BioCentury
ARTICLE | Top Story

FDA reviewers do not back Lemtrada

November 9, 2013 1:54 AM UTC

FDA reviewers said there are "significant concerns" on the safety profile and adequacy of efficacy data for Lemtrada alemtuzumab from the Genzyme Corp. unit of Sanofi (Euronext:SAN; NYSE:SNY) to treat relapsing multiple sclerosis (MS). Reviewers said there are "serious and potentially fatal safety issues" with Lemtrada, highlighting the incidence of autoimmune diseases, including idiopathic thrombocytopenia purpura (ITP), and malignancies, including thyroid cancer and melanoma, in MS patients. Reviewers also said these risks "may not be balanced" by the efficacy data that Genzyme submitted, which the reviewers said was "limited." The pivotal trials for Lemtrada -- the Phase II CAMMS223 trial and Phase III CARE-MS I and CARE-MS II trials -- had an open-label design, which the reviewers said raises "uncertainty" about the data.

The comments came in MabCampath/ Campath alemtuzumab from the European and U.S. markets to treat B cell chronic lymphocytic leukemia (CLL) to prepare for commercialization of the drug for MS. At the time, the company told BioCentury it decided "to focus on bringing alemtuzumab forward for MS," and that restricting access will help prevent off-label use of the drug in this indication. The U.S. label for Campath includes a black box warning on the risk of serious cytopenias, including ITP, as well as infusion reactions and infections (see BioCentury, Aug. 27, 2012). ...