Table of Content

1.0 Executive Summary
2.1 USA (FDA) and European (EMA) Orphan Drug Approvals
2.2 Venture Funding Increasing Over Time
2.3 Therapeutic Areas Dominating Approval
2.4 Orphan Drug Designation Trends
2.5 Repurposing Existing Therapeutics
2.6 Japan Approval Trends
2.7 South Korea Approval Trends
2.8 China Approval Trends
3.0 Rare Disease Market Analysis
3.1 Major Orphan Drug Market to 2025
3.2 Orphan Small Molecule Market Analysis to 2025
3.3 Orphan Antibody Market Analysis to 2025
3.4 Orphan Gene Therapy Market Analysis to 2025
3.5 Orphan Cell Therapy Market Analysis to 2025
3.6 Orphan Oligonucleotide Therapy Market Analysis to 2025
3.7 Geographical Breakdown of Rare Disease Market to 2025
3.8 Orphan Drug Market by Indication and Disease Type
3.9 Key Market Players
3.9.1 Bristol-Myers Squibb
3.9.2 Roche/Genentech
3.9.3 Alexion
3.9.4 J&J/Janssen Biotech
3.9.5 Biogen
3.9.6 Novartis
3.9.7 AstraZeneca
3.9.8 Eisai
3.9.9 Takeda
3.9.10 Abbvie
3.9.11 Gilead
3.9.12 Pfizer
3.9.13 Vertex Pharmaceuticals
3.9.14 Argenix
4.0 Pipeline Agents in Clinical Trials by Condition, Intervention and Phase
4.1 Select Clinical Trials Investigating Small Molecules and Multiple Myeloma
4.2 Select Clinical Trials Investigating Gene and Cell Therapy in Melanoma
4.3 Select Clinical Trials Investigating Gene and Cell Therapy in ADA-SCID
4.4 Current Clinical Trials Investigating the RPE65 Genetic Retinal Mutation
4.5 Select Clinical Trials involving Rare Eye Disorders and Orphan Agents
4.6 Select Clinical Trials involving Spinal Muscular Atrophy and Orphan Agents
4.7 Select Clinical Trials involving Sickle Cell Anaemia and Orphan Agents
4.8 Select Clinical Trials involving Gene and Cell Therapy agents in Hemophilia A and B
4.9 Select Phase III Clinical Trials in Cystic Fibrosis
4.10 Select Clinical Trials involving Gene and Cell Therapy agents in Cystic Fibrosis
4.11 Select Clinical Trials involving CAR-T Therapy agents in Pancreatic Cancer
4.12 Select Clinical Trials involving CAR-T Therapy agents in Multiple Myeloma
4.13 Select Clinical Trials involving Gene and Cell Therapies in Duchenne Muscular
Dystrophy
4.14 Select Clinical Trials involving Oligonucleotide Therapies in Rare Diseases
4.15 Select Clinical Trials Investigating Therapeutics in Erythropoietic Protoporphyria
4.16 Select Clinical Trials Investigating Therapeutics in Guillain-Barre Syndrome
4.17 Select Phase III Clinical Trials Investigating Therapeutics in Scleroderma
4.18 Select Phase III Clinical Trials Investigating Therapeutics in Alpha-1 Antitrypsin
Deficiency
4.19 Select Phase III Clinical Trials Investigating Therapeutics in Juvenile Idiopathic Arthritis
5.0 Pipeline Therapeutics Analysis
6.0 Future Outlook

Exhibit 2.1: Number of FDA and EMA Orphan Drug Approvals from 2001-2018
Exhibit 2.2: FDA Priority, Accelerated, Breakthrough and Fast Track Regulatory Reviews for Rare
Disease from 2008-2017
Exhibit 2.3: EMA PRIME, Accelerated Assessment, Exceptional Circumstance or Conditional
Marketing Approval Reviews for Rare Disease from 2000-2018
Exhibit 2.4: Venture Funding into Rare Disorders, Excluding Oncology, 2013-2018
Exhibit 2.5: FDA Orphan Drug Approvals by Therapeutic Area
Exhibit 2.6: EMA Orphan Drug Approvals by Therapeutic Area
Exhibit 2.7: Number of FDA Approved Rare Disease Designations by Drug Type 2017-2020
Exhibit 2.8: Number of FDA Applications for Orphan Designation 2020
Exhibit 2.9: Number of Orphan FDA Designated Agents 20203
Exhibit 2.10: Percentage of FDA Applications for Orphan Designation 2020
Exhibit 2.11: FDA Approved Agents for Rare Disease Indications 2020
Exhibit 2.12: FDA Approved Agents for Rare Disease Indications 2019
