A northern Israeli biotechnology firm has just filed its New Drug Application with the U.S. Food and Drug Administration for an innovative new treatment for Gaucher's disease.
Protalix Biotherapeutics Inc. (NYSE-Amex: PLX), located in Carmiel, says the new medication, taliglucerase alfa, will be cheaper than the current treatment marketed in the United States.
The Israeli-produced medication is an enzyme-replacement therapy that is derived from plants, as opposed to Cerezyme, which costs each patient an estimated $200,000 per year and is produced using hamster cells.
Taglucerase alfa is less expensive, says Protalix, simply because the manufacturing process does not require the large intial set-up investments involved in mammalian cell-based protein production, nor does it need the extensive ongoing costs associated with growth media and monitoring. In addition, the half-life of the medication appears to be significantly longer than that of Cerezyme, as indicated by publicly-available data.
The company also signed a lucrative deal with U.S. pharmaceutical giant Pfizer (NYSE: PFE) on December 1, 2009 to develop and commercialize the new medication, giving Pfizer exclusive worldwide licensing rights. Protalix retains commercialization rights in Israel, however.
"With this submission, Protalix and Pfizer take a significant step forward in making a cost-effective treatment alternative available for Gaucher's disease patients," said Dr. David Aviezer, president and CEO of Protalix. Aviezer holds a Ph.D. in Molecular Biology and Biochemistry from the Weizmann Institute of Science and an MBA from Bar Ilan University's School of Business.
Pfizer added in a statement, "Upon the receipt of regulatory approvals, we are poised to quickly make taliglucerase alfa available to patients suffering with Gaucher's disease who are in need of new treatment options."
In the meantime, taliglucerase alfa has been given an orphan product designation and was granted fast track development status by the FDA, and is currently being provided to Gaucher's patients in the U.S. under an expanded access protocol. It is also being provided to patients in the European Union under a compassionate use protocol.
The company has also filed its proposed pediatric investigation plan with the pediatric committtee of the European Medicines Agency (EMEA) for a clinical study in patients ages 2 to 18.
Gaucher's disease, an inherited condition, occurs in approximately one out of every 20,000 live births. People with Gaucher's disease do not have enough of an enzyme called glucocerebrosidase, which breaks down a certain type of fat molecule. As a result, lipid-engorged cells, called Gaucher cells, amass in different parts of the body -- primarily the spleen, liver and bone marrow. Accumulation of Gaucher cells may cause the spleen and liver to swell, as well as cause anemia, excessive bleeding and bruising, bone disease and a number of other symptoms.