The US FDA has approved Sanofi’s Enjaymo (sutimlimab-jome) for the treatment of adults diagnosed with the rare blood disorder, cold agglutinin disease (CAD).
This is the first treatment to be approved for patients with CAD and its approval was based on positive results from the pivotal phase 3 CARDINAL study.
CAD is a rare form of autoimmune haemolytic anaemia and is caused by cold agglutinins (antibodies) attaching to the surface of red blood cells. This begins a process which causes the body’s immune system to attack healthy red blood cells, leading to cell destruction known as haemolysis.
Patients with CAD are likely to suffer from severe anaemia, leading to fatigue, shortness of breath, light-headedness and chest pains, among other symptoms, due to the blood cells’ inability to carry oxygen around the body. It is estimated that CAD affects the lives of around 5,000 people in the US.
Enjaymo prevents the destruction of healthy red blood cells and therefore significantly reduces the need for patients with CAD to have red blood cell transfusions.
Bill Sibold, Sanofi’s executive vice president and head of Specialty Care said: “Until now, people living with cold agglutinin disease haven’t had an approved treatment option to manage the constant destruction of red blood cells. Without healthy, viable red blood cells, a chain reaction of debilitating signs and symptoms can be triggered, starting with severe anaemia.”
Catherine Broome, associate professor of medicine at Georgetown University Lombardi Comprehensive Cancer Center and a principal investigator in the CARDINAL study, said: “The relentless destruction of healthy red blood cells is a daily, silent reality for people with CAD. For the first time, we have a treatment that targets complement-mediated hemolysis, which is the underlying cause of the red blood cell destruction in many CAD patients.”
In the US, Enjaymo is expected to be available in the following weeks, with a list price of $1,800 per vial. The actual cost for patients is expected to be lower than this due to financial assistance from patient support programmes and insurance coverage.