Protalix Wins FDA Approval for Its First Drug on the Market

Protalix BioTherapeutics Inc. jumped as much as 27 percent in extended trading after the drugmaker won approval for its debut medicine, a treatment it developed with Pfizer Inc. for a rare genetic disorder.

The Food and Drug Administration cleared the product called Elelyso to treat a condition caused by lack of an enzyme that can lead to spleen and liver enlargement known as Gaucher disease. The drug, chemically known as taliglucerase, is approved for Type 1 Gaucher disease affecting about 6,000 people in the U.S., the agency said in a statement today.

While Gaucher disease is a rare condition, the market is lucrative because of “the severity of the symptoms and chronic nature of the disease,” according to Carmiel, Israel-based Protalix’s website. Elelyso will compete with Sanofi’s Cerezyme, approved in 1994, and Shire Plc’s Vpriv, approved in 2010.

Protalix and New York-based Pfizer are prepared to supply all 2,000 patients on Gaucher treatment in the U.S., Vera Wu, Pfizer’s global asset lead for Elelyso, said in a telephone interview. Pfizer, the world’s largest drugmaker, will share in 60 percent of revenue.

Protalix rose as high as $7.88 after closing at $6.19 in New York trading. Pfizer was unchanged after closing at $22.78.

Carrot Cells

The drug is an injection given by a health-care professional every other week, according to the FDA.

The approval “demonstrates FDA’s commitment to developing treatments for rare diseases,” Julie Beitz, director of the FDA’s Office of Drug Evaluation III, said in the statement.

The FDA delayed a decision originally slated in December to review additional clinical information the agency requested.

Elelyso is the first FDA-approved drug made from a genetically engineered plant, David Aviezer, president and chief executive officer of Protalix, said in a telephone interview. Other plant-based drugs consist of chemicals naturally extracted from plants, he said.

The drug is made from carrot cells. Cerezyme is a mammalian cell-based treatment, according to Protalix’s 2011 annual report. Mammalian cell-based systems are highly sensitive and require complex and expensive stainless steel bioreactors for growth, the company said. Protalix calls its bioreactors “simple” and “inexpensive.”

Lori Gorski, a spokeswoman for Sanofi’s Genzyme unit, said the company is “confident in Cerezyme’s position as the gold standard in the treatment of Gaucher disease Type 1, and all U.S. patients have returned to normal dosing.”

Revenue Sharing

Cerezyme brought in $614 million in revenue last year, according to data compiled by Bloomberg. Manufacturing issues caused contamination of Cerezyme at a Massachusetts plant in 2009 and led to shortages.

Elelyso is immune from mammalian-based viruses, Wu said. Pfizer also plans to keep a two-year supply of the drug in reserve for each patient prescribed Elelyso, she said. Reserves will be kept at plants in Israel, Germany, Kalamazoo, Michigan, and Memphis, Tennessee.

Pfizer paid Protalix $60 million for an exclusive agreement to sell the drug except in Israel, according to the annual report. Pfizer also agreed to pay Protalix $55 million for meeting certain regulatory requirements.

Aviezer declined to discuss in detail whether Pfizer partnerships are on the horizon for other drugs the company is developing with its plant-based system, including one for a rare genetic disorder known as Fabry disease. Further collaboration could be considered, he said.

“Pfizer has no rights to any other drugs, but on the other hand, we’ve established a very good working relationship,” he said.

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