FDA approves Enjaymo™ (sutimlimab-jome), first treatment for use in
patients with cold agglutinin disease
FDA approves Enjaymo™ (sutimlimab-jome), first
treatment for use in patients with cold agglutinin disease
- Enjaymo is the only approved treatment
to decrease the need for red blood cell transfusion due to
hemolysis, the destruction of red blood cells, in adults with cold
agglutinin disease (CAD)
- Enjaymo addresses a serious and
chronic unmet medical need for adults living with CAD, a rare blood
disorder
Paris, February 4, 2022. The
U.S. Food and Drug Administration (FDA) has approved Enjaymo™
(sutimlimab-jome) to decrease the need for red blood cell
transfusion due to hemolysis in adults with cold agglutinin disease
(CAD). Enjaymo is the first and only approved treatment for people
with CAD and works by inhibiting the destruction of red blood cells
(hemolysis).
Bill SiboldExecutive Vice
President, Head of Specialty Care“Until now, people living with
cold agglutinin disease haven’t had an approved treatment option to
manage the constant destruction of red blood cells. 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.”
CAD, a rare autoimmune hemolytic anemia, is
caused by antibodies called cold agglutinins binding to the surface
of red blood cells, which starts a process that causes the body’s
immune system to mistakenly attack healthy red blood cells and
cause their rupture (hemolysis). As red blood cells have the vital
job of carrying oxygen throughout the body, patients with CAD may
experience severe anemia, which can result in fatigue, weakness,
shortness of breath, light-headedness, chest pain, irregular
heartbeat, and other potential complications. CAD is a chronic and
rare blood disorder that impacts the lives of an estimated 5,000
people in the U.S.
Enjaymo, targeting C1s in the classical
complement pathway
Enjaymo is a humanized monoclonal antibody that
is designed to selectively target and inhibit C1s in the classical
complement pathway, which is part of the innate immune system. By
blocking C1s, Enjaymo inhibits the activation of the complement
cascade in the immune system and inhibits C1-activated hemolysis in
CAD to prevent the abnormal destruction of healthy red blood cells.
Enjaymo does not inhibit the lectin and alternative pathways.
Enjaymo Phase 3 pivotal CARDINAL study results
supporting approval
The approval of Enjaymo in the U.S. is based on
positive results from the 26-week open label, single arm pivotal
Phase 3 study in patients with CAD (n=24) who have a recent history
of blood transfusion, also known as the CARDINAL study.
Catherine Broome, MDAssociate
professor of medicine at Georgetown University Lombardi
Comprehensive Cancer Center, and a principal investigator in the
CARDINAL study "For people living with cold agglutinin disease, it
is as if their body’s immune system is waging a war on itself. The
relentless destruction of healthy red blood cells is a daily,
silent reality for people with CAD. 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. In the pivotal study, patients treated with sutimlimab
had an improvement in anemia as measured by hemoglobin and
bilirubin levels during the 26-week study.”
In the study, Enjaymo met its primary efficacy
endpoint, which was a composite endpoint defined as the proportion
of patients who achieved normalization of hemoglobin (Hgb) level
≥12 g/dL or demonstrated an increase from baseline in Hgb level ≥2
g/dL at the treatment assessment time point (mean value from weeks
23, 25, and 26) and no blood transfusion from weeks 5 through 26 or
medications prohibited per the protocol from weeks 5 through 26.
Secondary endpoints were also met, including improvements in
hemoglobin and normalization of bilirubin.
- The majority of patients (54%;
n=13) met the composite primary endpoint criteria with 63% (n=15)
of patients achieving a hemoglobin ≥ 12 g/dL or an increase of at
least 2 g/dL; 71% (n=17) of patients remaining transfusion-free
after week five; and 92% (n=22) of patients did not use other
CAD-related treatments.
- For the secondary measures on disease
process, patients enrolled experienced a mean increase in
hemoglobin level of 2.29 g/dL (SE: 0.308) at week 3 and 3.18 g/dL
(SE: 0.476) at the 26-week treatment assessment timepoint from the
mean baseline level of 8.6 g/dL. The mean reduction in bilirubin
levels (n=14) was by -2.23 mg/dL (95% CI: -2.49 to -1.98) from a
mean baseline level of 3.23 mg/dL (2.7-fold ULN).
In the CARDINAL study, the most common adverse
reactions occurring in 10 percent or more of patients were
respiratory tract infection, viral infection, diarrhea, dyspepsia,
cough, arthralgia, arthritis, and peripheral edema. Serious adverse
reactions were reported in 13 percent (3/24) of patients who
received Enjaymo. These serious adverse reactions were
streptococcal sepsis and staphylococcal wound infection (n=1),
arthralgia (n=1), and respiratory tract infection (n=1). None of
the adverse reactions led to discontinuation of Enjaymo in the
study. Dosage interruptions due to an adverse reaction occurred in
17 percent (4/24) of patients who received Enjaymo.
Following the completion of the 26-week
treatment period of CARDINAL (Part A), eligible patients continued
to receive Enjaymo in an extension study.
The recommended dose of Enjaymo is based on body
weight (6,500 mg for people 39-75 kg and 7,500 mg for people >75
kg). Enjaymo is administered intravenously weekly for the first two
weeks with administration every two weeks thereafter.
Enjaymo is expected to be available in the U.S. in
the coming weeks. The U.S. list price, or wholesale acquisition
cost, of Enjaymo is $1,800 per vial. Actual costs to patients are
generally anticipated to be lower as the list price does not
reflect insurance coverage, co-pay support, or financial assistance
from patient support programs. As part of our commitment to ensure
treatment access and affordability for innovative therapies,
Enjaymo Patient Solutions provides disease education, financial and
co-pay assistance programs and other support services to eligible
patients. For more information, please call 1-833-223-2428.
Enjaymo received FDA Breakthrough Therapy and
Orphan Drug designation, and priority review, which is reserved for
medicines that, if approved, would represent significant
improvements in safety or efficacy in treating serious conditions.
Outside of the U.S., sutimlimab has been submitted to regulatory
authorities in Europe and Japan and reviews are ongoing.
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