapy Indicated to Treat Acid Sphingomyelinase Deficiency","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"March 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"New Analysis Finds UPLIZNA\u00ae (inebilizumab-cdon) Effective for People with Newly-Presenting Neuromyelitis Optica Spectrum Disorder (NMOSD)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Large molecule","productStatus":"Approved","date":"March 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Horizon Therapeutics"},{"orgOrder":0,"company":"Nobelpharma America","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Approves Nobelpharma's HYFTOR\u2122 (sirolimus topical gel) 0.2%","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Nobelpharma America"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA approves Novartis Vijoice\u00ae (alpelisib) as First and Only Treatment for Select Patients With PIK3CA-Related Overgrowth Spectrum (PROS)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Marinus Pharmaceuticals Announces Publication in The Lancet Neurology of ZTALMY\u00ae (ganaxolone) Phase 3 Marigold Trial Results","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Marinus Pharmaceuticals"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Horizon Therapeutics plc Receives European Commission (EC) Approval of UPLIZNA\u00ae (inebilizumab) for the Treatment of Adults With Neuromyelitis Optica Spectrum Disorder (NMOSD)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Large molecule","productStatus":"Approved","date":"May 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Horizon Therapeutics"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultomiris Met Primary Endpoint in CHAMPION-NMOSD Phase III Trial in Adults With Neuromyelitis Optica Spectrum Disorder","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"Approved","date":"May 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"AstraZeneca"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CHMP Recommends Approval of Xenpozyme\u00ae (Olipudase Alfa), the First and Only Treatment for ASMD","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"May 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Zenyaku Kogyo","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Anti-CD20 Monoclonal Antibody Rituxan Approved for the Prevention of Recurrence of Neuromyelitis Optica Spectrum Disorder","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Zenyaku Kogyo"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"European Commission Approves Filsuvez\u00ae for the treatment of Dystrophic and Junctional EB","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Amryt Pharma"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Xenpozyme\u00ae (olipudase alfa) Approved by European Commission as First and Only treatment for ASMD","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amryt Presents New Analyses from the EASE Phase 3 Trial in Epidermolysis Bullosa at SPD 2022","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Amryt Pharma"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Kyowa Kirin","pharmaFlowCategory":"D","amount":"$500.0 million","upfrontCash":"Undisclosed","newsHeadline":"Ultragenyx Announces Sale of a Portion of Future North American Royalties on Crysvita (burosumab) for $500 Million to OMERS Capital Markets","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Upstaza\u2122 Granted Marketing Authorization by European Commission as First Disease-Modifying Treatment for AADC Deficiency","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"PTC Therapeutics"},{"orgOrder":0,"company":"Nobelpharma America","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Nobelpharma America Debuts New Product, HYFTOR\u2122 (sirolimus topical gel) 0.2%, at the 2022 World TSC Conference","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Nobelpharma America"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma and Sentynl Therapeutics Receive Positive CHMP Opinion for NULIBRY (fosdenopterin) for the Treatment of MoCD Type A","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"BridgeBio Pharma"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Xenpozyme TM (Olipudase Alfa-rpcp) Approved by FDA as First Disease-specific Treatment for ASMD (Non-CNS Manifestations)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Nobelpharma America","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Hyftor\u2122 (Sirolimus Topical Gel) 0.2%, the First FDA-approved Topical Treatment for Facial Angiofibroma Associated With Tuberous Sclerosis, is Now Available in the US","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"August 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Nobelpharma America"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma and Sentynl Therapeutics Receive Marketing Authorization in the EU for NULIBRY\u00ae (fosdenopterin) for the Treatment of MoCD Type A","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"BridgeBio Pharma"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Horizon Therapeutics plc Announces New UPLIZNA\u00ae (inebilizumab-cdon) Data in Neuromyelitis Optica Spectrum Disorder (NMOSD) to be Presented at ECTRIMS 2022","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Large molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Horizon Therapeutics"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"The British Journal of Dermatology Publishes Results from Amryt\u2019s EASE Phase 3 Trial in Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Amryt Pharma"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"New Analysis of UPLIZNA\u00ae (inebilizumab-cdon) Phase 3 Trial Data Demonstrates Importance of Reducing Plasmablasts to Help Prevent Neuromyelitis Optica Spectrum Disorder (NMOSD) Attacks","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Large molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Horizon Therapeutics"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AstraZeneca Expands Global Footprint In Rare Disease With Availability Of First Alexion Rare Disease Therapy For Patients In China","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"Approved","date":"November 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"AstraZeneca"},{"orgOrder":0,"company":"Altavant Sciences","sponsor":"Enzyvant Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Enzyvant Announces Merger with Altavant","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Approved","date":"December 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Altavant Sciences"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Brazilian Regulatory Authority Grants Approval for Waylivra\u2122 for Familial Partial Lipodystrophy","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"December 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"PTC Therapeutics"},{"orgOrder":0,"company":"Apellis Pharmaceuticals","sponsor":"Swedish Orphan Biovitrum AB","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sobi Receives Approval from Health Canada for Empaveli\u2122 (pegcetacoplan) for the Treatment of Certain Patients with Paroxysmal Nocturnal Hemoglobinuria","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Peptide","productStatus":"Approved","date":"December 2022","url1":"","url2":"","graph1":"Peptide","graph2":"Apellis Pharmaceuticals"},{"orgOrder":0,"company":"ANI Pharmaceuticals Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ANI Pharmaceuticals Announces the FDA Approval and Launch of Levocarnitine Tablets USP","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"ANI Pharmaceuticals Inc"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"UPLIZNA\u00ae (inebilizumab-cdon) Approved in Brazil for the Treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Large molecule","productStatus":"Approved","date":"December 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Horizon Therapeutics"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Analysis from the Phase 3 N-MOmentum Study Demonstrates the Effectiveness of UPLIZNA\u00ae (inebilizumab-cdon) Among Neuromyelitis Optica Spectrum Disorder Patients with Genetic Variations","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Large molecule","productStatus":"Approved","date":"May 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Horizon Therapeutics"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Chiesi Farmaceutici","pharmaFlowCategory":"D","amount":"$1,480.0 million","upfrontCash":"$1,250.0 million","newsHeadline":"Chiesi Farmaceutici S.p.A. to Acquire Amryt Pharma Plc","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"January 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Amryt Pharma"}]
Find Clinical Drug Development Pipelines & Deals | PipelineProspector
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.
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.
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.
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.
Filsuvez (oleogel-S10), a topical therapeutic gel, is a potential treatment for the cutaneous manifestations of Epidermolysis Bullosa, a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment.
UPLIZNA (inebilizumab-cdon), is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.
ANI’s Levocarnitine Tablets are the generic version of the Reference Listed Drug (RLD) Carnitor®. The launch of Levocarnitine Tablets is another example of ANI’s commitment to increasing patient access to affordable, high-quality medicines.
Empaveli™ having pegcetacoplan, is a targeted C3 therapy designed to regulate the activity of the complement cascade, which can lead to the onset and progression of many serious diseases.
RETHYMIC® (allogeneic processed thymus tissue-agdc) is a novel one-time tissue-based regenerative therapy used for immune reconstitution in pediatric patients with congenital athymia.
Lead Product(s):
Allogeneic Processed Thymus Tissue-agdc
Waylivra (volanesorsen), an antisense oligonucleotide designed to inhibit the formation of apoC-III, is a product of Ionis Pharmaceuticals, Inc.'s proprietary antisense technology. Waylivra has received conditional marketing approval in the EU as a treatment for FCS.
Market Place
Company
Pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme. 
Contact Supplier
