FDA OK for Gaucher drug opens new market for Pfizer, Protalix

After initially being rebuffed at the FDA Pfizer ($PFE) and Protalix have snagged an FDA OK for their new Gaucher disease drug Elelyso (taliglucerase), an orphan enzyme replacement therapy which will appear on the market at a discount over Genzyme's mainstay and carrying a pointed promise to avoid supply interruptions.

Israel's Protalix heralded the approval as an endorsement of its plant cell-based manufacturing system. Elelyso is made from carrot cells rather than the more complex mammalian cell-based system that Genzyme--now owned by Sanofi ($SNY)--uses. And with its first approval complete and a new revenue stream to rely on, Protalix's chances of inking more favorable deals on its pipeline drugs just improved considerably.

Pfizer bought global rights to the drug--outside of Israel--with a $60 million upfront and the promise of $55 million more in regulatory milestones. Protalix, which saw its share price ($PLX) spike 23% last night, gains a $25 million milestone on the approval. And now the two companies will split the revenue 60/40.

Only about 6,000 people in the U.S. have Gaucher's disease. But that's been a big enough market for Genzyme, which has turned Cerezyme into a big winner over the years. Pfizer, which needs all the new products it can get to replace the Lipitor franchise, says it will price the new therapy at a 25% discount to Cerezyme. In another jab at its rival, Pfizer also promises to carry a 24-month supply of the drug to avoid the manufacturing snafu that created lingering supply problems for Genzyme.  

Protalix was handed a CRL on Elelyso a year ago, but regulators limited their demands for more information to a few technical issues, without requiring more data from clinical studies.

- read the press release
- here's the Globes story
- get the report from Bloomberg