Targeted failure of the week. Post No 123. Lemtrada

Sanofi (SAN)’s Lemtrada hasn’t proven to help against disability in the treatment of relapsing multiple sclerosis, said U.S. advisers who questioned whether the company’s studies were conducted well enough to assess the drug.

 

While the U.S. Food and Drug Administration advisory panel decided that potential safety risks don’t preclude approval of Lemtrada, its members voted 14-2 that the drug didn’t help improve a patient’s disability. The agency isn’t required to accept the recommendations of its advisers.

Lemtrada, approved in Europe earlier this year, was a key part of Paris-based Sanofi’s $20 billion acquisition of Genzyme Corp. in 2011. The drug if cleared in the U.S. would enter a crowded field in which relapsing MS patients now have 10 treatment options with varying degrees of efficacy, including Biogen Idec Inc. (BIIB)’s Tecfidera and Teva Pharmaceutical Industries Ltd. (TEVA)’s Copaxone.

 

“If the study is biased, then everything that flows from the study can’t be trusted,” Robert Clancy, a panel member and professor of neurology and pediatrics at the University of Pennsylvania School of Medicine, said at yesterday’s meeting. The panel voted 11-6, with one abstention, that the two large studies Sanofi submitted on the drug’s behalf weren’t adequate and well-controlled.

 

Alemtuzumab (marketed as Campath, MabCampath or Campath-1H and currently under further development as Lemtrada) is a monoclonal antibody used in the treatment of chronic lymphocytic leukemia (CLL), cutaneous T-cell lymphoma (CTCL) and T-cell lymphoma. It is also used in some conditioning regimens for bone marrow transplantation, kidney transplantation and Islet cell transplantation.

 

Alemtuzumab binds to CD52, a protein present on the surface of mature lymphocytes, but not on the stem cells from which these lymphocytes are derived. After treatment with alemtuzumab, these CD52-bearing lymphocytes are targeted for destruction.