A first-in-class cold agglutin disease treatment that won approval based on clinically confirmed efficacy and safety has arrived in Korea.
On Wednesday, the Ministry of Food and Drug Safety said it approved Sanofi’s Enjaymo (sutimlimab) to treat hemolysis in adult patients with cold agglutinin disease.
Enjaymo is a first-in-class humanized monoclonal antibody targeting the C1 protein, which activates the classical complement pathway. It has been confirmed to increase hemoglobin levels while reducing hemolysis and extreme fatigue in patients with cold sores.
Cold agglutinin is an extremely rare autoimmune blood disorder where the body’s immune system attacks its red blood cells, causing persistent and recurrent red blood cell destruction. When patients are exposed to below body temperature, it causes anemia due to chronic hemolysis, extreme fatigue, shortness of breath, hemoglobinuria, cyanosis of the extremities, and thromboembolism, with a median survival time of 8.5 years after diagnosis.
Cold agglutinin disease is extremely rare, affecting 1 in 1 million people annually. It does not even have a disease code in Korea, making it difficult to count the number of patients accurately.
Enjaymo has just won approval in Korea based on the results of two clinical studies confirming the efficacy and safety profile in adult patients with cold agglutinin.
In February 2022, Enjaymo also won orphan drug status, priority review, and breakthrough therapy designations by the U.S. Food and Drug Administration in recognition of this efficacy and safety profile. It then got the nod from the Japanese Ministry of Health, Labor and Welfare in June 2022 and the European Commission in November 2022 for widespread use.
“Until now, patients in Korea have been suffering from risky interventions, such as blood transfusions and unapproved drugs with unconfirmed efficacy and safety profiles, as there are no approved drugs for the treatment of cold agglutinin,” said Park Hee-kyung, Sanofi-Aventis Korea’s president for specialty care. “We are pleased to be able to introduce the first drug with a confirmed efficacy and safety profile to Korean patients with cold agglutinin.”
Park added that Sanofi will continue to work to raise awareness of the disease and improve access to treatment so that patients in Korea can receive the best possible care and management.