Elelyso by Protalix/Pfizer has Gotten Approval for the Treatment of Gaucher Disease

Author: Usman Zafar Paracha
Published: May 02, 2012 at 5:38 am

U.S. Food and Drug administration (FDA) has approved the new treatment of a rare disease i.e. Gaucher disease that can cause liver and neurological problems.

The new drug is Elelyso (taliglucerase alfa) developed by Protalix BioTherapeutics Inc. and licensed to Pfizer Inc. This medicine has been approved as an orphan drug for the treatment of type 1 Gaucher disease. As an orphan drug this medicine has no competition from any other product up to seven years.

Gaucher disease is estimated to affect about 6000 people in U.S. The people with this disease lack an essential enzyme leading to the dangerous buildup of fatty chemicals in the liver, bones, spleen and nervous system. According to FDA, the major signs of the disease are spleen and liver damage, low counts of red blood cells and platelets and bone problems. Elelyso works as a replacement of that enzyme.

“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.”

The most common side effects of this drug are headache, chest pain, hives, fatigue, weakness, skin redness, back and joint pain and flushing.

Other drugs that are in use to treat Gaucher’s disease are Shire PLC's Vpriv and Sanofi's Cerezme.

Bloomberg has reported an increase of 27% in extended trading of Protalix BioTherapeutics Inc. after the approval of the medicine.


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Article Author: Usman Zafar Paracha

Usman Zafar Paracha has been blogging in science and technology for more than 6 years. He is CEO of SayPeople.com. Email: uzparacha@yahoo.com Twitter: @jeepakistan

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