FDA Approves Therapy to Treat Gaucher Disease
New drug will offer a treatment alternative for patients with rare
The U.S. 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.
Gaucher disease occurs in people who do not produce enough of an enzyme
called glucocerebrosidase. Without this enzyme, harmful amounts of a
certain fatty substance (lipid) 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.
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. Cerezyme is currently in short supply.
“The approval of VPRIV will provide a safe and effective alternative
treatment for patients with Gaucher disease,” said Julie Beitz, M.D.,
director of the FDA’s Office of Drug Evaluation III. “Patients who
previously received Cerezyme as an enzyme replacement therapy for their
Type 1 Gaucher disease can be safely switched to VPRIV.”
The safety and effectiveness of VPRIV was assessed in three clinical
studies involving 82 patients with Type 1 Gaucher disease ages 4 years
and older. The studies included patients who switched to VPRIV after
being treated with Cerezyme.
The most common adverse reactions to VPRIV are allergic reactions. Other
observed adverse reactions with VPRIV are headache, dizziness,
abdominal pain, back pain, joint pain, nausea, fatigue/weakness, fever,
and prolongation of activated partial thromboplastin time, a measure of
VPRIV is manufactured by Shire Human Genetic Therapies Inc. of