Ultra orphan diseases definition
Lysosomal Storage Disease: an Example of a Lucrative Ultra-Orphan Market
The pharmaceutical industry has shown an increasing interest in developing drugs for rare, orphan diseases. Amongst the orphan diseases is a subgroup of extremely rare disorders, termed ultra-orphan. The lysosomal storage disorders (LSDs), each of which occurs in less than 10, 000 people worldwide, are examples of ultra-orphan diseases. Despite the very low prevalence of these conditions, therapies for ultra-orphan drugs have yielded high returns for the developing companies, as exemplified by Genzyme’s blockbuster Cerezyme (imiglucerase) for the ultra-rare Gaucher disease. This short review will assess the LSD market as an example for the market opportunity of targeting such ultra-orphan treatments.
Increasing interest in orphan drugs
Until the introduction of the US Orphan Drugs Act in 1983, followed by similar legislation in other markets such as Japan in 1997 and the EU in 1999, the pharma industry had limited interest in the development of therapies for rare diseases with a highly unmet medical need as they did not seem worthwhile investments. With this legislation providing new incentives, including tax credits for the costs of clinical research, extended marketing exclusivity compared with non-orphan drugs, R&D grants and high rates of regulatory success, the industry has come to realize the revenue potential of orphan drugs; a previous analysis by Thomson Reuters determined that orphan drug development is now an economically viable strategy for pharma companies.
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