Product and Pipeline Assessment of the Global Orphan Drugs Market

Attractive Market Lures Hundreds of Innovators, Large and Small

Regular Price: USD 3,950

Special Price USD 3,555

10% OFF

* Required Fields

Regular Price: USD 3,950

Special Price USD 3,555

PAY BY INVOICE

Be the first to review this product

Breakthroughs in drug discovery have led to rich and competitive pipelines for rare diseases such as rare cancers as well as neurologic disorders and musculoskeletal disorders, among many other therapeutic areas. This research service provides a comprehensive global overview and analysis of global orphan drugs development, with an emphasis on the US market. Included are market background and global overview and analysis of drug development and regulatory activity by disease area and geography, detailed product and pipeline analysis of orphan drugs by therapeutic area and disease, and company analysis, including activity analysis and key companies to watch.

Table of Contents

Executive SummaryExecutive SummaryExecutive Summary (continued)Methodology and ScopeMethodology and ScopeMarket BackgroundMarket BackgroundMarket Background (continued)Market Background (continued)NIH Orphan Diseases by TypeNIH Orphan Diseases by Type (continued)Global OverviewGlobal Overview and AnalysisGlobal Overview and Analysis (continued)US and European MarketsOrphan Diseases with FDA-approved TherapiesOrphan Diseases with FDA-approved Therapies (continued)Orphan Diseases with FDA-approved Therapies (continued)Orphan Diseases with FDA-approved Therapies (continued)Orphan Diseases with FDA-approved Therapies (continued)Orphan Diseases with FDA-approved Therapies (continued)Orphan Diseases with FDA-approved Therapies (continued)FDA Approvals and Designations by Therapeutic AreaFDA Orphan Designations by Therapeutic AreaEMA Orphan Designations by Therapeutic AreaCount of US FDA and European EMA Orphan Drug Designations by YearCount of US FDA and European EMA Orphan Drug Approvals by YearCount of US FDA Orphan Drug Designations and Approvals by YearCount of European EMA Orphan Drug Designations and Approvals by YearTop Rare Cancer DesignationsTop Rare Cancer Designations (continued)Annual Count of FDA Rare Cancer DesignationsAnnual Count of FDA Pediatric Orphan DesignationsPipeline Analyses for Select Rare Diseases—Blood and Lymphatic System DiseasesTop Indications by Activity and Disease OverviewSCD—Orphan Drugs on the MarketSCD—Orphan Drugs in DevelopmentSCD—Orphan Drugs in Development (continued)MDS—Orphan Drugs on the MarketMDS—Orphan Drugs in DevelopmentTTP—Orphan Drugs in DevelopmentPipeline Analyses for Select Rare Diseases—Cardiovascular DiseasesTop Indications by Activity and Disease OverviewPAH—Orphan Drugs on the MarketPAH—Orphan Drugs in Development PAH—Orphan Drugs in Development (continued)Calciphylaxis—Orphan Drugs in DevelopmentPipeline Analyses for Select Rare Diseases—Lung DiseasesTop Indications by Activity and Disease OverviewCF—Orphan Drugs on the MarketCF—Orphan Drugs in Development CF—Orphan Drugs in Development (continued) CF—Orphan Drugs in Development (continued) CF—Orphan Drugs in Development (continued) IPF—Orphan Drugs on the MarketIPF—Orphan Drugs in Development IPF—Orphan Drugs in Development (continued) IPF—Orphan Drugs in Development (continued) Pipeline Analyses for Select Rare Diseases—Congenital and Genetic DiseasesTop Indications by Activity and Disease OverviewFragile X Syndrome—Orphan Drugs in Development HIBM—Orphan Drugs in DevelopmentPipeline Analyses for Select Rare Diseases—Connective Tissue/Skin DiseasesTop Indications by Activity and Disease OverviewSSc—Orphan Drugs in DevelopmentEB—Orphan Drugs in DevelopmentPipeline Analyses for Select Rare Diseases—Digestive/Nutritional DiseasesTop Indications by Activity and Disease OverviewPed CD—Orphan Drugs on the MarketPed CD—Orphan Drugs in DevelopmentPed CD—Orphan Drugs in Development (continued)Ped UC—Orphan Drugs on the MarketPed UC—Orphan Drugs in DevelopmentPed UC—Orphan Drugs in Development (continued)Pouchitis—Orphan Drugs in Development Pipeline Analyses for Select Rare Diseases—Endocrine DiseasesTop Indications by Activity and Disease OverviewT1D with Residual Beta-cell Function—Orphan Drugs in Development T1D with Residual Beta-cell Function—Orphan Drugs in Development (continued)Pipeline Analyses for Select Rare Diseases—Exposure/ToxicityTop Indications by Activity and Disease OverviewARS—Orphan Drugs in Development ARS—Orphan Drugs in Development (continued)Pipeline Analyses for Select Rare Diseases—Eye DiseasesTop