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

Posted under Blog, Cerezyme, Companies, Diagnostics, Elelyso, Funding, Gaucher disease, Genzyme, Medical Devices, Medical Supply, Pfizer, Pharmaceuticals, Pipeline, Protalix, Regulatory, Startups, Universities, Videos by john

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

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Genzyme's shortage opens door to rival Protalix
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FDA hands Protalix a CRL for Gaucher's disease drug
 

Protalix shares dive on Gaucher’s drug delay at FDA

Posted under Blog, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, Pharmaceuticals, Protalix, Startups, Universities, Videos by John Carroll

Protalix BioTherapeutics ($PLX) reported this morning that the FDA is putting off its deadline for a final decision on its drug for Gaucher's disease. The new deadline is May 1, and the news quickly triggered a fresh drop in the company's share price as investors responded to the delay. Story