FDA approves U-M invented drug for Gaucher disease

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

The company said it expected 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. Cerdelga offers an alternative to the other approved Gaucher disease treatment – an intravenous enzyme replacement. Cerdelga is administered orally and could become a treatment for Gaucher type 1 disease.

The strategy of treating Gaucher disease by inhibition of glycolipid synthesis was proposed by the late Dr. Norman Radin, a U-M neurochemist, more than 40 years ago. The work directly leading to Cerdelga began 25 years ago when Dr. James Shayman, 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 suffered by more than 10,000 patients worldwide that affects many of the body's organs and tissues.

"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."

Genzyme's road to FDA approval included clinical trials including phase 3 trials conducted in 60 medical centers in 29 countries, which comprised the largest group of clinical studies ever conducted on Gaucher disease representing over 500 patient years of exposure in approximately 400 individual patients.

"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."

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