Big Pharma and Biotech Increasingly Tackling Rare and Niche Diseases

15-05-2012: Rare and niche diseases are being increasingly spotlighted by pharmaceutical and biotechnology corporations in the hope of developing initial treatments or improving upon existing therapies for these conditions, according to a new report released by GlobalData.

The new report, "Rare and Niche Diseases Catalyst Monitor - Key Event Analysis H1 2012", reviews catalyst events in the second quarter of 2012 regarding new and additional therapies that are being developed for several rare and niche diseases. These include Duchenne muscular dystrophy (DMD), Gaucher disease, Philadelphia chromosome negative (Ph-) acute lymphocytic leukemia (ALL), Fabry disease, and idiopathic pulmonary fibrosis (IPF).

One product, Elelyso (taliglucerase alfa) has already been approved by the FDA for the treatment of Gaucher disease, and provides patients with an alternative, lower-priced enzyme replacement therapy (ERT) option to Genzyme’s Cerezyme (imiglucerase), the market leader and standard of care, and Shire’s Vpriv (velaglucerase alfa).

The passage of the Orphan Drug Act in the United States in 1983 was the initial impetus behind the development of products for niche and rare diseases, and since that time, similar legislation has been enacted in the UK, Europe, Japan, Singapore and Australia. As a result of this global legislation, numerous products have been brought to market to treat diseases for which there were no existing therapies, saving countless lives worldwide.

Pharmaceutical and biotechnology companies are also increasingly focusing on developing products for these diseases as a means of reinvigorating their product pipelines. The market for these products is virtually wide open and will continue to be a major driving force in the industry.