Racing to plug a market gap created by the sudden shortage of Cerezyme, the UK's Shire says that it has filed for FDA approval of its new therapy for Gaucher disease ahead of schedule. The developer says that it has wrapped two late stage trials of velaglucerase alfa , hitting all their primary and secondary endpoints, and plans to start offering the drug for free to patients stranded by the unexpected shutdown of a key manufacturing facility.
In a rare display of public affection, the FDA has openly encouraged Shire as well as Israel's Protalix BioTherapeutics to push ahead with their development programs for Gaucher disease--an enzyme deficiency that triggers the accumulation of fat cells in the spleen and other parts of the body--as Genzyme worked to clean up the viral contamination that temporarily shuttered its Allston Landing facility in Boston.
Analysts note that Shire's intention to make its therapy available to patients for free could prove profitable in the long run. Patients rarely switch meds for Gaucher disease and have proven willing to pay $250,000 a year for the drug. Genzyme's contamination issue and the FDA's sense of urgency offer Shire an unusual opening. Shire's vela could hit the market in mid-2010.
Genzyme, meanwhile, is being hit with fresh questions about the Allston plant after EMEA inspectors recently raised a "major observation" following an inspection. Genzyme isn't explaining, but some of the analysts wonder why the facility still has problems as it gets back into full production.