Enjaymo’s Now Approved for US Patients With No Prior Transfusions

Therapy first approved last year for patients who had received blood transfusions

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Note: This story was updated Jan. 30, 2023,  to correct that the CARDINAL trial did not include a placebo group.

The U.S. Food and Drug Administration (FDA) has approved an expansion of the indication for Enjaymo (sutimlimab-jome) to include cold agglutinin disease (CAD) patients without a history of blood transfusions.

The therapy was first approved by the FDA nearly a year ago for patients who had previously received blood transfusions. The label expansion also allows Enjaymo to be available in an undiluted preparation, meaning the infusion time can be reduced to about an hour.

“This milestone underscores our ongoing commitment to improve outcomes for the rare blood disorder community,” Bill Sibold, executive vice president and head of specialty care at Sanofi, which markets Enjaymo, said in a company press release.

“As a first-in-class therapy, Enjaymo can help people living with cold agglutinin disease better manage their symptoms, including the impact of fatigue, which may positively affect their daily life,” Sibold added.

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Enjaymo’s label expansion mainly based on data from Phase 3 CADENZA trial

CAD is an autoimmune disease caused by abnormal immune responses that attack and destroy red blood cells. Enjaymo works to block the activity of the complement cascade, a part of the immune system that plays a critical role in this autoimmune attack. The therapy is administered via an infusion into the bloodstream every other week.

The FDA’s initial approval of Enjaymo was based mainly on data from the Phase 3 CARDINAL clinical trial (NCT03347396), which tested the medication in 24 people with CAD who’d had a recent blood transfusion.

Results showed that six months of treatment with Enjaymo reduced anemia, prevented red blood cell destruction, and improved patients’ quality of life.

The label expansion is supported primarily by data from another Phase 3 trial called CADENZA (NCT03347422), which enrolled 42 CAD patients who had not received a blood transfusion in the six months prior to entering the study.

Six months of Enjaymo treatment were found to outperform a placebo at easing anemia and preventing a need for blood transfusions. Participants treated with Enjaymo also reported less fatigue and improvements in life quality, while safety data were generally consistent with results from earlier studies.

The most common side effects reported in CADENZA included sneezing or a congested, drippy nose, headache, high blood pressure, Raynaud’s phenomenon, and acrocyanosis (bluish discoloration of the skin).

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Data so far support Enjaymo’s safety profile and sustained efficacy

Participants who completed the initial placebo-controlled portion of the CADENZA study had the option to enroll in the open-label extension portion, where all received Enjaymo for longer periods.

Available data so far support Enjaymo’s favorable safety profile and sustained efficacy for up to 2.5 years.

Notably, in both CADENZA and CARDINAL studies, recurrence of CAD symptoms was observed about two months after patients received their last Enjaymo dose in the extension portion, according to Sanofi.

Enjaymo was approved by the European Commission to treat CAD patients in December. That approval, based on data from both CARDINAL and CADENZA, did not specify transfusion status in the indication.