Indications by Activity and Disease OverviewRP—Orphan Drugs in DevelopmentRP—Orphan Drugs in Development (continued)Stargardt Disease—Orphan Drugs in DevelopmentUveitis—Orphan Drugs on the MarketUveitis—Orphan Drugs in DevelopmentPipeline Analyses for Select Rare Diseases—Immunological/Inflammatory DiseasesTop Indications by Activity and Disease OverviewBehcet's Disease—Orphan Drugs in DevelopmentPipeline Analyses for Select Rare Diseases—Infectious/Parasitic DiseasesTop Indications by Activity and Disease OverviewCMV Infection—Orphan Drugs on the MarketCMV Infection—Orphan Drugs in DevelopmentMalaria—Orphan Drugs on the MarketMalaria—Orphan Drugs in DevelopmentLeishmaniasis—Orphan Drugs on the MarketLeishmaniasis—Orphan Drugs in Development Pipeline Analyses for Select Rare Diseases—Metabolic DiseasesTop Indications by Activity and Disease OverviewTop Indications by Activity and Disease Overview (continued)MPS—Orphan Drugs on the MarketMPS—Orphan Drugs in DevelopmentMPS—Orphan Drugs in Development (continued)Acromegaly—Orphan Drugs on the MarketAcromegaly—Orphan Drugs in DevelopmentUrea Cycle Disorders—Orphan Drugs on the MarketUrea Cycle Disorders—Orphan Drugs in DevelopmentNPC Disease—Orphan Drugs on the MarketNPC Disease—Orphan Drugs in DevelopmentPompe Disease—Orphan Drugs on the MarketPompe Disease—Orphan Drugs in DevelopmentPipeline Analyses for Select Rare Diseases—Musculoskeletal DiseasesTop Indications by Activity and Disease OverviewDMD—Orphan Drugs in DevelopmentDMD—Orphan Drugs in Development (continued)DMD—Orphan Drugs in Development (continued)DMD—Orphan Drugs in Development (continued)SMA—Orphan Drugs in DevelopmentPipeline Analyses for Select Rare Diseases—Neurologic DiseasesTop Indications by Activity and Disease OverviewALS—Orphan Drugs in DevelopmentALS—Orphan Drugs in Development (continued)ALS—Orphan Drugs in Development (continued)ALS—Orphan Drugs in Development (continued)HD—Orphan Drugs on the MarketHD—Orphan Drugs in DevelopmentFA—Orphan Drugs in DevelopmentPipeline Analyses for Select Rare Diseases—Rare CancersTop Indications by Activity and Disease OverviewPancreatic Cancer—Orphan Drugs on the MarketPancreatic Cancer—Orphan Drugs in DevelopmentPancreatic Cancer—Orphan Drugs in Development (continued)Pancreatic Cancer—Orphan Drugs in Development (continued)Pancreatic Cancer—Orphan Drugs in Development (continued)Pancreatic Cancer—Orphan Drugs in Development (continued)Pancreatic Cancer—Orphan Drugs in Development (continued)Glioma—Orphan Drugs on the MarketGlioma—Orphan Drugs in DevelopmentGlioma—Orphan Drugs in Development (continued)Glioma—Orphan Drugs in Development (continued)Glioma—Orphan Drugs in Development (continued)Glioma—Orphan Drugs in Development (continued)AML—Orphan Drugs on the MarketAML—Orphan Drugs in DevelopmentAML—Orphan Drugs in Development (continued) AML—Orphan Drugs in Development (continued) AML—Orphan Drugs in Development (continued) AML—Orphan Drugs in Development (continued) Company AnalysisTop 20 Companies by ActivityTop Companies by Activity per Therapeutic AreaTop Earning Orphan DrugsSelect Recent LaunchesCompanies to Watch—Alexion PharmaceuticalsCompanies to Watch—BioMarin Pharmaceutical Inc.Companies to Watch—Celgene CorporationCompanies to Watch—PTC TherapeuticsCompanies to Watch—Spotlight on InnovationStrategic Partnership AssessmentStrategic Partnership Assessment (continued)Strategic Partnership Assessment (continued)ConclusionsConclusions and RecommendationsLegal DisclaimerAppendixList of Rare Cancers in “Others”List of Rare Cancers in “Others” (continued)List of Rare Cancers in “Others” (continued)The Frost & Sullivan StoryThe Frost & Sullivan StoryValue Proposition: Future of Your Company & CareerGlobal PerspectiveIndustry Convergence360º Research PerspectiveImplementation ExcellenceOur Blue Ocean Strategy




Keyword1

Keyword2

Keyword3

Related Research

Release Date : 08-Dec-17

Region : Asia Pacific

Release Date : 06-Dec-17

Region : Asia Pacific

Release Date : 27-Nov-17

Region : Asia Pacific

Why Frost & Sullivan

Working with the CEO’s growth team to create a vision based on a transformation growth strategy

Creating content-based digital marketing strategies that leverage our research perspective to differentiate and “tell your story”

Tracking over 1000 emerging technologies and analyzing the impact by industry and application to reveal the companies to watch in each sector

The Frost & Sullivan team is based in our 45 global offices and have developed a powerful global understandings of how industries operate on a global level.