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FDA approves U-M-invented drug for Gaucher disease

ANN ARBOR, Mich. — Cerdelga, a treatment for Gaucher disease type 1 developed at the University of Michigan Medical School and licensed to Genzyme Corp., has been approved by the U.S. Food and Drug Administration.

The company says it expects to make Cerdelga available to patients within the month.

Cerdelga represents the first class of chemical entities conceived and developed at U-M to achieve FDA approval. An oral medication, cerdelga offers an alternative to intravenous enzyme replacement ­-- the other approved Gaucher disease treatment.  

The strategy of treating Gaucher disease by inhibition of glycolipid synthesis was proposed by the late Norman Radin, Ph.D.,a U-M neurochemist, more than 40 years ago. The work directly leading to Cerdelga began 25 years ago when James Shayman, M.D., a nephrologist trained in lipid biology and pharmacology, sat down with Radin and began their collaboration.

Their shared work continued at least until 1995 after which Radin retired, but research continued by the Shayman group to design, synthesize and test glycolipid synthesis inhibitors. That included a series of "proof of concept studies" in experimental models of lysosomal storage diseases. Patents covering these compounds and related inventions were licensed to Genzyme, a Sanofi company, in 2000 for clinical development.

Gaucher disease is an inherited disorder that affects many of the body's organs and tissues. More than 10,000 patients worldwide are diagnosed with Gaucher disease.

"On a personal level, the success of this research program has been tremendously gratifying," Shayman said. "The FDA approval of Cerdelga provides further motivation for me and my collaborators to bring other drugs through the pipeline."

The safety and effectiveness of Cerdelga were evaluated in two clinical trials with 199 participants with Type 1 Gaucher disease.

"The approval by the FDA is an important milestone for Genzyme, our inventors and the university," said Ken Nisbet, associate vice president for research, U-M Tech Transfer. "Cerdelga promises to be a landmark therapy for patients afflicted with Gaucher disease, and we congratulate all who helped bring this innovation to market."

Press release courtesy of U-M News Service.

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