ROCKVILLE, Md. The Food and Drug Administration has approved a new drug for treating a rare genetic disorder, the agency announced Friday.
The FDA approved Shire Human Genetic Therapies’ VPRIV (velaglucerase alfa) as a treatment for Type 1 Gaucher disease. The disease, which affects between 1-in-50,000 and 1-in-100,000 people in the general population, causes underproduction of the enzyme glucocerebrosidase, causing harmful levels of a fatty substance to build up in the liver, bones, nervous system and spleen and preventing cells and organs from working properly.
The drug is seen as an alternative to Genzyme’s Cerezyme (imiglucerase), which currently is in short supply.