Global Genetic Disorders Drugs Market outlook 2023: Cystic Fibrosis (CF), Duchenne Muscular Dystrophy (DMD), Lysosomal Storage Disease (LSD), Paroxysmal Nocturnal Hemoglobinuria (PNH)

Press Release

The genetic disorders therapy area consists of indications that arise because of an abnormality in a person’s DNA. This report focuses on the key therapy area indications of cystic fibrosis (CF), Duchenne muscular dystrophy (DMD), lysosomal storage disease (LSD) and paroxysmal nocturnal hemoglobinuria (PNH).

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Genetic disorders are frequently inherited from an individual’s parents but can also arise because of a new (de novo) mutation. The treatment landscape for genetic disorders is dominated commercially by orphan drugs for the treatment of rare genetic disorders.

Overall, 1,390 products are being actively developed in the genetic disorders pipeline. The pipelines of CF, DMD, LSD and PNH contain 163, 109, 174 and 24 products, respectively. The pipeline is dominated by targeted therapies that aim to treat the underlying cause, which is often specific to the particular indication.


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?

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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.”

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