Fabry disease patients have new option

Fabry disease sufferers in the US have simultaneously pursued several courses of action in hopes of obtaining relief from the life-threatening situation in which they find themselves. ABC News has a good overview. (See my earlier post below about compulsory licensing ) One hopeful outcome, the result of a letter-writing campaign by the Fabry Association, is that Shire sought and obtained approval from the European Medicines Agency to manufacture Replagal in a Lexington, MA factory. FDA approval is now being sought. The FDA previously approved a clinical trial with 140 Fabry patients who were switched from Genzyme's Fabrazyme to Shire's Replagal. The trial results were positive. FDA approval of Replagal has also been fast tracked. A review (second and third paragraphs) by Sylvie Grégoire, Shire's President of Human Genetic Therapies, presents the company's pre-approval efforts to help US patients as well as those in countries where Replagal has already been approved.

Fabry Patients Petition For Compulsory License of Fabrazyme