Pfizer adds focus on rare diseases

Cambridge-based group may compete with Genzyme

By Robert Weisman
Globe Staff / June 16, 2010

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Pfizer Inc. is setting up a rare disease research and development group in Cambridge, a move that will put the world’s largest drug maker in direct competition with local biotechnology firms such as Genzyme Corp.

Pfizer signaled its intentions to increase its focus on making drugs to treat rare diseases Dec. 1, when it said it would spend up to $110 million to license global rights to a treatment being developed by Israeli start-up Protalix BioTherapeutics Inc. for the genetic disorder Gaucher disease.

Genzyme’s treatment for Gaucher disease, Cerezyme, is the Cambridge company’s top-selling product. Also, the Lexington-based human genetic therapies division of British drug maker Shire PLC has asked the Food and Drug Administration for approval to market a rival treatment for Gaucher.

Production problems at Genzyme’s plant in Allston last summer caused the temporary rationing of Cerezyme, creating an opening for competing drugs in a market where treatments can cost as much as $300,000 a year per patient. Pfizer was quick to seek to capitalize.

At an appearance in Boston on the day the Protalix alliance was disclosed, Pfizer chief executive Jeff Kindler said the deal with the Israeli company was “the first of many we’re trying to meet unmet medical needs in a way we haven’t done before.’’ Kindler said “diseases that otherwise might not be well served are an opportunity for us.’’

Shares of Pfizer edged up 19 cents, or 1.2 percent, to $15.50 on the New York Stock Exchange yesterday. Genzyme shares climbed 88 cents, or 1.8 percent, to $50.38 on the Nasdaq exchange.

Genzyme spokeswoman Lori Gorski declined to comment on Pfizer’s foray into rare disease research.

The move follows that of another pharmaceutical giant, Britain’s GlaxoSmithKline, which in February unveiled plans for a stand-alone rare diseases unit.

Pfizer’s research will draw on the expertise the company has in marketing a treatment for hemophilia, said Jose Carlos Gutierrez-Ramos, the company’s Cambridge-based senior vice president for biotherapeutics research and development.

“The whole idea for getting into this space was first of all due to the need and, second, that we were already there for hemophilia,’’ Gutierrez-Ramos said. “A lot of it is internal expertise, but we will build a small new group.’’

Pfizer’s rare diseases research operation will be led by Dr. Edward Masciolo, most recently the founder and chief executive of Dapis Capital, a private equity firm investing in health care and life sciences businesses. Gutierrez-Ramos said it was too early to say how many additional researchers Pfizer would hire for the new Cambridge unit.

In addition to hemophilia, the unit will initially concentrate on treatments for muscular dystrophy and serious diseases caused by genetic mutations.

Anne E. Wilson, a Pfizer spokeswoman in New York, said the size of the company’s investment “will be proportional to the medical need’’ in the field of rare diseases. Pfizer will seek to strike partnerships with Boston area universities and biotechnology firms, she said.

Pfizer’s decision to concentrate on rare disease treatments comes less than a month after the company disclosed plans to eliminate about 6,000 manufacturing jobs worldwide over the next five years. That includes about 300 at the Andover biotechnology plant it acquired when it purchased Wyeth Pharmaceuticals for $68 billion last year.

The job cuts, which will be done through layoffs and attrition, will leave Pfizer with just over 2,000 employees in Massachusetts, including 750 at two research and development sites in Cambridge.

Robert Weisman can be reached at