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[DewDiligence]

Protalix Falls As FDA Rejects Gaucher Drug

[My comment on this, FWIW, is in #msg-60329097.]

http://www.bloomberg.com/news/2011-02-25/pfizer-protalix-fail-to-win-fda-approval-of-cerezyme-rival-1-.html

›By Catherine Larkin - Feb 25, 2011 4:27 PM ET

Protalix BioTherapeutics Inc. fell the most in two years in New York trading after it failed to win U.S. approval of a plant-based medicine it’s developing with Pfizer Inc. to treat Gaucher disease.

Protalix, based in Carmiel, Israel, dropped $1.73, or 18 percent, to $7.63 at 4:04 p.m. in New York Stock Exchange composite trading, the biggest percentage decline since Dec. 1, 2008. Pfizer fell 4 cents to $18.86.

The Food and Drug Administration asked for more data from two studies and information on testing specifications in the new drug application for taliglucerase alfa, developed to treat Gaucher disease, an inherited illness that causes fat build-up that can lead to lung disease and brain damage, Protalix said today in a statement. No new trials were requested. The drug would compete with Genzyme Corp.’s top-selling Cerezyme and Shire Plc’s Vpriv.

“We don’t think this is going to be a very long process,” Protalix Chief Executive Officer David Aviezer told analysts and investors on a conference call today. The company plans to work closely with Pfizer and request a meeting with the FDA as soon as possible to determine what needs to be done for approval.

Taliglucerase is the first product for Protalix. The medicine is designed to replace an enzyme missing in people with Gaucher disease, a disorder that causes fat to build up in the liver, spleen, bone marrow and nervous system.

Rivals’ Sales

Cerezyme had global sales of $719.6 million last year for Cambridge, Massachusetts-based Genzyme, a 42 percent decline from two years ago because of manufacturing issues. Vpriv, chemically known as velaglucerase alfa, generated sales of $143 million for Dublin-based Shire after being approved by the FDA in February 2010.

“Importantly, it does not appear that the FDA has significant safety concerns with taliglucerase,” said Brian Abrahams, an analyst at Wells Fargo Securities in New York, in a note to clients today. “Rather, given the improving supply of Genzyme’s Cerezyme and availability of Shire’s velaglucerase, the agency likely prefers to wait for a more complete dataset for patients on taliglucerase prior to approval.”

Gaucher disease affects about 1 in every 50,000 to 100,000 people and can lead to bone pain, anemia, lung disease or brain damage, according to the National Human Genome Research Institute.

Taliglucerase, once approved, may win at least 15 percent of patients “through strong marketing and competitive pricing,” said Ritu Baral, an analyst at Canaccord Genuity in New York, in a Feb. 22 note to clients. Cerezyme costs more than $200,000 a year.

Pfizer, the world’s largest drugmaker, paid $60 million for rights to taliglucerase in November 2009 and promised Protalix an additional $55 million if the drug passed certain regulatory hurdles [#msg-44047104]. New York-based Pfizer is entitled to 60 percent of the drug’s revenue under the licensing agreement.‹