FDA Approves New Drug for Treating Genetic Disease Gaucher From Protalix and Pfizer–Will Compete With Genzyme and Help Fill Drug Shortages

The competitor for the drug for the rare genetic disease was purchased by Sanofi last year for a huge amount and they also create drugs that treat other rare diseases.  There are only about 2000 patients in the US being treated for the condition.

Genzyme Sale to Sanofi-Aventis Finally Going to Take Place for Over $20 Billion

What also makes this drug interesting is the fact that it is made from carrot cells and is the first FDA approved drug made from a genetically engineered plant.  Protalix is located in Israel and Pfizer paid to have the distribution in the US.  BD 

WASHINGTON (Reuters) - U.S. drug regulators gave the nod on Tuesday to Protalix Biotherapeutics Inc and Pfizer Inc's experimental biotech drug for a form of the rare genetic disease Gaucher.

The Food and Drug Administration approved the intravenous drug, known chemically as taliglucerase alfa, after rejecting it last year and asking the companies for more data.

Shares of tiny Israeli biopharmaceutical company Protalix jumped nearly 23 percent to $7.60 in after-market trading following the approval of its leading product, which the companies plan to sell under the name Elelyso.

Gaucher disease stems from an enzyme deficiency that prevents the breakdown of certain fats in the body. It can cause organ damage or death and affects about one in 50,000 to 100,000 people. It is particularly prevalent among Ashkenazi Jews, according to the National Institutes of Health.

http://www.chicagotribune.com/health/sns-rt-us-fda-gaucherbre8401jz-20120501,0,5155428.story