FDA approves Enjaymo for rare form of anemia
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The FDA approved sutimlimab-jome infusion for the treatment of adults with a rare type of anemia.
Sutimlimab-jome (Enjaymo, Sanofi) is intended to reduce the need for red blood cell transfusion due to hemolysis among adults with cold agglutinin disease (CAD).
CAD is a rare autoimmune disorder characterized by red blood cell destruction. This leads to anemia and other circulatory symptoms, including discoloration of or pain in the toes and fingers. Patients with CAD often require red blood cell transfusions.
Sutimlimab-jome is a humanized monoclonal antibody that selectively inhibits the C1 complex of the classical complement pathway, which activates hemolysis among patients with CAD.
The FDA based approval on results of a study that included 24 adults with CAD who received a blood transfusion in the prior 6 months.
All study participants received sutimlimab-jome for up to 6 months. Depending on body weight, participants received 6.5 g or 7.5 g on day 0, day 7, and every 14 days through week 25. Study participants had the choice to continue therapy in the second part of the trial.
More than half (54%) of study participants responded to treatment. Researchers defined response as an increase in hemoglobin of 2 g/dL or greater; no red blood cell transfusions after the first 5 weeks of treatment; and no other therapies for CAD.
The most common adverse events associated with sutimlimab-jome treatment include respiratory tract infection, viral infection, diarrhea, dyspepsia, cough, arthralgia, arthritis, and swelling in the lower legs and hands.
The FDA previously granted sutimlimab-jome orphan drug designation, breakthrough therapy designation and priority review for this indication.
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