Jumping into rare diseases, Pfizer inks $115M Protalix deal

Pfizer is plunking down $60 million upfront and promising up to $55 million in milestones to license the worldwide rights to Protalix's drug for Gaucher disease--a move the puts the global pharma company in head-to-head competition with Genzyme as the big biotech company wrestles to overcome a plague of manufacturing problems.

Israel's Protalix had been thrust into the spotlight with its late-stage treatment for Gaucher disease after contamination issues forced Genzyme to halt production of Cerezyme at its Boston facility. While only a relative handful of patients suffer from the rare disease, Genzyme has brought in blockbuster revenue by charging $200,000 a year for the drug. And Protalix is expected to file for an approval soon, with marketing expected to get underway next year.

New York Times biotech reporter Andrew Pollack notes that big pharma has traditionally avoided drugs for rare diseases and focused on mega-blockbusters capable of generating huge profits. But with patents expiring and long-term revenue in doubt, programs like these offer an opportunity companies like Pfizer can no longer afford to overlook.

- check out the Protalix release
- read the story from the New York Times 
- here's the report from Reuters