The U.S. Food and Drug Administration gave approval on Tuesday - late Tuesday night, Israel time - for Protalix Biotherapeutics to market its experimental biotech drug for a form of the rare genetic disease Gaucher.
The decision came two and a half years after the successful completion of clinical trials for the drug, which Protalix plans to market in partnership with Pfizer under the brand name Elelyso.
Protalix shares soared 22% in after-market trading after the announcement, which followed nail-biting trading sessions in Tel Aviv and New York, where the stock fell 6.2% and 10.9% respectively.
Breakthrough research
Elelyso is based on breakthrough research by Dr. Yoseph Shaaltiel, the company's chief scientist. He generated medicinal proteins from genetically modified carrot cells, rather than the more expensive and bacterial-infection prone mammalian cells generally used for biotech drugs.
Gaucher disease is a rare genetic deficiency or malfunction of the enzymes that break up fatty deposits, resulting in buildup in internal organs like the spleen and liver, impaired red blood cell production and skeletal damage.
It affects an estimated 10,000 people worldwide, 60% of whom live in the United States. Because of the high cost, just 6,000 of Gaucher patients undergo treatment, primarily because it costs $250,000 a year per person.
Sales of existing treatments, Sanofi's Cerezyme and Ireland-based Shire's VPRIV, totaled $864 million over the past 12 months. Both companies have been hurt by production capacity limitations: At Sanofi, the market leader, these stemmed from quality control problems.
Protalix will produce the new drug and market it outside of Israel under a partnership agreement with Pfizer dating from November 2009, receiving 40% of the product's markup plus an expected one-time $25 million payment from Pfizer.
Elelyso is among just a handful of original drugs developed in Israel that have won FDA approval.
