NEW YORK, Fev. 1, 2018 /PRNewswire/ — Read the full report: https://www.reportlinker.com/p05304553
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