Pfizer, Protalix to develop Gaucher’s disease treatment

NEW YORK Pfizer and Protalix have entered an agreement potentially worth more than $100 million to develop a drug for treating an inherited disorder that occurs in one-in-20,000 live births, Pfizer announced.

The two companies will work to develop and commercialize taliglucerase alfa, a treatment for Gaucher’s disease that recently completed late-stage clinical trials. Protalix is planning to file a regulatory approval application with the Food and Drug Administration, which has given the drug orphan drug designation and fast-track approval status. The drug is made from genetically engineered carrot cells.

Gaucher’s is a lysosomal storage disorder in which amounts of the enzyme beta-glucosidase are not enough to break down a certain fat molecule, causing fat-engorged cells to amass around the body, mostly in the spleen, liver and bone marrow. Accumulation of the cells, called Gaucher cells, may cause enlargement of the spleen and liver, anemia, excessive bleeding and bruising, bone disease and other symptoms.

Under the agreement, Pfizer will receive exclusive worldwide licensing rights for the commercialization of the drug, while Protalix will retain exclusive commercialization rights in Israel. Pfizer will pay Protalix $60 million upfront, as well as milestone payments of up to $55 million. The two companies will share future and revenues and expenses, with Pfizer getting a 60% share and Protalix getting a 40% share.