KEI asks FTC to investigate Shire decision to abandon efforts to compete in US market for Fabry’s disease treatments

July 15, 2014

Knowledge Ecology International (KEI) asks the FTC to investigate Shire decision to abandon efforts to compete in US market for Fabry’s disease treatments. Letter to FTC provides evidence of possible conspiracy to segment markets, involving licensing of an NIH funded invention for use in Europe, in return for abandoning efforts to enter US market.

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James Love, Knowledge Ecology International
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On Tuesday, July 15, 2014, Knowledge Ecology International (KEI) asked the United States Federal Trade Commission (FTC) to investigate the decision by Shire PLC in 2012 to withdraw an application to the US FDA for marketing approval for Replagal, a treatment for Fabry’s disease.

KEI’s request is included in a six-page letter accompanied by an eleven-page timeline of events regarding the development of two competing treatments for Fabry’s disease, Fabrazyme, marketed by Genzyme, now a subsidiary of Sanofi, and Replagal, now marketed by Shire. Both products were initially developed with support from the NIH by U.S. biotechnology firms.

The KEI complaint focuses on events that took place between 2009 and 2012, during the time when Genzyme’s Orphan Drug market exclusivity was ending in the United States, and Genzyme was experiencing several manufacturing problems, causing the company to lose global market share for Fabrazyme to Shire’s Replagal.

Mount Sinai School of Medicine, recently renamed the Icahn School of Medicine at Mount Sinai, was simultaneously aggressively pursuing patent infringement actions in Europe against Shire, and facing a compulsory licensing request in the United States and Germany, for an invention that had been funded by the NIH. The Mount Sinai patent was subject to Bayh-Dole Act march-in provisions as well as an NIH royalty free right to use the patent worldwide.

During the same period, Carl Icahn, a trustee of Mount Sinai, was controlling 4.9 percent of the shares of Genzyme, and his 2010 proxy battle for Genzyme played a role in the 2011 sale of Genzyme to Sanofi.

In March 2012, Shire withdrew its application to the FDA to market Replagal in the United States, even though the Genzyme Orphan Drug exclusivity had expired, and Shire was already providing Replagal to roughly 20 percent of U.S Fabry patients, under an FDA approved initiative to address shortages of Fabrazyme in the United States. At the time, speculation in the United States centered on regulatory barriers that Shire faced for a new BLA approval. The KEI letter to the FTC focuses on a different issue: the use of a European license to an NIH funded patent to induce Shire to withdraw from the U.S. market.

Earlier a US Court ruled that Replagal did not infringe the Mount Sinai patent. But in 2010 a German court ruled the opposite, creating a situation where Mount Sinai could block competition in Europe, but not the United States.

In May 2012, following the Shire decision to abandon efforts to compete in the United States, Mount Sinai granted Shire the right to practice an NIH funded patent for Fabry’s treatment, in Europe.

KEI’s complaint asks the FTC to determine if the March 2012 withdrawal of the US FDA application to sell Replagal was part of an agreement to segment markets globally, to the disadvantage of U.S. patients and taxpayers.

More on Fabry issues at

Media Coverage:

The Fabrazyme FTC documents”: