Genzyme gets FDA Complete Response Letter for Lemtrada sBLA

Genzyme, a Sanofi company (EURONEXT:SAN and NYSE:SNY), announced today that it has received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) for its supplemental Biologics License Application seeking approval of Lemtrada (alemtuzumab) for the treatment of relapsing forms of multiple sclerosis.

A Complete Response Letter informs companies that an application is not ready for approval. FDA has taken the position that Genzyme has not submitted evidence from adequate and well-controlled studies that demonstrate the benefits of Lemtrada outweigh its serious adverse effects. Genzyme understands that the conclusion is related to the design of the completed Phase 3 active comparator studies of Lemtrada in relapsing-remitting MS patients. FDA has also taken the position that one or more additional active comparator clinical trials of different design and execution are needed prior to the approval of Lemtrada.

Genzyme strongly disagrees with the FDA's conclusions and plans to appeal the agency's decision.

"We are extremely disappointed with the outcome of the review and the implications for patients in the U.S. suffering with multiple sclerosis who remain in need of alternative therapies to manage a devastating disease," said Genzyme President and CEO, David Meeker, M.D. "We strongly believe that the clinical development program, which was designed to demonstrate how Lemtrada compares against an active comparator as opposed to placebo, provides robust evidence of efficacy and a favorable benefit-risk profile. This evidence was also the basis for the approvals of Lemtrada by other regulatory agencies around the world."

Lemtrada is approved in the European Union, Canada, and Australia, and additional marketing applications for Lemtrada are under review by regulatory agencies around the world.

Sanofi does not anticipate that the CVR milestone of U.S. approval of Lemtrada by March 31, 2014 will be met.