COVID-19 update included
The content of this report will be updated with the latest scenarios based on the global COVID-19 Pandemic

Genetic disorders are caused by abnormalities in a person’s genome and represent a broad therapy area, with over 200 individual diseases in the pipeline, including cystic fibrosis (CF), Duchenne muscular dystrophy (DMD), lysosomal storage disease (LSD) and paroxysmal nocturnal hemoglobinuria (PNH). Many genetic disorders are progressive, debilitating and/or life threatening, with a severe need of treatment.

In the US, an orphan drug is defined as a drug intended to treat a disease affecting fewer than 200,000 people. Orphan drugs are able to generate high revenue because of developmental incentives such as marketing exclusivity, which allows for longer periods before generics and biosimilars can enter the market. Furthermore, other incentives such as tax credits and accelerated approval lower the developmental costs faced by pharmaceutical companies, reducing the inherent financial risks involved and increasing the likelihood of recouping developmental costs from product sales.

Currently there are 1,390 products in active development for the treatment of genetic disorders, making this the sixth largest therapy area in terms of size. The key indications of LSD, CF and DMD have the highest levels of pipeline activity, with 174, 163 and 109 products in development respectively. While PNH has a relatively small pipeline with just 24 products in development, this indication includes Soliris, which generated revenue of $2.8 billion in 2016 and is anticipated to be the best-selling treatment within genetic disorders throughout the forecast period.

Soliris, the best-selling drug within genetic disorders in 2016, is anticipated to remain the best-selling therapy within this cluster throughout the forecast period. The key patents for Soliris extend until 2021 in the US, and while there are Soliris biosimilars in development, they are not expected to enter the market during the forecast period. This lack of alternative treatments – as well as the rarity and life-threatening nature of the treated diseases – allow for high treatment costs and the generation of substantial revenue, despite the low prevalence of the diseases.

The report "Global Genetic Disorders Drugs Market to 2023 - A Rapidly Growing Treatment Landscape Driven by Targeted Complement System Inhibitors and Enzyme Replacement Therapies to Treat PNH and Lysosomal Storage Disease" provides an introduction to the genetic disease cluster, and some detailed background information regarding the key indications cystic fibrosis, Duchenne muscular dystrophy, lysosomal storage disease and paroxysmal nocturnal hemoglobinuria. Information is also included regarding symptoms, etiology and pathophysiology, comorbidities and epidemiology.

In depth, this report provides the following -

  • Provides an overview of the genetic disorders marketed products landscape, including product profiles of key marketed products, and revenue projections for each of these drugs.
  • Analyzes the genetic disorders pipeline, detailing, among other parameters, drug distribution by stage of development, molecule type and molecular target, along with an analysis of the key indications cystic fibrosis, Duchenne muscular dystrophy, lysosomal storage disease and paroxysmal nocturnal hemoglobinuria.
  • Presents forecast projections to 2023 on a global level, with forecasts provided for the US, plus aggregate revenue forecasts of the top five EU markets (the UK, France, Germany, Italy and Spain) and Japan. Additionally, revenue forecasts for the major product categories and key players are provided.
  • Assesses the company landscape for the disease cluster, with an analysis of the key players, the types of company involved, and whether the disease cluster comprises a large proportion of each company’s pipeline portfolio and total revenues.
  • Analyses trends in co-development and licensing deals relating to genetic disorder products. Some of the most prominent deals are discussed in detail.


  • Global revenue from the genetic disorders market is forecast to increase from $19.6 billion in 2016 to $47.7 billion in 2023, at a compound annual growth rate of 13.55%. What is driving this growth?
  • The leading companies in terms of market share are Alexion, Sanofi, Shire and Vertex. Which of these are forecast to experience the largest growth?
  • There are new approvals and late-stage products set to enter the market during the forecast period. Which drugs will achieve blockbuster status?
  • The market has been dominated by complement system inhibitors and enzyme replacement therapies. To what extent will these classes of drugs and others dominate the market over the forecast period?
  • There are 1,390 genetic disorders products in the pipeline. What molecular targets are most abundant in the pipeline and what role will pipeline product approvals play in market growth?
  • Genetic disorders clinical trials have an overall attrition rate of around 93%, what can companies do to maximize their chance of success?

Reasons To Buy

  • Understand the current treatment landscape, with portfolios of key marketed products and a focus on historical and forecast sales patterns, and an overview of each drug’s mechanism of action.
  • Analyze the genetic disorders pipeline through a comprehensive review of the pipeline segmented by stage of development, molecule type and molecular target. This review also provides a detailed look at products for the treatment of genetic disorders to provide an insight into the risk associated with attempting to bring pipeline products to market.
  • Predict growth in market size, with in-depth market forecasting from 2016 to 2023. The forecasts will provide an understanding of how epidemiology trends, new drug entries, and patent expirations will influence market value.
  • Identify commercial opportunities in the genetic disorders deals landscape by analyzing trends in licensing and co-development deals.