US FDA approves Sanofi’s Enjaymo for cold agglutinin disease treatment

The US Food and Drug Administration (FDA) has approved Sanofi’s Enjaymo (sutimlimab-jome) to treat cold agglutinin disease (CAD) in adult patients.

Enjaymo is intended to reduce the need for red blood cell transfusion caused due to hemolysis in CAD patients.

The drug is a humanised monoclonal antibody that selectively targets and inhibits C1s in the classical complement pathway that is part of the innate immune system.

It blocks C1s and inhibits C1-activated haemolysis in CAD patients for the prevention of abnormal destruction of healthy red blood cells.

The company claimed that the drug is the first and only treatment approved for CAD patients and inhibits the destruction of red blood cells (hemolysis).

Sanofi executive vice-president and Speciality Care head Bill Sibold 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.

“Enjaymo is the only approved treatment to inhibit red blood cell destruction in CAD and help stop the chain reaction from the start.”

The regulatory approval was based on the data obtained from the Phase III pivotal CARDINAL trial, a 26-week single-arm, open-label pivotal study conducted in patients with CAD who received a blood transfusion.

Enjaymo met the primary efficacy endpoint as well as the secondary endpoints of the study, including improvements in haemoglobin and normalisation of bilirubin.

The most common adverse reactions in the CARDINAL study included diarrhoea, dyspepsia, viral infection, respiratory tract infection, cough, arthralgia, arthritis, and peripheral oedema.