Genzyme fails to win FDA approval for MS drug Lemtrada

Agency demands additional clinical trials from Sanofi subsidiary

CAMBRIDGE, Mass. — The Food and Drug Administration has declined to approve a drug made by Genzyme Corp. for certain forms of multiple sclerosis, the company said Monday.

Genzyme, a subsidiary of French drug maker Sanofi, said it received a complete response letter from the FDA for Lemtrada (alemtuzumab) as a treatment for relapsing MS. A complete response letter means that the FDA has finished reviewing a regulatory application, but can't approve it due to questions that remain. In Genzyme's case, the FDA said the company had not submitted sufficient evidence demonstrating that Lemtrada's benefits outweigh serious potential side effects, and that it needs at least one clinical trial of a different design from the ones already performed.

"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," Genzyme president and CEO David Meeker said. "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."

Genzyme said it "strongly disagrees" with the agency's decision and plans to appeal it, adding that its goal of gaining U.S. approval for the drug by March 31 will not be met. Lemtrada is already approved in the European Union, Canada and Australia.

"This evidence was also the basis for the approvals of Lemtrada by other regulatory agencies around the world," Meeker said, referring to the existing clinical trials.

Sanofi's stock fell by 0.87% to $52.34 per share Monday morning on the New York Stock Exchange, following the news.

Lemtrada was a centerpiece of Sanofi's $20.1 billion purchase of Genzyme in 2011, with Genzyme shareholders set to receive milestone payments related to the drug. Genzyme, which had become a leader in treatments for rare diseases but had run into production problems, had been an attractive acquisition target for Sanofi, then known as Sanofi-Aventis, since 2010, and the French drug maker said it planned to make Genzyme its "global center for excellence" in rare diseases.

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