Enjaymo Becomes 1st Treatment Approved in US for Adults With CAD
The U.S. Food and Drug Administration (FDA) has approved Enjaymo (sutimlimab-jome) to decrease the need for red blood cell transfusions due to hemolysis, or red blood cell destruction, in adults with cold agglutinin disease (CAD).
Enjaymo, which works by preventing hemolysis, is the first and only FDA-approved therapy for CAD, according to its developer, Sanofi.
“Until now, people living with cold agglutinin disease haven’t had an approved treatment option to manage the constant destruction of red blood cells,” Bill Sibold, Sanofi’s executive vice president and head of specialty care, said in a press release.
“Without healthy, viable red blood cells, a chain reaction of debilitating signs and symptoms can be triggered, starting with severe anemia. Enjaymo is the only approved treatment to inhibit red blood cell destruction in CAD and help stop the chain reaction from the start,” Sibold added.
Red blood cells are responsible for transporting oxygen through the bloodstream and delivering it to tissues. CAD is caused by the immune system erroneously launching an attack that destroys these cells through hemolysis.
“For people living with cold agglutinin disease, it is as if their body’s immune system is waging a war on itself,” said Catherine Broome, MD, associate professor of medicine at Georgetown University Lombardi Comprehensive Cancer Center. “The relentless destruction of healthy red blood cells is a daily, silent reality for people with CAD.”
Part of the immune attack that causes hemolysis in CAD is driven by a group of immune proteins that make up the complement cascade. Enjaymo blocks a complement protein called C1, effectively preventing hemolysis driven by the excessive activity of the complement cascade.
“For the first time, we have a treatment that targets complement-mediated hemolysis, which is the underlying cause of the red blood cell destruction in many CAD patients,” Broome said.
Enjaymo’s approval was supported by data from a Phase 3 trial called CARDINAL (NCT03347396), which was sponsored by Bioverativ (a Sanofi company). The trial enrolled 24 people with CAD who were treated with Enjaymo for 26 weeks, or about six months.
The study’s main goal was to determine the effect of treatment on the levels of hemoglobin — the protein that red blood cells use to carry oxygen. Over half (54%) of participants met the primary endpoint, with hemoglobin levels rising by at least two grams per deciliter (g/dL) or remaining at a minimum of 12 g/dL, while requiring no blood transfusions from weeks five through 26.
More specifically, 63% of patients had hemoglobin levels of at least 12 g/dL or saw them increase by at least 2 g/dL, and 71% did not need blood infusions after week five. Over 90% of patients were off of other CAD treatments.
Hemoglobin levels increased by a mean of more than 3 g/dL over the 26-week study. Levels of bilirubin — a waste product produced when hemoglobin breaks down during hemolysis — decreased with treatment, by a mean of 2.23 mg/dL over 26 weeks.
“In the pivotal study, patients treated with sutimlimab [Enjaymo] had an improvement in anemia as measured by hemoglobin and bilirubin levels during the 26-week study,” said Broome.
The most common adverse events reported in CARDINAL included respiratory tract infection, viral infection, diarrhea, indigestion, cough, joint pain, arthritis, and swelling. Three patients given Enjaymo experienced serious adverse events, which included sepsis, a bacterial wound infection, and a respiratory tract infection.
None of these adverse events led to treatment discontinuations. Treatment interruptions due to an adverse event occurred in four patients who received Enjaymo.
Enjaymo is administered via infusion into the bloodstream every week for two weeks, and every other week thereafter. The recommended dose is 6,500 mg for people who weigh 39–75 kg (about 86–165 lbs) and 7,500 mg for those who weighing more than 75 kg.
The therapy is expected to be available in the coming weeks. It will be sold in vials containing 1,100 mg of the active ingredient.
According to Sanofi, the U.S. list price of Enjaymo is $1,800 per vial. The company noted that the full price is not typically paid by patients, since it does not account for insurance coverage or financial assistance programs.
Sanofi is offering a program called Enjaymo Patient Solutions to provide education, financial support, and other assistance services to patients. Additional information is available by phone at 1-833-223-2428.
Enjaymo had been given priority review by the FDA, as well as orphan drug and breakthrough therapy designations. Orphan drug designation entitles Sanofi to seven years of market exclusivity now that the therapy has been approved.
The treatment is also under consideration for possible approval in Europe and Japan.