Pfizer said it entered into an agreement with Protalix to license the Israeli company's experimental drug to treat Gaucher's Disease, a rare, inherited disorder that can prevent the proper functioning of the spleen, liver, lungs, bones and sometimes one’s brain. The experimental enzyme replacement represents a competitive threat to Genzyme’s Cerezyme, which has been the primary treatment since that drug’s approval in 1994.

 

Following a successful late-stage trial, Protalix is preparing to complete a rolling New Drug Application with the U.S. Food and Drug Administration for its treatment known as taliglucerase alfa. The drug is unusual in that it is produced in genetically altered carrot cells and represents the first enzyme replacement therapy derived from plant cells.

Under the agreement, Pfizer will make an upfront payment of $60 million to Protalix, which is also eligible for additional milestone payments of up to $55 million. The companies will split any revenue from the drug with 60 percent going to Pfizer and 40 percent to Protalix.

 

“By joining our advances in biologics manufacturing and protein development with Pfizer’s global strengths in patient services and reimbursement we expect to help make taliglucerase alfa an important and cost-effective treatment choice for Gaucher’s patients throughout the world,” says David Aviezer, president and CEO of Protalix.

 

A match between Pfizer’s interest in emerging markets and the cost-effectiveness of taliglucerase alfa, which may be less expensive than Cerezyme, could be part of what drew Pfizer to partner with Protalix, according to some observers.

 

The competition comes at a particularly challenging time for Genzyme, which had to halt production of Cerezyme in June after finding a virus in its Massachusetts production plant. Those production problems created a shortage of the drug. The company has since resumed production and is ramping up for expanded shipments by the end of the year.

 

U.K.-based Shire is also seeking regulatory approval for its own Gaucher’s Disease treatment.