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Rare Disease & Orphan Therapeutic Market Analysis & Forecast to 2025

Rare Disease & Orphan Therapeutic Market Analysis & Forecast to 2025

  • January 2021
  • 197 pages
  • ID: 6000014
  • Format: PDF
  • Kelly Scientific Publications


Table of Contents

This industry analysis report tackles the major orphan drugs within the rare disease therapeutic market and encompasses the most prevalent and dominant therapies in this space. The market value for the current major orphan therapeutic space is analysed and forecast to 2025 with corresponding CAGR percentages. Market details are provided by drug type, therapeutic area, geography and also specific sales and forecast of the major drugs in the field. Key industry players and developments are covered along with the growing pipeline and clinical trials. Regional insights and approval trends are given, as are the strengths, opportunities and challenges of this growing market.

The scope of the report provides in-depth analysis of the therapeutics space by value and forecast to 2025 that is sub divided into:
• Small molecules
• Antibodies
• Oligonucleotides
• Gene therapy
• Cell therapy

The report reviews the most dominant therapeutics in each sub-market and provides clinical evaluation, sales forecast and market share to 2025. It gives a comparison between the current market values of the main therapies and how the landscape will change by 2025 with the launch of new pipeline agents. This includes some of the main blockbusters such as:

• Revlimid
• Trikafta
• Darzalex
• Spinraza
• Hemlibra
• Venclexta
• Soliris
• Adcetris
• Jakafi
• Lenvima
• Lynparza
• Ultomiris
• Yescarta
• Pomalyst
• Vyndaqel
• Ninlaro
• Liso-cel

Therefore the reader is provided with data on the current main dominant therapeutics in the area and also the drugs that will dominate the space by 2025. This analysis is provided for each sub-therapeutic area. Geographical breakdown analysis is further provided and is segmented into North America, Europe, Asia Pacific and the Rest of the World.

The scope of the report also includes the orphan drug market by indication and disease type with specific detail given to oncology, hematological disorders, CNS, respiratory, immunomodulation, cardiovascular, musculoskeletal, anti-infectives, endocrine and gastrointestinal disease.

Key players and company profiles in the rare disease market are provided including:
• Bristol Myers Squibb
• Roche/Genentech
• Alexion
• Janssen J&J
• Biogen
• Novartis
• AstraZeneca Merck
• Eisai
• Takeda
• Abbvie Genentech
• Gilead (Kite)
• Pfizer
• Vertex
• Argenx

The report also includes a detailed description of the following trends and market shapers:
• USA (FDA) and European (EMA) Orphan Drug Approvals
• Venture Funding Increasing Over Time
• Therapeutic Areas Dominating Approval
• Orphan Drug Designation Trends
• Repurposing Existing Therapeutics
• Japan Approval Trends
• South Korea Approval Trends
• China Approval Trends

The growing rare disease pipeline and clinical trial environment is evaluated with specific consideration given to:
• Small Molecules and Multiple Myeloma
• Gene and Cell Therapy in Melanoma
• Gene and Cell Therapy in ADA-SCID
• Gene and Cell Therapy agents in Hemophilia A and B
• CAR-T Therapy agents in Pancreatic Cancer
• CAR-T Therapy agents in Multiple Myeloma
• Gene and Cell Therapies in Duchenne Muscular Dystrophy
• RPE65 Genetic Retinal Mutation
• Rare Eye Disorders 
• Spinal Muscular Atrophy 
• Sickle Cell Anaemia 
• Cystic Fibrosis
• Erythropoietic Protoporphyria
• Guillain-Barre Syndrome
• Scleroderma
• Alpha-1 Antitrypsin Deficiency
• Juvenile Idiopathic Arthritis

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