Exhibit 2.13: FDA Approved Agents for Rare Disease Indications 2018
Exhibit 2.14: FDA Approved Agents for Rare Disease Indications 2017
Exhibit 2.15: EMA Approved Rare Disease Therapies to Date
Exhibit 3.1: Major Rare Disease Therapeutic Market by Drug Class 2018 - 2025
Exhibit 3.2 Major Rare Disease Therapeutic Market by Small Molecules, Antibodies, Gene
Therapies, Cell Therapies and Oligonucleotides, 2018-2025
Exhibit 3.3: Percentage Market Share of Rare Disease Drugs by Subtype, 2018 Vs 2025
Exhibit 3.4: Top Rare Disease Drug Sales ($ Billion) 2018
Exhibit 3.5: Top Rare Disease Drug Sales Forecast ($ Billion) 2025
Exhibit 3.6: Current Small Molecules Dominating the Rare Disease Market and Forecast to
2025
Exhibit 3.7: Future Small Molecules within the Rare Disease Market and Forecast to 2025
Exhibit 3.8: Small Molecule Orphan Drug Market 2018-2025
Exhibit 3.9: Current & Future Antibodies Dominating the Rare Disease Market and Forecast to
2025
Exhibit 3.10: Orphan Antibody Market Analysis 2018-2025
Exhibit 3.11: Current & Future Gene Therapies Dominating the Rare Disease Market and
Forecast to 2025
Exhibit 3.12: Orphan Gene Therapy Market Analysis 2018-2025
Exhibit 3.13: Current & Future CART Cell Therapies Dominating the Rare Disease Market and
Forecast to 2025
Exhibit 3.14: Orphan Cell Therapy Market Analysis 2018-2025
Exhibit 3.15: Current & Future Oligonucleotide Therapies Dominating the Rare Disease Market
and Forecast to 2025
Exhibit 3.16 Orphan Oligonucleotide Therapy Market Analysis 2018-2025
Exhibit 4.1: Select Clinical Trials Investigating Small Molecules and Multiple Myeloma
Exhibit 4.2: Select Phase III Clinical Trials Investigating Small Antibodies and Multiple Myeloma
Exhibit 4.3: Select Clinical Trials Investigating Gene and Cell Therapy in Melanoma
Exhibit 4.4: Select Clinical Trials Investigating Gene and Cell Therapy in ADA-SCID
Exhibit 4.5: Current Clinical Trials Investigating the RPE65 Genetic Retinal Mutation
Exhibit 4.6: Select Clinical Trials involving Rare Eye Disorders and Orphan Agents
Exhibit 4.7: Select Clinical Trials involving Spinal Muscular Atrophy and Orphan Agents
Exhibit 4.8: Select Clinical Trials involving Sickle Cell Anaemia and Orphan Agents
Exhibit 4.9: Select Clinical Trials involving Gene and Cell Therapy agents in Hemophilia A/B
Exhibit 4.10: Select Phase III Clinical Trials in Cystic Fibrosis
Exhibit 4.11: Select Clinical Trials involving Gene and Cell Therapy agents in Cystic Fibrosis
Exhibit 4.12: Select Clinical Trials involving CAR-T Therapy agents in Pancreatic Cancer
Exhibit 4.13: Select Clinical Trials involving CAR-T Therapy agents in Multiple Myeloma
Exhibit 4.14: Select Clinical Trials involving Gene and Cell Therapies in Duchenne Muscular
Dystrophy
Exhibit 4.15: Select Clinical Trials involving Oligonucleotide Therapies in Rare Diseases
Exhibit 4.16: Select Clinical Trials Investigating Therapeutics in Erythropoietic Protoporphyria
Exhibit 4.17: Select Clinical Trials Investigating Therapeutics in Guillain-Barre Syndrome
Exhibit 4.18: Select Phase III Clinical Trials Investigating Therapeutics in Scleroderma
Exhibit 4.19: Select Phase III Clinical Trials Investigating Therapeutics in Alpha-1 Antitrypsin
Deficiency
Exhibit 4.20: Select Phase III Clinical Trials Investigating Therapeutics in Juvenile Idiopathic
Arthritis
Exhibit 5.1: Total Number of Orphan Drugs in Phase I, II, III and NDA/BLA Clinical Programs
Exhibit 5.2: Number of Orphan Drugs (Oncology/Ex-Oncology) in Phase I, II, III and NDA/BLA
Clinical Programs
Exhibit 5.3: Number of Therapies in Development for Rare Diseases
Exhibit 5.4: Top Selling Orphan Drugs Forecast to 2025
Exhibit 5.5: Top Orphan Drugs by Type and Mode of Action by 2025