Big pharma and biotech increasingly tackling rare and niche diseases, new report finds

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 study released by GlobalData.

The new report 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, Pfizer’s Elelyso (taliglucerase alfa) has already been approved by the Food and Drug Administration for the treatment of Gaucher disease (The Pharma Letter May 2), 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).