Orphan Drugs Market 2019 - Major Participants- Research Covers Top 10 Players as Hoffmann- La Roche, Celgene Corporation, Alexion Pharmaceuticals, Inc., Novartis AG, Takeda Pharmaceuticals Company Limited, Biogen Idec Limited

This Report Provide to Pharmacological Class Protein Replacement, Enzyme Replacement, Gene and Chromosome Expression and also Provide Premium Applications Blood-Related Disorders, Oncology, Nephrology, Neurology and Infectious Diseases – Prime Repot Avail to MRFR

Competition Analysis:  

The key players operating the market for orphan drugs are Alexion Pharmaceuticals Inc., Bayer AG, Sanofi, Celgene Corporation, Novartis AG, Biogen Idec Limited, Hoffmann- La Roche, Takeda Pharmaceuticals Company Limited, Bristol-Myers Squibb, Vertex Pharmaceuticals Inc. Johnson & Johnson, and others.

Segmentation Analysis:

Globally, the market for orphan drugs has been segmented on the basis of application, pharmacological class, source, technology, and region.

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The market has been segmented into enzyme replacement, protein replacement, and gene chromosome expression based on pharmacological class.

The market has been segmented into oncology, blood-related disorders, neurology, nephrology, and infectious diseases based on applications. Among these, oncology is predicted to dominate the market owing to the increasing drug approvals for treating rare cancers.

The market has been segmented into Transgenic, DNA recombination, and gene expression based on technology.

The market has also been segmented into biological orphan drug and non-biological orphan drug based on source. Among these, biological orphan drug leads the market in terms of revenue and is predicted to maintain its dominance over the assessment period owing to the high efficacy of biologic products for treating diseases.

Global Orphan Drugs Market - Synopsis    

Orphan drugs are generally used for treating rare disorders and diseases that affect a small percentage of the population. There are approximately 30 million in Europe, 30 million people in the US, and 350 million people worldwide who suffer from 7,000 types of rare diseases.

Disorders among which four-fifths of the rare diseases are genetic and 50 % of the crowd affected being children. Only 5 % of the rare diseases had approved drug treatment with 326 new drugs being approved by FDA and brought to the market.

Future Drivers and Constraints Affecting the Market 

Growing investment in R&D, unmet requirement for rare diseases, economic, legislative, and tax benefits offered by the government, high ROI, development of latest technologies like hybridoma, gene mapping, and DNA recombinant, extensive exclusivity, and faster uptake and low marketing cost are some of the major factors driving the market over the forecast period.

There has been a growing demand for drugs for rare diseases such as Yellow Fever, Von Willebrand Disease, Aarskog Syndrome, Waardenburg Syndrome, diabetic macular edema, Abetalipoproteinemia, Autism spectrum disorder (ASD), Yellow Nail syndrome, Asperger syndrome, Von Hippel-Lindau Disease, and others which is contributing towards the growth of the market. Moreover, FDA free waivers, R&D grants by the government, favorable reimbursement policy along with premium pricing owing to the low competition also contribute the market growth. 

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Meanwhile, high cost of development, extensive post-market surveillance, costly clinical trials, monopolized and unitary market, concern over safety, competition from biosimilars and generics are some of the restraining factors inhibiting the growth of the market over the forecast period. Moreover, active distribution and commercialization can be hindered by differential availability of developing and developed countries, owing to socio-economic factors.

Global Orphan Drugs Market - Industry News                                              

The FDA has granted orphan drug designation to Surface Oncology's lead investigational candidate SRF231 which is a potential treatment for patients suffering from multiple myeloma. The SRF231 is a human monoclonal antibody therapeutic targeting CD47, a protein overexpressed on cancer cells to prevent them from being eliminated and engulfed by macrophage-mediated phagocytosis.

North America to Dominate the Market           

Geographically, the market for orphan drugs has been spanned across regions such as North America, Europe, Asia Pacific, Middle East and Africa.

North America has been estimated to hold the largest market share owing to well-established healthcare infrastructure, favorable regulations for drug approval for rare diseases, initiatives by non-governmental and governmental agencies to increase awareness related to use of orphan drugs, and drug exclusivity status received by orphan drugs. Moreover, US contributes substantially to the market which calls for strong R&D for development of orphan drugs.

Table of Contents  

1 Introduction  

1.1 Definition

1.2 Scope of Study

1.3 Research Objective

1.4 Assumptions & Limitations

1.5 Market Structure

2 Research Methodology

2.1 Research Process

2.2 Primary Research

2.3 Secondary Research

3 Market Dynamics

3.1 Drivers

3.2 Restraints

TOC Continued……..!

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