Enzon Inc. said Tuesday that it exclusively licensedrecombinant alpha galactosidase for use in combination with itsPegnology drug delivery technology as a treatment for Fabry'sdisease, a rare fatal inherited metabolic disorder.
Enzon (NASDAQ:ENZN) of South Plainfield, N.J., signed anagreement with Research Corporation Technologies, whichrepresented the City University of New York (CUNY), whoseresearchers developed the enzyme. Alpha galactosidase wasgranted an orphan drug designation in 1991. No financial termsof the deal were released.
Enzon's Pegnology technology couples polyethyleneglycol (PEG)strands to proteins, such as alpha galactosidase, with the aim ofextending their duration in the bloodstream and reducing theirtoxicity. Used alone, recombinant alpha galactosidase would betoo toxic and remain in the body for too short a time to beeffective, said Donna Chappina, Enzon's corporatecommunications manager.
Fabry's afflicts up to 10,000 people annually in the UnitedStates, and usually strikes during childhood or adolescence. Itcauses the accumulation of fatty acids in the heart and kidney,leading to their failure. "It is a very, very painful, fatalcondition," Chappina said. There is no available treatment forthe disease, and Enzon believes it is the only company workingon one.
Research will continue at both Enzon and at the City Universityof New York under the direction of David Calhoun, the inventorof recombinant alpha galactosidase. Enzon officials could offerno timetable on the product's development.
Research Corporation Technologies, a technology licensingcompany that represents colleges, universities and non-profitlaboratories, has filed a patent application on the recombinantenzyme.
Enzon's drug Adagen was approved in 1990 for treatinganother genetic disorder, severe combined immunodeficiencydisease (SCID), one of the rarest of genetic disorders. Enzon'sdrug is used to treat 30 patients worldwide.
"We are specifically forging ahead in genetic disorders,"Chappina said. Enzon expects to announce additionalcollaborations aimed at developing therapeutics to treat geneticdisorders.
-- Steve Payne BioWorld Staff
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