Lysosomal storage disorders are a particularly lucrative corner of ultra-orphan market

A new report by Thomson Reuters has highlighted the economic power of ultra-orphan diseases, the informal category for those illnesses that affect fewer than 10,000 people.

It particularly focuses on the potential of lysosomal storage disorders as ultra-orphan diseases, which is a group of more than 50 rare inherited metabolic disorders, with each occurring in less than 10,000 people across the world. This extremely small target population means treatment for lysosomal storage disorders can command extremely high prices in order to recoup R&D expenditure.

Treatments currently in the pipeline for these diseases are Sanofi (Euronext: SAN) subsidiary Genzyme’s substrate reduction therapy Cerdelga (eliglustat tartrate). Genzyme has achieved blockbuster sales with another ultra-orphan therapy, Cerezyme (imiglucerase for injection)  for Gaucher disease type 1, exceeding $1 billion in 2006 and 2008, and expected to top $1.2 billion in 2018, according to Thomason Reuters Cortellis Competitive Intelligence.