FDA Denies Approval of Sanofi’s Treatment for Cold Agglutinin Disease

Rare Daily Staff

The U.S. Food and Drug Administration said it would not approve Sanofi’s Biologics License Application for sutimlimab, an experimental monoclonal antibody for the treatment of hemolysis in adults with cold agglutinin disease, until a third party manufacturer addresses certain deficiencies.

Sanofi said the complete response letter refers to certain deficiencies identified by the agency during a pre-license inspection of a third-party facility responsible for manufacturing and noted that there were no clinical or safety deficiencies noted in the letter with respect to the application.

Cold agglutinin disease (CAD) is a rare form of autoimmune hemolytic anemia where the immune system makes antibodies that attack the body’s own red blood cells, causing them to die prematurely (hemolysis). Symptoms may include pallor and fatigue, back and leg pain, headache, vomiting, diarrhea, and dark urine. CAD gets its name from the fact that the antibodies react at temperatures below normal body temperature, so a cold environment may trigger the condition or make it worse. Treatment often involves avoidance of cold temperatures.

Sutimlimab is a C1 inhibitor that Sanofi acquired through its $11.6 billion purchase of Biogen spinout Bioverativ and its hemophilia portfolio in early 2018.

“Satisfactory resolution of the observations by the third-party manufacturer is required before the BLA can be approved and Sanofi remains in close contact with the FDA and the third-party manufacturer to reach a resolution in a timely manner,” the company said.