The Food and Drug Administration (FDA) has rejected Sanofi’s Genzyme unit’s bid for the multiple sclerosis (MS) drug alemtuzumab (Lemtrada), citing inadequate data and concerns about life threatening side effects. The drug company says it disagrees with the FDA’s decision and will appeal.
MS is a debilitating autoimmune disease that affects the central nervous system, causing symptoms such as coordination problems and spasticity. Alemtuzumab is a novel treatment designed to down-regulate the inflammatory activity in MS. It is administered as an initial five-day course of infusions followed by a second three-day course a year later.
The drug was pitted against beta-interferon, a standard treatment for relapsing-remitting MS, in two clinical trials, during which alemtuzumab was found to be better at reducing relapses as well as reducing progression of disability. However, the drug has been associated with serious and potentially fatal safety issues including risk of cancer as well as autoimmune and thyroid diseases.
Despite those concerns, an FDA advisory committee in November voted to recommend approval. The FDA is not required to follow the recommendations of its expert panels but it usually does. In this case, though, the agency said it needed evidence from adequate and well-controlled studies that demonstrate the benefits of the drug outweigh the potential risks before it could approve medication.
Alemtuzumab was approved by the European Union in September followed by drug regulators in Australia and Canada.