U.S. Food and Drug Administration (FDA) has approved the drug Elelyso (taliglucerase alfa) for the treatment of type 1 Gaucher disease on Tuesday.
Gaucher disease is a liver and neurological disorder. In Gaucher disease, there is a lack of an essential enzyme resulting in the storage of fatty chemicals in the liver, spleen, bones, and nervous system. FDA has reported that the major signs of the disease are spleen and liver damage, low red blood cells and platelet count and bone problems. This disease has affected about 6000 people in U.S.
[hana-code-insert name=’StumbleUpon’ /][hana-code-insert name=’Reddit’ /]Elelyso is developed by Protalix BioTherapeutics Inc. and licensed to Pfizer Inc. and has been approved as an orphan drug. This drug will work as an enzyme replacement. As an orphan drug, competing versions of the drug could not be approved for up to seven years.
“Today’s approval provides for a new enzyme replacement therapy for the select number of patients with Type 1 Gaucher disease,” said Julie Beitz, M.D., director of the Office of Drug Evaluation III in FDA’s Center for Drug Evaluation and Research. “It also demonstrates FDA’s commitment to developing treatments for rare diseases.”
Elelyso is effective in reducing the spleen volume at a dose of either 30 units per kilogram or 60 units/kg.
The most common side effects of this drug are headache, hives, fatigue, chest pain, weakness, back and joint pain, skin redness and flushing.
Other drugs that are in use to treat Gaucher’s disease are Shire PLC’s Vpriv and Sanofi’s Cerezme.