FDA has approved Enjaymo (sutimlimab-jome) infusion to decrease the need for red blood cell transfusion due to hemolysis (red blood cell destruction) in adults with cold agglutinin disease (CAD).
Disease or Condition
CAD is an autoimmune disorder characterized by red blood cell destruction, which leads to anemia (a decreased number of red blood cells) and cold-induced circulatory symptoms, such as pain and discoloration of fingers or toes. The disease is called “cold” agglutinin disease because the red cell destruction occurs at cold temperatures. Patients with CAD have a range in severity of anemia symptoms, which include fatigue, weakness, shortness of breath, fast heart rate, dizziness, and chest pain. Many patients with CAD need red blood cell transfusions to manage their disease. The disease is rare, affecting about one person per million annually, and mostly develops in individuals between ages 40 and 80 years.
The effectiveness of Enjaymo was assessed in a study of 24 adults with CAD who had a blood transfusion within the past six months. All participants received Enjaymo for up to six months and could choose to continue therapy in a second part of the trial. Based on body weight, participants received either a 6.5g or 7.5g infusion of Enjaymo into their vein on day 0, day 7, and every 14 days through week 25.
In total, 54% of participants responded to Enjaymo. The response was defined in the study as:
- an increase in hemoglobin (an indirect measurement of the amount of red blood cells that are not destroyed) of 2 g/dL or greater (or to 12 g/dL or greater), and
- no red blood cell transfusions after the first five weeks of treatment; and no other therapies for CAD as defined in the study
Patients with allergies to sutimlimab-jome or any of the inactive ingredients must not take Enjaymo. Individuals should be vaccinated against encapsulated bacteria and monitored for early signs and symptoms of infection. Patients also should be monitored for infusion reactions; signs and symptoms of autoimmune disease; and signs and symptoms of hemolysis recurrence if treatment is interrupted.
The most common side effects Enjaymo are respiratory tract infection, viral infection, diarrhea, dyspepsia (indigestion), cough, arthralgia (joint stiffness), arthritis, and swelling in the lower legs and hands.
Please see the prescribing information for additional information on the risks associated with Enjaymo.
This application received orphan drug designation, breakthrough therapy designation and priority review.