NEW YORK (GBI Research), 21 May 2012 - Groundbreaking therapies are on the horizon as scientists explore new treatment options for rare genetic disorders, according to a new report by healthcare experts GBI Research.
The new report looks at Cystic Fibrosis (CF), Duchenne Muscular Dystrophy (DMD), Fabry disease and Pompe disease, which are all chronic, degenerative genetic disorders – fatal if left untreated. DMD is characterized by a decline in muscle function, leading most patients to die in their mid-twenties, while CF causes irreversible damage to lung tissue. Existing treatments are able to prolong life or reduce symptoms, but are not disease-modifying, and are therefore unable to significantly alter the path of the disease. Cures are therefore desperately sought, while drug treatments to counter symptoms remain a lucrative market.
According to the report, enzyme replacement therapies for Pompe and Fabry disease currently command prices of around $300,000 a year per patient. Despite serving very low patient populations, enzyme replacement therapies have provided an important boost to the rare diseases market, not only in providing revenue, but in demonstrating their potential to other companies. The ability to price orphan drugs highly has encouraged the interest of large pharmaceutical companies, as the potential in this medical sector has become apparent.
In the future, more companies are expected to cultivate a presence in the rare disease space, in an attempt to counter falling revenue from more stagnant traditional therapeutic areas and the patent cliff. While the development pipeline for orphan genetic disorders is small, the challenging nature of the diseases has encouraged innovative mechanisms of action to be developed in order to meet unmet market needs.
In DMD, RNAi therapeutics have seen some success in clinical trials and have advanced to Phase III. Exon-skipping therapies are being developed by several companies, showing the promise this innovative solution might hold. To date, there has been very little success in bringing antisense therapeutics to the market, but, as the report indicates, there is hope that with the significant backing of large pharmaceutical companies and the technical expertise of smaller companies in collaboration (as seen with GlaxoSmithKline and Prosensa), exon-skipping may make it to the market. In addition, further gene therapies in the form of PTC’s Ataluren are also in the late-stage pipeline for DMD and CF, targeting those patients with nonsense mutations.
The global orphan diseases in genetic disorders therapeutics market is growing at a strong rate. Having grown from $559m in 2004 at a compound annual growth rate (CAGR) of 17.3%, the market was worth $1.5 billion in 2010, and is expected to increase from this to $4.1 billion by 2018 at a CAGR of 13.8%.
*Orphan Disease Therapeutics in Genetic Disorders to 2018 - Emerging Agents in Cystic Fibrosis Offer strong Opportunities for Investment and Licensing Activity
This report provides insights into the global orphan diseases in genetic disorders therapeutics market, including market forecasts until 2018. It provides an in-depth analysis of major orphan genetic disease indications, covering Cystic Fibrosis (CF), Duchenne Muscular Dystrophy (DMD), Fabry disease and Pompe disease. The report also includes insights into the orphan genetic disease therapeutics R&D pipeline. It analyzes the competitive landscape, including M&As, and licensing and co-development deals.
The report is built using data and information sourced from proprietary databases, primary and secondary research and in-house analysis by GBI Research’s team of industry experts.
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