FDA rejects MS drug

FDA has rejected alemtuzumab (Lemtrada, Genzyme, a Sanofi company) for the treatment of relapsing forms of multiple sclerosis.

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 1 or more additional active comparator clinical trials of different design and execution are needed prior to the approval of Lemtrada

"FDA's request for Sanofi's Genzyme  to submit additional evidence to demonstrate that benefits outweigh the risks of the drug alemtuzumab [Lemtrada] for multiple sclerosis is not only designed safeguard the welfare of the general public, but also illustrates the strict and high level evaluations that are required before approval is given to any drug for marketing," said Abimbola Farinde, PharmD, MS, who serves on the faculty at Columbia Southern University, Orange Beach, Ala.

Genzyme strongly disagrees with 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, MD, in a company press release. “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 US approval of Lemtrada by March 31, 2014 will be met.