Genzyme, a Sanofi company, announced today that the United States Food and Drug Administration (FDA) has accepted for review the supplemental Biologics License Application (sBLA) for Lemtrada™ (alemtuzumab), a proposed treatment for relapsing forms of multiple sclerosis (MS). This application was recently submitted after the FDA did not approve Lemtrada in late December 2013, when Genzyme stated that they had received a Complete Response Letter from the FDA. This letter informed them that the application for their drug was “not ready for approval.” A new decision is expected in the last quarter of 2014.
The reason that the drug did not receive approval, according to the FDA, lies in the design of the Phase III studies. These compared the effectiveness and adverse effects of Lemtrada against an approved treatment for MS, versus a placebo (with no active ingredients). Because of dramatically different side effects between the two different medications, these studies were “unblinded,” meaning that both the patients and treating doctors were aware of which drug each of the patients was receiving. Whenever possible, clinical trials are double-blinded to prevent any potential bias toward the treatment being evaluated.
In order to resubmit the application, Genzyme and the FDA had “constructive discussions” to determine what would be necessary for the medication to be reconsidered for approval. In the resubmitted application, Genzyme provided additional data to address the specific issues that are of concern to the FDA. These data are from the same studies that were included in the original application.
Lemtrada is a humanized monoclonal antibody given in annual courses by intravenous (IV) infusion. It works by depleting the circulating lymphocytes (B and T cells). Lymphocytes are immune-system cells produced to fight infection and disease, and with MS, these cells are misdirected to cause damage to the nerves of the central nervous system (CNS) and the nerves’ protective covering (myelin). Lemtrada was originally approved by the FDA in 2001 for the treatment of a certain type of leukemia (B-cell chronic lymphocytic leukemia), using “Campath” as the trade name.
Lemtrada is already approved in 33 countries for the treatment of MS, including 28 countries in the European Union. Additional marketing applications for this MS medication are under review by other regulatory agencies around the world.
Lemtrada™ (alemtuzumab, formerly Campath) did not receive approval from the United States Food and Drug Administration (FDA) for the treatment of relapsing forms of multiple sclerosis (MS) in December, 2013 because of the design of the Phase III studies.