The US Food and Drug Administration has approved velaglucerase alfa for injection (VPRIV) to treat children and adults with a form of the rare genetic disorder Gaucher disease. VPRIV is manufactured by Shire Human Genetic Therapies, Inc. (Cambridge, MA).
VPRIV provides long-term enzyme replacement therapy for Type 1 Gaucher disease, the most common form of the genetic disorder. It is an alternative to Cerezyme (imiglucerase), another enzyme replacement therapy, manufactured by Genzyme (Cambridge, MA). Cerezyme currently is in short supply because of recent manufacturing problems at the Genzyme plants that produce Cerezyme (see previous coverage). Shire had received an FDA-approved treatment protocol to allow early patient access to velaglucerase alfa to address the Cerezyme shortage.
Gaucher disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase. Without this enzyme, harmful amounts of the lipid glucocerebroside can build up in the liver, spleen, bones, bone marrow, and nervous system, and can prevent cells and organs from working properly. About 1 in 50,000 to 1 in 100,000 people in the general population have Gaucher disease.