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The FDA announced Friday that it has approved Sanofi's Enjaymo (sutimlimab-jome) to decrease the need for red blood cell transfusions due to haemolysis in adults with cold agglutinin disease (CAD). The decision follows a complete response letter issued by the FDA for the complement C1s inhibitor in late 2020 citing "deficiencies" that had been uncovered at a third-party manufacturing plant. Sanofi later said it planned to resubmit the filing in the second half of 2021.
The company's application was based on the single-arm Phase III CARDINAL trial, which included 24 CAD patients who had a recent blood transfusion. All participants received Enjaymo infusion for up to six months and could choose to continue therapy in a second part of the trial.
Results showed that 54% of patients met the criteria for the composite primary endpoint. Response was defined as an increase in haemoglobin (Hb) of ≥2 g/dL or of ≥12 g/dL depending on when the treatment assessment endpoint was measured, no red blood cell transfusions after the first five weeks of treatment, and no other therapies for beyond what was permitted per protocol.
The FDA noted that common side effects with Enjaymo are respiratory tract infection, viral infection, diarrhoea, dyspepsia, cough, arthralgia, arthritis and swelling in the lower legs and hands.
List price of $1800 per vial
Sanofi said Enjaymo, which is expected to be available in the US in the coming weeks, will carry a list price $1800 per vial. Bill Sibold, head of specialty care at the drugmaker, noted that "Enjaymo is the only approved treatment to inhibit red blood cell destruction in CAD and help stop the chain reaction from the start."
Last year, Sanofi reported positive data from a second Phase III trial of Enjaymo. Part A of the CADENZA study recruited 42 CAD patients who had not had a blood transfusion within the prior six months, and who were randomly assigned to either Enjaymo or placebo.
On the primary composite endpoint, 73% of patients in the Enjaymo group achieved a response compared to 15% for placebo. Response was defined as a ≥1.5 g/dL improvement in Hb from baseline at specific time points up to week 26, as well as no blood transfusions and no need for CAD-related therapies beyond what was permitted, from weeks 5 through 26.