Guidelines for the restart of imiglucerase in patients with Gaucher disease: recommendations from the European Working Group on Gaucher disease.

Between July and December 2009, global shortage of imiglucerase for the treatment of Gaucher disease has occurred due to a viral contamination of the manufacturer's (Genzyme) single production facility, which necessitated its temporary shut-down. As a result, only 20% of the prior global supply has been available, leading to either interruption of treatment, dose reduction, or starting of alternative treatments. The European Working Group on Gaucher Disease (EWGGD), supported by the European Gaucher Alliance (EGA), issued guidelines as how to protect the most vulnerable patients and discussed options for alternative treatments. In addition, an emergency programwas launched for patients that met criteria for being at risk for the development of complications [1]. With the pending restoration of the full production of imiglucerase from the end of 2009 onwards, Genzyme suggested to the EWGGD to produce recommendations for the re-installation of imiglucerase. An unfortunate concern has risen with respect to contamination of b1% vials of imiglucerase (as well as of additional enzymes produced at the same manufacturer facility) with foreign particles, including stainless steel fragments, non-latex rubber from the vial stopper, and fiber-like material. The FDA has required that a “Dear Healthcare Professional Letter” was issued with recommendations for inspection of the solution and for the use of 0.2or 0.22-μm filters [2]. These recent events raise the following considerations: