GSK sees new value in treatments for rare diseases

Pharma giant GlaxoSmithKline has decided that it may be in its interest to develop drugs for diseases that very few people get. On Monday, GSK announced an alliance with two Italian groups, Fondazione Telethon and Fondazione San Raffaele, to develop and sell gene therapy for ADA severe combined immune deficiency, also known as the "bubble boy disease." While it's made headlines occasionally, it only effects about 350 children worldwide.

Bloomberg reports that the decision is part of a general trend among pharmaceutical companies seeking new ways to generate revenue as their drugs face generic competition.

"We want to create a diverse portfolio of compounds looking at a broad spectrum of rare diseases," Marc Dunoyer, global head of the GSK unit, said during a conference call. "The strong GSK pipeline will give us enormous scope and access to novel compounds with mechanisms we understand that could be utilized to treat rare diseases."

GSK's rare-disease unit will focus on "four therapeutic clusters"--metabolism and inherited disorders, the central nervous system and muscle disorders, immune-inflammation, hematology and rare malignancies, Bloomberg quotes Dunoyer as saying.

- read Bloomberg's report
- Robert Langreth blogs about it at Forbes
- get Jeanne Whalen's take at the Wall Street Journal