CHMP recommends approval of Enjaymo first and only approved treatment for hemolytic anemia in adult patients with cold agglutinin disease

CAD is a rare, serious, and chronic autoimmune hemolytic anemia.

The positive CHMP opinion is based on data from two Phase III clinical trials: CADENZA, a double-blind, placebo-controlled clinical trial of adults with CAD without a recent history of blood transfusion (within the past 6 months), and CARDINAL, a 26-week open label, single-arm pivotal study in patients with CAD who have had a recent blood transfusion. In the CADENZA trial, eligible patients were randomized 1:1 to receive a fixed weight-based dose (6.5g or 7.5g) of sutimlimab or placebo via intravenous infusion on Day 0, Day 7 and then once every other week up to Week 26. The positive results of the study were presented at the European Hematology Association (EHA) 2021 Congress. The open-label Part B of the study assessed long-term safety as well as durability of response to sutimlimab in patients with CAD.

In the CARDINAL trial, patients received a fixed weight-based dose (6.5g or 7.5g) of sutimlimab via intravenous infusion on Day 0, Day 7 and then once every other week up to Week 26. The positive results were presented at the Late-Breaking Abstracts Session of the 61st Annual Meeting of the American Society of Hematology in 2019. Part B of the study evaluated the long-term safety as well as durability of response to sutimlimab in patients with CAD over a 2-year follow up and the positive results were presented at EHA 2022.